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Two New Treatments for IPF

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FDA approves Esbriet to treat idiopathic pulmonary fibrosis.

October 15, 2014

The U.S. Food and Drug Administration today approved Esbriet (pirfenidone) for the treatment of idiopathic pulmonary fibrosis (IPF).

Idiopathic pulmonary fibrosis is a condition in which the lungs become progressively scarred over time. As a result, patients with IPF experience shortness of breath, cough, and have difficulty participating in everyday physical activities. Current treatments for IPF include oxygen therapy, pulmonary rehabilitation, and lung transplant.

“Esbriet provides a new treatment option for patients with idiopathic pulmonary fibrosis, a serious, chronic lung disease,” said Curtis J. Rosebraugh, M.D., M.P.H., director of the Office of Drug Evaluation II in the FDA’s Center for Drug Evaluation and Research. “We continue to help advance medication therapies by approving products that treat conditions that impact public health.”

The FDA granted Esbriet fast track, priority review, orphan product, and breakthrough designations. Esbriet is being approved ahead of the product’s prescription drug user fee goal date of Nov. 23, 2014, the date the agency was scheduled to complete the review of the drug application.

Esbriet acts on multiple pathways that may be involved in the scarring of lung tissue. Its safety and effectiveness were established in three clinical trials of 1,247 patients with IPF. The decline in forced vital capacity – the amount of air which can be forcibly exhaled from the lungs after taking the deepest breath possible – was significantly reduced in patients receiving Esbriet compared to patients receiving placebo.

Esbriet is not recommended for patients who have severe liver problems, end-stage kidney disease, or who require dialysis. Esbriet should be taken with food to minimize the potential for nausea and dizziness. Patients should avoid or minimize exposure to sunlight and sunlamps and wear sunscreen and protective clothing, as Esbriet may cause patients to sunburn more easily.

The most common side effects of Esbriet are nausea, rash, abdominal pain, upper respiratory tract infection, diarrhea, fatigue, headache, dyspepsia, dizziness, vomiting, decreased/loss of appetite, gastro-esophageal reflux disease, sinusitis, insomnia, decreased weight, and arthralgia.

The FDA also today approved Ofev (nintedanib) for the treatment of IPF.

Esbriet is manufactured for InterMune, Inc., Brisbane, California.

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.




FDA approves Ofev to treat idiopathic pulmonary fibrosis.

October 15, 2014

The U.S. Food and Drug Administration today approved Ofev (nintedanib) for the treatment of idiopathic pulmonary fibrosis (IPF).

Idiopathic pulmonary fibrosis is a condition in which the lungs become progressively scarred over time. As a result, patients with IPF experience shortness of breath, cough, and have difficulty participating in everyday physical activities. Current treatments for IPF include oxygen therapy, pulmonary rehabilitation, and lung transplant.

“Today’s Ofev approval expands the available treatment options for patients with idiopathic pulmonary fibrosis, a serious, chronic condition,” said Mary H. Parks, M.D., deputy director of the Office of Drug Evaluation II in the FDA’s Center for Drug Evaluation and Research. “Providing health care professionals and patients with additional treatment options helps enable appropriate care decisions based on a patient’s need.”

The FDA granted Ofev fast track, priority review, orphan product, and breakthrough designations. Ofev is being approved ahead of the product’s prescription drug user fee goal date of Jan. 2, 2015, the date the agency was scheduled to complete the review of the drug application.

Ofev is a kinase inhibitor that blocks multiple pathways that may be involved in the scarring of lung tissue. Its safety and effectiveness were established in three clinical trials of 1,231 patients with IPF. The decline in forced vital capacity – the amount of air which can be forcibly exhaled from the lungs after taking the deepest breath possible – was significantly reduced in patients receiving Ofev compared to patients receiving placebo.

Ofev is not recommended for patients who have moderate to severe liver problems. Ofev can cause birth defects or death to an unborn baby. Women should not become pregnant while taking Ofev. Women who are able to get pregnant should use adequate contraception during and for at least three months after the last dose of Ofev.

The most common side effects of Ofev are diarrhea, nausea, abdominal pain, vomiting, liver enzyme elevation, decreased appetite, headache, decreased weight, and high blood pressure.

The FDA also today approved Esbriet (pirfenidone) for the treatment of IPF.

Ofev is distributed by Boehringer Ingelheim Pharmaceuticals, Inc., Ridgefield, Connecticut.

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.




InterMune Announces Expanded Access Program for Pirfenidone to Treat Idiopathic Pulmonary Fibrosis (IPF) in the United States

Date(s): 5/16/14 8:00 AM

For a complete listing of InterMune news releases, please click here

BRISBANE, Calif., May 16, 2014 /PRNewswire/ — InterMune, Inc. (NASDAQ: ITMN) today announced it will provide compassionate use of pirfenidone through a multi-center Expanded Access Program (EAP) in the United States to be conducted under InterMune’s U.S. IND. Pirfenidone is an investigational therapy in the U.S. and has not been approved by the U.S. Food and Drug Administration (FDA).

Expanded access programs provide a mechanism for early access to an investigational drug in the pre-approval period to treat patients with a serious or immediately life-threatening disease or condition that has no comparable or satisfactory alternative treatment options.

“We are pleased to offer this expanded access program for eligible patients in the U.S.,” said Jonathan Leff, M.D., Executive Vice President of Research and Development, InterMune. “This EAP provides a mechanism for eligible patients to access pirfenidone as a treatment option, following the recent successful completion of our ASCEND Phase 3 trial and prior to FDA’s final decision on the approvability of pirfenidone in the United States.”

To enroll in the EAP, a patient must meet specific clinical criteria. Eligible patients must have a clinical and radiographic diagnosis of IPF with the presence of a usual interstitial pneumonia (UIP) pattern on high-resolution computed tomography (HRCT). Additional criteria for the EAP are listed on www.clinicaltrials.gov. It is important to note that only a physician who is participating in the EAP can assess a potential patient for eligibility. The EAP protocol contains provisions for stopping enrollment of patients in the EAP upon a decision by the FDA on the approvability of a pirfenidone New Drug Application (NDA).

There are currently a limited number of sites accepting patients for enrollment to the pirfenidone EAP and InterMune expects that all sites will be participating by September of 2014. InterMune is working with the Pulmonary Fibrosis Foundation (PFF), the Coalition for Pulmonary Fibrosis (CPF) and other advocacy groups to enable patients with IPF to obtain information about the pirfenidone EAP.

For more information about the pirfenidone EAP, including eligibility criteria and participating clinical centers, contact InterMune Medical Information at 888-486-6411 or the Pulmonary Fibrosis Foundation (PFF) at 844-TalkPFF (844-825-5733) or visit www.clinicaltrials.gov.

About IPF
Idiopathic pulmonary fibrosis (IPF) is an irreversible and ultimately fatal disease characterized by progressive loss of lung function due to fibrosis (scarring) in the lungs, which hinders the ability of lungs to absorb oxygen. IPF inevitably causes shortness of breath, and a deterioration in lung function and exercise tolerance. IPF patients follow different and unpredictable clinical courses and it is not possible to predict if a patient will progress slowly or rapidly, or when the rate of decline may change. Periods of transient clinical stability in IPF, when they occur, inevitably give way to continued disease progression. The median survival time from diagnosis is two to five years, with a five-year survival rate of approximately 20-40 percent, which makes IPF more rapidly lethal than many malignancies, including breast, ovarian and colorectal cancers. IPF typically occurs in patients over age 45, and tends to affect slightly more men than women.

About Pirfenidone
Pirfenidone is an orally active, anti-fibrotic agent that inhibits the synthesis of TGF-beta, a chemical mediator that controls many cell functions including proliferation and differentiation, and plays a key role in fibrosis. Pirfenidone also inhibits the synthesis of TNF-alpha, a cytokine that is known to have an active role in inflammation.

About InterMune
InterMune is a biotechnology company focused on the research, development and commercialization of innovative therapies in pulmonology and orphan fibrotic diseases. In pulmonology, the company is focused on therapies for the treatment of idiopathic pulmonary fibrosis (IPF), a progressive, irreversible, unpredictable and ultimately fatal lung disease. Pirfenidone is not approved for marketing in the United States. InterMune’s research programs are focused on the discovery of targeted, small-molecule therapeutics and biomarkers to treat and monitor serious pulmonary and fibrotic diseases. For additional information about InterMune and its R&D pipeline, please visit www.intermune.com.

Forward-Looking Statements
This news release contains forward-looking statements within the meaning of section 21E of the Securities Exchange Act of 1934, as amended, that reflect InterMune’s judgment and involve risks and uncertainties as of the date of this release, including without limitation InterMune’s expectations regarding the availability of its Expanded Access Program for patients in the U.S. with IPF. All forward-looking statements and other information included in this press release are based on information available to InterMune as of the date hereof, and InterMune assumes no obligation to update any such forward-looking statements or information. InterMune’s actual results could differ materially from those described in InterMune’s forward-looking statements.

Other factors that could cause or contribute to such differences include, but are not limited to, those discussed in detail under the heading “Risk Factors” in InterMune’s most recent annual report on Form 10-K filed with the Securities and Exchange Commission (SEC) on February 24, 2014 (the “Form 10-K”) and other periodic reports filed with the SEC, including but not limited to the following: (i) the risks related to the uncertain, lengthy and expensive clinical development process for the company’s product candidates, including having no unexpected safety, toxicology, clinical or other issues and having no unexpected clinical trial results such as unexpected new clinical data and unexpected additional analysis of existing clinical data; (ii) risks related to the regulatory process for the company’s product candidates, including the possibility that the results of the new 52-week Phase 3 clinical trial (ASCEND) having an FVC endpoint may not be satisfactory to the FDA for InterMune to receive regulatory approval for pirfenidone in the United States; (iii) risks related to unexpected regulatory actions or delays or government regulation generally; and (iv) risks related to the company’s manufacturing strategy, which relies on third-party manufacturers and which exposes InterMune to additional risks where it may lose potential revenue. The risks and other factors discussed above should be considered only in connection with the fully discussed risks and other factors discussed in detail in the Form 10-K and InterMune’s other periodic reports filed with the SEC, all of which are available via InterMune’s web site at www.intermune.com.

SOURCE InterMune, Inc.

Investors: Jim Goff, InterMune, Inc., 415-466-2228, jgoff@intermune.com, Media: Geoff Curtis, Edelman, 312-550-8138, geoff.curtis@edelman.com



Lundbeck’s SABRIL® (vigabatrin) Now Approved by U.S. FDA as an Adjunctive Treatment Option for Children 10 and older with Refractory Complex Partial Seizures

Deerfield, Ill., October 28, 2013 – The U.S. Food and Drug Administration (FDA) approved SABRIL (vigabatrin) as add-on therapy for the treatment of refractory complex partial seizures (CPS) in children 10 years of age and older who have inadequately responded to several other treatments and if the possible benefit outweighs the risk of vision loss.1 This approval expands upon the age range of SABRIL’s previous indication as adjunctive therapy for adults with refractory CPS. SABRIL is not indicated as a first-line agent for refractory CPS.

Of the more than two million Americans affected by epilepsy,2 approximately 35 percent have CPS, which originates from a single region of the brain and can cause impaired consciousness.3 Approximately 30 to 36 percent of those with CPS continue to have seizures despite trying multiple therapies, and are considered to have refractory CPS.4,5,6

“It is crucially important for people with challenging seizures like refractory CPS to not give up and continue striving for improved seizure management, and this expanded Sabril indication provides another consideration for the treatment of those ten and older with refractory CPS,” said Philip Gattone, president and CEO of the Epilepsy Foundation. “We encourage people living with such challenging seizures and their loved ones to have ongoing conversations with their doctor about available options to help manage this intractable seizure disorder.”

When SABRIL was first approved in 2009, a patient registry was established to collect information on all patients who are prescribed SABRIL. To date, more than 5,600 patients have been treated with SABRIL, a substantial number of whom have been treated for refractory CPS.7 In evaluating whether to start SABRIL, doctors, patients and their caregivers work together to assess the risk of permanent vision loss versus the benefit of seizure reduction. There are other serious risks associated with SABRIL. Please see the important safety information below for more details.

“With so many children still having seizures due to refractory CPS, we are very pleased that the FDA has approved SABRIL for patients 10 and older who may benefit from a new add-on treatment option,” said Amy Magro, Director of Epilepsy Marketing at Lundbeck. “For those caring for a child as young as 10, we hope this new indication provides encouragement to speak with their child’s doctor about the risks and potential benefits of adding SABRIL for refractory CPS.”

In addition to its refractory CPS indication, SABRIL is approved for use in babies one month to two years of age with infantile spasms if the possible benefit outweighs the potential risk of vision loss.

For more information, please visit www.SABRIL.net.

About SABRIL® (vigabatrin) 1
SABRIL is a prescription oral antiepileptic drug developed in the United States by Lundbeck. SABRIL is available in 500-mg tablets or 500-mg packets of powder for oral suspension. Because of the risk of permanent vision loss, SABRIL is available only through a restricted program under a REMS called the SHARE Program. (1-888-45-SHARE).

SABRIL (vigabatrin) is a prescription medicine used with other treatments in adults and children 10 years of age and older with refractory complex partial seizures (CPS), who have not responded well enough to several other treatments, and if the possible benefits outweigh the risk of vision loss. SABRIL should not be the first medicine used to treat CPS.

SABRIL (vigabatrin) is a prescription medicine used in babies, 1 month to 2 years old, with infantile spasms (IS), if the possible benefits outweigh the possible risk of vision loss.

Important Safety Information


See Medication Guide and full Prescribing Information for complete information

In all people who take SABRIL:

• You are at risk for vision loss with any amount of SABRIL

• Your risk of vision loss may be higher the more SABRIL you take daily and the longer you take it

• It is not possible for your healthcare provider to know when vision loss will happen. It could happen soon after starting SABRIL or any time during treatment. It may even happen after treatment has stopped.

Please see SABRIL Medication Guide, full Prescribing Information including Boxed Warning, and Instructions for Use; go to www.sabril.net, or call toll-free 1-888-45-SHARE (1-888-457-4273).

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.

About Lundbeck in the U.S.
A wholly owned subsidiary of H. Lundbeck A/S of Denmark, Lundbeck in the United States is headquartered in Deerfield, Illinois, and is committed to providing innovative specialty therapies that fulfill unmet medical needs of people with central nervous system (CNS) disorders, including several therapies for people with challenging seizure disorders.

With a special commitment to the epilepsy community, Lundbeck actively supports and participates in hundreds of community-based initiatives. Learn more about our epilepsy community programs at http://www.lundbeck.com/us/our-commitment/community-involvement.

About H. Lundbeck A/S
Lundbeck is a global pharmaceutical company highly committed to improving the quality of life of people living with brain diseases. For this purpose, Lundbeck is engaged in the entire value chain throughout research, development, production, marketing and sales of pharmaceuticals across the world. The company’s products are targeted at disorders such as depression and anxiety, psychotic disorders, epilepsy, Huntington’s, Alzheimer’s and Parkinson’s diseases. Lundbeck’s pipeline consists of several mid- to late-stage development programs.

Lundbeck employs more than 5,800 people worldwide, 2,000 of whom are based in Denmark. We have employees in 57 countries and our products are registered in more than 100 countries. We have research centers in Denmark, China and the United States and production facilities in Italy, France, Mexico, China and Denmark. Lundbeck generated revenue of approximately DKK 15 billion in 2012. Lundbeck’s shares are listed on the stock exchange in Copenhagen under the symbol “LUN.” Lundbeck has a sponsored Level 1 ADR programme listed in the US (OTC) under the symbol “HLUYY.” For additional information, we encourage you to visit our corporate site www.lundbeck.com.


  1. SABRIL® (vigabatrin) full Prescribing Information, Deerfield, IL. Lundbeck. 2013.
  2. Epilepsy Foundation. About Epilepsy: Statistics. http://www.epilepsyfoundation.org/aboutepilepsy/index.cfm/statistics. Accessed 10/2/13.
  3. Carroll E. Medscape. Complex Partial Seizures. http://emedicine.medscape.com/article/1183962-overview. Accessed 10/2/13.
  4. Kwan P, Brodie MJ. Early identification of refractory epilepsy. N Engl J Med. 2000; 342:314-9.
  5. Devinsky O. Patients with refractory seizures. N Engl J Med. 1999;340:1565-70.
  6. Rielo DM. Medscape. Vagus Nerve Stimulation. 2011. http://emedicine.medscape.com/article/1186123-overview. Accessed 10/2/13.
  7. Data on File.


Matt Flesch

SABRIL is a registered trademark of Lundbeck.



Actelion receives U.S. FDA approval of Opsumit (macitentan) for the treatment of pulmonary arterial hypertension.

Allschwil, Switzerland, October 18, 2013 – Actelion Ltd (SIX: ATLN) announced today that the United States Food and Drug Administration (FDA) has approved the use of the orally available endothelin receptor antagonist Opsumit® (macitentan) 10 mg once daily for the treatment of pulmonary arterial hypertension (PAH) to delay disease progression.

Opsumit is indicated for the treatment of pulmonary arterial hypertension (PAH, WHO Group I) to delay disease progression. Disease progression included: death, initiation of intravenous (IV) or subcutaneous prostanoids, or clinical worsening of PAH (decreased 6-minute walk distance, worsened PAH symptoms and need for additional PAH treatment). Opsumit also reduced hospitalization for PAH.

Effectiveness was established in a long-term study in PAH patients with predominantly WHO Functional Class II-III symptoms treated for an average of 2 years. Patients were treated with Opsumit® monotherapy or in combination with phosphodiesterase-5 inhibitors or inhaled prostanoids. Patients had idiopathic and heritable PAH (57%), PAH caused by connective tissue disorders (31%), and PAH caused by congenital heart disease with repaired shunts (8%).

Dr. Vallerie McLaughlin, Director of the Pulmonary Hypertension Program in the Division of Cardiovascular Medicine at the University of Michigan, commented: “Over the past twenty years, great strides have been made in treating PAH patients. However, there has been a medical need for innovative treatments that improve long-term outcomes. Opsumit® is the first clinically proven and only oral treatment option indicated to delay disease progression and reduce the need for PAH hospitalization.”

Dr. McLaughlin concluded: “These effects were demonstrated in SERAPHIN, the first and largest PAH outcome study to date, where Opsumit® was given on average for 2 years, as a monotherapy or in combination with phosphodiesterase-5 inhibitors or inhaled prostanoids. I am very pleased that PAH patients will have this new treatment option.”

Jean-Paul Clozel, M.D. and Chief Executive Officer of Actelion commented: “Today’s approval of Opsumit® by the FDA is providing the PAH community with a unique treatment option, the only oral PAH medicine that has proven to delay disease progression. Over the last 14 years, Actelion has worked tirelessly to first discover and then develop Opsumit® in the largest, longest and first-ever outcome study in PAH. I would like to express my gratitude to all the members of the PAH community. Without their contribution, Opsumit® would not have become a reality. We will now leverage our existing PAH expertise and infrastructure to bring Opsumit® to patients within the coming weeks.”

The US label for Opsumit® carries a Boxed Warning alerting patients and health care professionals that the drug should not be used in pregnant women because it can harm the developing fetus. Female patients can receive the drug only through the Opsumit REMS Program. All female patients must be enrolled in the program, comply with pregnancy testing requirements and be counselled regarding the need for contraception.

The most common adverse reactions (more frequent than placebo by 3% or more) observed in patients treated with Opsumit® were anemia, nasopharyngitis/pharyngitis, bronchitis, headache, influenza, and urinary tract infection.

Physicians are advised to measure hemoglobin and liver enzymes prior to initiation of Opsumit® and repeat during treatment as clinically indicated.

In the United States, Actelion expects Opsumit® to become available to patients in November. Outside of the United States, Actelion continues to work with health authorities to obtain regulatory approval for Opsumit® .

The FDA approval was based in part on data from the landmark phase III SERAPHIN study. Published in the New England Journal of Medicine in August 2013, the SERAPHIN study showed the risk of the first occurrence of a morbidity or mortality event, the primary endpoint of the study, was reduced by 45% (p<0.0001) with macitentan 10 mg compared to placebo. This effect was observed irrespective of whether or not patients were already treated with other therapies for PAH. SERAPHIN also showed a risk reduction in PAH related hospitalization and death of 50% (p<0.0001) compared to placebo. [1].

Pulmonary arterial hypertension (PAH) is a chronic, life-threatening disorder characterized by abnormally high blood pressure in the arteries between the heart and lungs of an affected individual. The symptoms of PAH are non-specific and can range from mild breathlessness and fatigue during normal daily activity to symptoms of right heart failure and severe restrictions on exercise capacity and ultimately reduced life expectancy.


Opsumit® (macitentan) is a novel dual endothelin receptor antagonist (ERA) that resulted from a tailored drug discovery process with the target of developing an ERA to address efficacy and safety [3].

SERAPHIN (Study with an Endothelin Receptor Antagonist in Pulmonary arterial Hypertension to Improve cliNical outcome) was the largest and longest randomized, controlled study in PAH patients to include a clearly defined morbidity/mortality primary endpoint [2]. The pivotal Phase III study was designed to evaluate the efficacy and safety of Opsumit®(macitentan) – a novel dual endothelin receptor antagonist that resulted from a tailored drug discovery process – through the primary endpoint of time to first morbidity and all-cause mortality event in patients with symptomatic PAH.

Global enrollment was completed in December 2009 with a total of 742 patients. Patients were randomized 1:1:1 to receive two different doses of macitentan (3 mg and 10 mg once daily) or placebo. Patients were allowed to receive PAH background therapy throughout the study, either PDE-5 inhibitors or oral/inhaled prostanoids. This event-driven study was conducted in 151 centers from almost 40 countries in North America, Latin America, Europe, Asia-Pacific and Africa and was completed in the first half of 2012, with 287 patients having an adjudicated event.

Dr. McLaughlin is a consultant to Actelion and was an investigator in the SERAPHIN trial.

Patients were randomized to placebo (n=250), macitentan 3 mg (n=250), or macitentan 10 mg (n=242). The primary end point occurred in 46.4%, 38.0%, and 31.4% of the patients in these groups, respectively. The hazard ratio for macitentan 3 mg versus placebo was 0.70 (97.5% CI, 0.52 to 0.96; p=0.0108) and the hazard ratio for macitentan 10 mg versus placebo was 0.55 (97.5% CI, 0.39 to 0.76; p<0.0001). Worsening of pulmonary arterial hypertension was the most frequent primary end point event. The effect of macitentan on this end point was observed irrespective of background therapy for pulmonary arterial hypertension. [1]

Opsumit is contraindicated in pregnancy because it may harm the developing fetus. Females of reproductive potential should be counselled on the use of reliable contraception and have a negative pregnancy test prior to initiating therapy and monthly thereafter.

Other ERAs have been associated with elevations of aminotransferases, hepatotoxicity, and liver failure. Liver enzyme tests should be obtained prior to initiation of Opsumit® and repeated during treatment as clinically indicated. If clinically relevant aminotransferase elevations occur, or if elevations are accompanied by clinical symptoms of hepatoxicity, discontinue Opsumit®.

Decreases in hemoglobin concentration and hematocrit occurred following administration of other ERAs and were observed in clinical studies with OPSUMIT. The decreases occurred early and stabilized thereafter. Decreases in hemoglobin seldom require transfusion. Initiation of Opsumit® is not recommended in patients with severe anemia. Hemoglobin should be measured prior to initiation of treatment and repeat during treatment as clinically indicated.

Should signs of pulmonary edema occur, consider the possibility of associated PVOD. If confirmed, discontinue Opsumit®.

Other ERAs have been associated with adverse effects on spermatogenesis. Men should be counseled about potential effects on fertility.

The use of Opsumit® with strong CYP3A4 inducers or inhibitors should be avoided.

The most common adverse reactions (more frequent than placebo by 3% or more) observed in patients treated with Opsumit were anemia, nasopharyngitis/pharyngitis, bronchitis, headache, influenza, and urinary tract infection.


Approval of the new drug application for Opsumit® (macitentan) was issued by the US Food and Drug Administration (FDA) on 18October 2013 for the treatment of pulmonary arterial hypertension (PAH, WHO Group I) to delay disease progression. Disease progression included: death, initiation of intravenous (IV) or subcutaneous prostanoids, or clinical worsening of PAH (decreased 6-minute walk distance, worsened PAH symptoms and need for additional PAH treatment). The need for PAH hospitalization was also reduced.

Regulatory reviews are ongoing in Europe, Canada, Switzerland, Australia, Taiwan, Korea and Mexico.


Pulmonary arterial hypertension (PAH) is a chronic, life-threatening disorder characterized by abnormally high blood pressure in the arteries between the heart and lungs of an affected individual. The symptoms of PAH are non-specific and can range from mild breathlessness and fatigue during normal daily activity to symptoms of right heart failure and severe restrictions on exercise capacity and ultimately reduced life expectancy.

PAH is one group within the classification of pulmonary hypertension (PH). This group includes idiopathic PAH, heritable PAH and PAH caused by factors which include connective tissue disease, HIV infection and congenital heart disease.

The last decade has seen significant advances in the understanding of the pathophysiology of PAH, which has been paralleled with developments of treatment guidelines and new therapies. Drugs targeting the three pathways that have been established in the pathogenesis of PAH are endothelin receptor antagonists (ERAs), prostacyclins and phosphodiesterase-5 inhibitors. PAH treatments have transformed the prognosis for PAH patients from symptomatic improvements in exercise tolerance 10 years ago to delayed disease progression today. Improved disease awareness and evidence-based guidelines developed from randomized controlled clinical trial data have highlighted the need for early intervention, goal-oriented treatment and combination therapy.

In PAH, survival rates are unacceptably low and PAH remains incurable.


  1. Pulido T et al. Macitentan and Morbidity and Mortality in Pulmonary Arterial Hypertension. N Engl J Med 2013;369:809-18.
  2. Proceedings of the 4th world symposium on pulmonary hypertension. J Am CollCardiol 2009;54(1 Suppl).
  3. Bolli MH et al. The Discovery of N-[5-(4-Bromophenyl)-6-[2-[(5-bromo-2-pyrimidinyl)oxy]ethoxy]-4-pyrimidinyl]-N’-propylsulfamide (Macitentan), an Orally Active, Potent Dual Endothelin Receptor Antagonist. J Med Chem. 2012; 55:7849-61.
  4. Gatfield J, Mueller Grandjean C, Sasse T, Clozel M, Nayler O (2012). Slow Receptor Dissociation Kinetics Differentiate Macitentan from Other Endothelin Receptor Antagonists in Pulmonary Arterial Smooth Muscle Cells. PLOS ONE 7(10): e47662. doi:10.1371/journal.pone.0047662
  5. Iglarz M et al. Pharmacology of macitentan, an orally active tissue-targeting dual endothelin receptor antagonist. J PharmacolExpTher. 2008;327(3):736-45.
  6. Sidharta PN et al. Macitentan: Entry-into-humans study with a new endothelin receptor antagonist. Eur J ClinPharmacol. 2011;67(10):977-84
  7. Bruderer S et al. Absorption, distribution, metabolism, and excretion of macitentan, a dual endothelin receptor antagonist, in humans. Xenobiotica. 2012 Sep;42(9):901-10
  8. Bruderer S et al. Effect of cyclosporine A and rifampin on the pharmacokinetics of macitentan, a tissue-targeting dual endothelin receptor antagonist. AAPS J. 2012;14(1):68-78.
  9. Galiè N, Hoeper MM, Humbert M, et al; ESC Committee for Practice Guidelines (CPG). Guidelines for the diagnosis and treatment of pulmonary hypertension: the Task Force for the Diagnosis and Treatment of Pulmonary Hypertension of the European Society of Cardiology (ESC) and the European Respiratory Society (ERS), endorsed by the International Society of Heart and Lung Transplantation (ISHLT). Eur Heart J 2009;30:2493-537
  10. Benza RL, Miller DP, Barst RJ, Badesch DB, Frost AE, McGoon MD. An evaluation of long-term survival from time of diagnosis in pulmonary arterial hypertension from REVEAL. Chest 2012;142:448-56.

Actelion Ltd
Actelion Ltd is a biopharmaceutical company with its corporate headquarters in Allschwil/Basel, Switzerland. Actelion’s first drug Tracleer® (bosentan), an orally available dual endothelin receptor antagonist, has been approved as a therapy for pulmonary arterial hypertension. Actelion markets Tracleer through its own subsidiaries in key markets worldwide, including the United States (based in South San Francisco), the European Union, Japan, Canada, Australia and Switzerland. Actelion, founded in late 1997, is a leading player in innovative science related to the endothelium – the single layer of cells separating every blood vessel from the blood stream. Actelion’s over 2,300 employees focus on the discovery, development and marketing of innovative drugs for significant unmet medical needs. Actelion shares are traded on the SIX Swiss Exchange (ticker symbol: ATLN) as part of the Swiss blue-chip index SMI (Swiss Market Index SMI®).

For further information please contact:
Roland Haefeli
Senior Vice President, Head of Investor Relations & Public Affairs
Actelion Pharmaceuticals Ltd, Gewerbestrasse 16, CH-4123 Allschwil
+41 61 565 62 62
+1 650 624 69 36

The above information contains certain “forward-looking statements”, relating to the company’s business, which can be identified by the use of forward-looking terminology such as “estimates”, “believes”, “expects”, “may”, “are expected to”, “will”, “will continue”, “should”, “would be”, “seeks”, “pending” or “anticipates” or similar expressions, or by discussions of strategy, plans or intentions. Such statements include descriptions of the company’s investment and research and development programs and anticipated expenditures in connection therewith, descriptions of new products expected to be introduced by the company and anticipated customer demand for such products and products in the company’s existing portfolio. Such statements reflect the current views of the company with respect to future events and are subject to certain risks, uncertainties and assumptions. Many factors could cause the actual results, performance or achievements of the company to be materially different from any future results, performances or achievements that may be expressed or implied by such forward-looking statements. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those described herein as anticipated, believed, estimated or expected.


News Release Intended for U.S. Media Only

FDA Approves Bayer’s New Class of Drug Adempas® (riociguat) tablets to Treat Adults with PAH and Persistent, Recurrent or Inoperable CTEPH First and only drug approved in U.S. to Treat Two Forms of Pulmonary Hypertension (WHO Group 1 and 4)

Whippany, N.J., October 8, 2013 – Bayer HealthCare announced today that the United States Food and Drug Administration (FDA) has approved Adempas®  (riociguat) tablets for: (i) the treatment of adults with persistent/recurrent chronic thromboembolic pulmonary hypertension (CTEPH) (WHO*  Group 4) after surgical treatment or inoperable CTEPH to improve exercise capacity and WHO functional class; and (ii) the treatment of adults with pulmonary arterial hypertension (PAH) (WHO Group 1) to improve exercise capacity, improve WHO functional class and delay clinical worsening. In PAH, efficacy was shown in patients on Adempas monotherapy or in combination with endothelin receptor antagonists (ERAs) or prostanoids (inhaled, oral or subcutaneous). Studies establishing effectiveness included predominately patients with WHO functional class II-III and etiologies of idiopathic or heritable PAH (61%) or PAH associated with connective tissue diseases (25%). Adempas is the only treatment approved in the U.S. for use in two types of pulmonary hypertension (WHO Group 1 and 4). It is the first and only FDA-approved drug therapy for persistent/recurrent CTEPH after surgical treatment or inoperable CTEPH. It is also the only approved oral therapy in PAH with efficacy shown in monotherapy or in combination with ERAs or prostanoids. For all female patients, Adempas is available only through a restricted program called the Adempas Risk Evaluation and Mitigation Strategy (REMS) Program.

1 World Health Organization

“CTEPH and PAH are serious and life-threatening diseases,” said Nick H. Kim, Associate Clinical Professor of Medicine, Division of Pulmonary and Critical Care Medicine; Director, Pulmonary Vascular Medicine; Director, Fellowship Program; University of California San Diego. “The approval of Adempas equips physicians with a new approach to treating PAH patients, and it gives us the first approved drug treatment for patients with inoperable CTEPH or with persistent/recurrent CTEPH after surgery. While surgery should always be considered as the first treatment option for CTEPH, the fact remains that up to forty percent of CTEPH patients are not eligible for surgery, and ten to thirty-five percent of CTEPH patients have disease that persists after surgery.” PAH is a disease characterized by elevated pressure in the pulmonary arteries. CTEPH is a form of pulmonary hypertension in which blood clots and thromboembolic occlusion of the pulmonary vessels leads to increased pressure in the pulmonary arteries. The standard treatment for CTEPH is pulmonary endarterectomy, a potentially curative surgery that clears clots and scar material from the blood vessels of the lung. “Bayer is deeply committed to bringing new treatment options to patients with life-threatening diseases. Adempas is an excellent example of this commitment, because it is the result of years of dedicated research in our Bayer laboratories into a new way of treating two forms of pulmonary hypertension,” said Pamela A. Cyrus, MD, Vice President and Head, U.S. Medical, Bayer HealthCare Pharmaceuticals. “We are pleased to bring this new class of treatment to patients with PAH or with inoperable CTEPH or persistent/recurrent CTEPH after surgical treatment.” Rino Aldrighetti, President and CEO, Pulmonary Hypertension Association added, “From a patient’s perspective, living with pulmonary hypertension remains difficult. We know that not all treatments work for all people. We get excited when there is a new treatment option for PAH patients, and we are thrilled there is finally an approved drug treatment for people living with persistent/recurrent CTEPH after surgical treatment or inoperable CTEPH.” Adempas, a stimulator of soluable guanylate cyclase (sGC), represents a new class of drug now available in the U.S. Pulmonary hypertension is associated with endothelial dysfunction, impaired synthesis of nitric oxide (NO) and insufficient stimulation of the NO-sGC-cGMP pathway. Adempas sensitizes sGC to endogenous NO by stabilizing the NO-sGC binding. Adempas also directly stimulates sGC via a different binding site independently of NO. Adempas restores the NO-sGC-cGMP pathway and leads to increased generation of cGMP with subsequent vasodialation. The most common adverse reactions occurring more frequently (>3%) on Adempas than placebo were headache (27% vs 18%), dyspepsia/gastritis (21% vs. 8%), dizziness (20% vs 13%), nausea (14% vs 11%), diarrhea (12% vs 8%), hypotension (10% vs 4%), vomiting (10% vs 7%), anemia (7% vs 2%), gastroesophageal reflux disease (5% vs 2%), and constipation (5% vs 1%). Other events that were seen more frequently in Adempas compared to placebo and potentially related to treatment were: palpitations, nasal congestion, epistaxis, dysphagia, abdominal distension and peripheral edema. About Patient Assistance Program Bayer offers patient assistance through the Adempas Aim Support Center program, which will assist with obtaining coverage and patient support of Adempas. Patients and providers may contact the program at 1-855-4ADEMPAS for additional information. IMPORTANT SAFETY INFORMATION 

WARNING: EMBRYO-FETAL TOXICITY Do not administer Adempas (riociguat) tablets to a pregnant female because it may cause fetal harm.

Females of reproductive potential: Exclude pregnancy before the start of treatment, monthly during treatment, and 1 month after stopping treatment. Prevent pregnancy during treatment and for one month after stopping treatment by using acceptable methods of contraception.

For all female patients, Adempas is available only through a restricted program called the Adempas Risk Evaluation and Mitigation Strategy (REMS) Program.

Contraindications. Adempas is contraindicated in:

Warnings and Precautions Embryo-Fetal Toxicity. Adempas may cause fetal harm when administered during pregnancy and is contraindicated for use in women who are pregnant. In females of reproductive potential, exclude pregnancy prior to initiation of therapy, advise use of acceptable contraception and obtain monthly pregnancy tests. For females, Adempas is only available through a restricted program under the Adempas REMS Program. Adempas REMS Program. Females can only receive Adempas through the Adempas REMS Program, a restricted distribution program. Important requirements of the Adempas REMS program include the following:

Further information, including a list of certified pharmacies, is available at www.AdempasREMS.com or 1-855-4ADEMPAS. Hypotension. Adempas reduces blood pressure. Consider the potential for symptomatic hypotension or ischemia in patients with hypovolemia, severe left ventricular outflow obstruction, resting hypotension, autonomic dysfunction, or concomitant treatment with antihypertensives or strong CYP and P-gp/BCRP inhibitors. Consider a dose reduction if patient develops signs or symptoms of hypotension. Bleeding. In the placebo-controlled clinical trials program, serious bleeding occurred in 2.4% of patients taking Adempas compared to 0% of placebo patients. Serious hemoptysis occurred in 5 (1%) patients taking Adempas compared to 0 placebo patients, including one event with fatal outcome. Serious hemorrhagic events also included 2 patients with vaginal hemorrhage, 2 with catheter site hemorrhage, and 1 each with subdural hematoma, hematemesis, and intra-abdominal hemorrhage. Pulmonary Veno-Occlusive Disease. Pulmonary vasodilators may significantly worsen the cardiovascular status of patients with pulmonary veno-occlusive disease (PVOD). Therefore, administration of Adempas to such patients is not recommended. Should signs of pulmonary edema occur, the possibility of associated PVOD should be considered and if confirmed, discontinue treatment with Adempas.  Most Common Adverse Reactions  The most common adverse reactions occurring more frequently (>3%) on Adempas than placebo were headache (27% vs 18%), dyspepsia/gastritis (21% vs. 8%), dizziness (20% vs 13%), nausea (14% vs 11%), diarrhea (12% vs 8%), hypotension (10% vs 4%), vomiting (10% vs 7%), anemia (7% vs 2%), gastroesophageal reflux disease (5% vs 2%), and constipation (5% vs 1%). Other events that were seen more frequently in Adempas compared to placebo and potentially related to treatment were: palpitations, nasal congestion, epistaxis, dysphagia, abdominal distension and peripheral edema. For important risk and use information, please see the full Prescribing Information, including Boxed Warning, at www.adempas-us.com. About Bayer HealthCare Pharmaceuticals Inc. Bayer HealthCare Pharmaceuticals Inc. is the U.S.-based pharmaceuticals business of Bayer HealthCare LLC, a subsidiary of Bayer AG. Bayer HealthCare is one of the world’s leading, innovative companies in the healthcare and medical products industry, and combines the activities of the Animal Health, Consumer Care, Medical Care, and Pharmaceuticals divisions. As a specialty pharmaceutical company, Bayer HealthCare provides products for General Medicine, Hematology, Neurology, Oncology and Women’s Healthcare. The company’s aim is to discover and manufacture products that will improve human health worldwide by diagnosing, preventing and treating diseases. Bayer® and the Bayer Cross® are registered trademarks of Bayer. Intended for U.S. media only U.S. Media Contact: Marcy Funk, Communications, Bayer HealthCare Telephone: (862) 404-5385 E-Mail: marcy.funk@bayer.com Forward-Looking Statements This release may contain forward-looking statements based on current assumptions and forecasts made by Bayer Group or subgroup management. Various known and unknown risks, uncertainties and other factors could lead to material differences between the actual future results, financial situation, development or performance of the company and the estimates given here. These factors include those discussed in Bayer’s public reports which are available on the Bayer website at www.bayer.com. The company assumes no liability whatsoever to update these forward-looking statements or to conform them to future events or developments.


News Release Intended for U.S. Media Only


If approved by the FDA, riociguat will be the first treatment for inoperable CTEPH or persistent/recurrent CTEPH after surgery and a new treatment for PAH

Whippany, N.J., August 6, 2013– Bayer HealthCare today announced that the U.S. Food and Drug Administration’s (FDA) Cardiovascular and Renal Drugs Advisory Committee recommended approval for investigational riociguat, proposed trade name Adempas™, in two forms of pulmonary hypertension. The Committee voted 11 to 0 that riociguat should be approved for the treatment of pulmonary arterial hypertension [PAH] of WHO1 Group 1. The Committee also voted 11 to 0 that riociguat should be approved for the treatment of chronic thromboembolic pulmonary hypertension (CTEPH) of WHO Group 4. In February 2013, Bayer submitted a new drug application for riociguat in two indications: (i) the treatment of PAH (WHO Group 1) to improve exercise capacity, improve WHO functional class and delay clinical worsening; and (ii) the treatment of persistent/recurrent CTEPH (WHO Group 4) after surgical treatment or inoperable CTEPH to improve exercise capacity and WHO functional class. “We appreciate the Committtee’s discussion today around the safe and appropriate use of riociguat and are pleased with the outcome of the votes,” said Pamela A. Cyrus, MD, Vice President and Head of U.S. Medical Affairs, Bayer HealthCare Pharmaceuticals. “If approved, riociguat will offer a new treatment option for patients with PAH and will also provide the first approved non-surgical treatment option for CTEPH patients who are inoperable or who have recurrent or persistent disease. We look forward to continued dialogue with the FDA in order to make riociguat available to patients.”

1 World Health Organization

PAH and CTEPH are both life-threatening forms of pulmonary hypertension that cause significantly increased pressure in the pulmonary arteries. Riociguat is an investigational, oral medication for the treatment of adult patients with PAH or inoperable or persistent/recurrent CTEPH. If approved by the FDA later this year, it would create a new class of therapy available in the U.S. PH is associated with endothelial dysfunction, impaired synthesis of nitric oxide (NO) and insufficient stimulation of soluble guanylate cyclase (sGC). Riociguat stimulates sGC independent of NO and increases the sensitivity of sGC to NO. Data presented at today’s advisory committee meeting included results from the global Phase 3 clinical program, which enrolled 704 patients across two Phase 3 studies. Both studies met their primary endpoint by demonstrating a statistically significant improvement in the six-minute walk test (6MWT), after 16 and 12 weeks respectively. Riociguat was also associated with improvements across multiple, relevant, secondary endpoints in the studies. The most common treatment-emergent adverse events with riociguat were headache, dizziness, dsypesia, peripheral edema, nausea, diarrhea and vomiting. The advisory committee’s vote will be taken into consideration by the FDA when making its decision on the approvability of Bayer’s NDA for riociguat, which was submitted in February 2013. After acceptance of the NDA, the FDA granted riociguat priority review designation, which is given to drugs that have the potential to offer significant improvement in treatment or provide a treatment option where no adequate therapy exists. About Pulmonary Arterial Hypertension (PAH) In PAH, a rare and life-threatening disease, the blood pressure in the pulmonary arteries (the arteries that take de-oxygenated blood to the lungs from the heart) is significantly increased. PAH is characterized by morphological changes to the endothelium of the arteries of the lungs causing remodeling of the tissue, vasoconstriction and thrombosis-in-situ. As a result of these changes, the blood vessels in the lungs are narrowed, making it difficult for the heart to pump blood through to the lungs. In most cases, PAH has no known cause and, in some cases, it can be inherited. About Chronic Thromboembolic Pulmonary Hypertension (CTEPH) CTEPH is also a rare and life-threatening disease in which it is believed that thromboembolic occlusion (organized blood clots) of pulmonary vessels gradually lead to an increased pressure in the pulmonary arteries, resulting in an overload of the right heart. CTEPH may evolve after prior episodes of acute pulmonary embolism, but the pathogenesis is not yet completely understood. The standard treatment for CTEPH is pulmonary endarterectomy (PEA), a surgical procedure in which the blood vessels of the lungs are cleared of clot and scar material. However, CTEPH is inoperable in an estimated 20 to 40 percent of patients, and, in some cases, the disease persists or reoccurs after surgery. About Bayer HealthCare Pharmaceuticals Inc.Bayer HealthCare Pharmaceuticals Inc. is the U.S.-based pharmaceuticals business of Bayer HealthCare LLC, a subsidiary of Bayer AG. Bayer HealthCare is one of the world’s leading, innovative companies in the healthcare and medical products industry, and combines the activities of the Animal Health, Consumer Care, Medical Care, and Pharmaceuticals divisions. As a specialty pharmaceutical company, Bayer HealthCare provides products for General Medicine, Hematology, Neurology, Oncology and Women’s Healthcare. The company’s aim is to discover and manufacture products that will improve human health worldwide by diagnosing, preventing and treating diseases. Bayer® and the Bayer Cross® are registered trademarks of Bayer.

Intended for U.S. media only Media Contact: Marcy Funk, Communications, Bayer HealthCare Telephone: (862) 404-5385 E-Mail: marcy.funk@bayer.com

Forward-Looking Statements This release may contain forward-looking statements based on current assumptions and forecasts made by Bayer Group or subgroup management. Various known and unknown risks, uncertainties and other factors could lead to material differences between the actual future results, financial situation, development or performance of the company and the estimates given here. These factors include those discussed in Bayer’s public reports which are available on the Bayer website at www.bayer.com. The company assumes no liability whatsoever to update these forward-looking statements or to conform them to future events or developments.

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Learning to Live with Pulmonary Hypertension

Posted on by Charlie

48-year-old Mari Jackson had worked since the age of 12. But last year, a diagnosis of pulmonary arterial hypertension forced her to slow down and approach life differently.

Mari Jackson and her husband, William.

Mari Jackson and her husband, William. Photo by Anthony Nesossis

I got diagnosed with pulmonary hypertension last March. The symptoms started in February of last year, right around my husband’s birthday. It was like something out of the blue.

We had gone to dinner to celebrate his birthday and came home, and I said, “I’m going to get ready for work.”

I went upstairs to take a shower, and when I got off the bed to walk towards the bathroom, I just blacked out. When I woke up, I was on the floor, and I was face down by the sink.

I called my husband, and he and my son came running up the stairs, and my husband said, “What are you doing on the floor?”

I said, “I have no idea. I just blacked out.” My body had just shut down. I was so embarrassed.

But I didn’t pay it any attention. I worked for a mortgage company. Believe it or not, Monday through Friday, I drove every day to work—44 miles round trip. I worked anywhere from 10 to 12 hours a day, and I worked weekends and overtime.

I had an episode every weekend. I would pass out. I actually had a seizure one night. My husband noticed that every time I would go upstairs, I would get really, really light-headed and either fall backwards or lose my balance.

So, one night he came upstairs with me and said, “Let me just help you into the room.” I was using the bathroom, and I was sliding off the seat. I had a seizure, and he was right there to catch me, and I bit my tongue, and it was so weird because I had never had that happen.

He took me to the ER, and they ran a lot of tests. They said, “We don’t see anything, but we do see that you have pneumonia.” So, they gave me antibiotics for that, and I went home.

The first Saturday in March, I went to work. I got upset about something, and the whole left side of my arm turned ice cold. It was numb, and I kept shaking it. Then, I started sweating.

I told my manager, “I’m going home. I’m not going to be able to stay.” My manager said, “You don’t look well,” and he called my husband.

My husband came and got me and took me to the emergency room. My blood pressure was 280 over 190. They said, “You’re not going anywhere, so get comfortable.” I ended up having to be admitted. They ran some more tests, and I stayed there for a week.

They did a lung biopsy, and then a right heart catheter. They did a crash blood pressure drop on me, and they would give me blood pressure medication three times a day. I felt like somebody was coming in and beating me.

I said, “Why do I feel this way?” They said, “We have to get your blood pressure down.” I had no idea, because my pressure was always high.

Once I got all the test results back, I met with my pulmonologist. He told me I had pulmonary arterial hypertension, that basically, my heart and lungs are not friends right now. I had no idea that my life was going to change.

I was devastated. I wanted to go back to work. And I couldn’t. I couldn’t even go up the stairs in my house. I either stayed upstairs or downstairs. To walk from my kitchen to my bathroom, I was gassed out. If I got up too fast, I fell out. If I dropped my head too long to tie my shoe, I passed out.

I couldn’t understand it. I got mad. I questioned myself. I questioned God, and I know that wasn’t the right thing to do, but I didn’t know what else to do. I wanted answers. I prayed for understanding.

Then I just started learning. I got on the websites and started learning about PH. There were things I had to do differently. I was confined to my room for six weeks because I couldn’t go up and down the stairs. I had to sit on the stairs and go up backwards. I was a very independent person. Then to have to rely on someone to cook me breakfast, to get me water, I thought, ‘This is unreal.’

I had to reform my thinking. My doctor told me I had to lose weight. And I thought, ‘Well, how is that going to happen if I can’t exercise?’

So I had to learn how to cut back on carbs, sodium, and to fall in love with water. I was 325 pounds, and now I’m 260, and I feel much better than I did a year ago. My doctor actually told me last week, “Compared to how you were last year, you would have never known.”

It just happened. I have had asthma since I was a little girl, but I only had one flare up as an adult. PAH hides behind asthma, and then it explodes all of the sudden. People with asthma have to be very careful and have things checked out extensively. There were no gradual signs.

Every time I would go to the doctor, my blood pressure would be high, and they would ask, “Do you have high blood pressure in your family?” And I would say, “Yes.” And they would ask, “Well, how do you feel?” And I would say, “I feel fine.”

But passing out and having seizures, never had I experienced that before. I was going through my own thing.

I didn’t really want to talk to people, because I couldn’t talk. I was gassing out. The longer I talked, the worse it got. You could hear I was struggling to talk. Last year, I was in a wheelchair.

I was scared, because you read things on the internet about your life expectancy. Then you read about all the new medications that have been FDA-approved. You’re hoping and praying that one of those will work for you.

This year is like a complete turnaround. I have a lot to be thankful for. I’m able to walk distances at my own pace. I still use a wheelchair in big places. I don’t do the mall. I get anxiety around a lot of people, because I feel like, if something happens, I’m not going to be able to get out. I try not to go to a lot of places by myself, and if I go somewhere by myself, I let someone know where I am. I have a Life Alert.

When I walk, I do so at my own pace. If I get tired, I stop. I do all right. [Recently] I went to the doctor by myself. I walked in on my own. But I took my time.

I can’t work anymore—that was the biggest thing. My husband’s work is seasonal. Financially it’s very hard. So, I communicate a lot with other PAH patients. I’m on the website with them. I try to get other ideas from people about what they’re doing.

If I could do volunteer work, that would be good. It’s difficult, because I like working with people with disabilities. But I have to be real cautious about my surroundings. If I catch something, I can’t take anything over the counter.

I have two grown children; they’re 20 and 27. My husband has five. I have four grandchildren who live in Washington, D.C. Last year I couldn’t travel because I was sick. Maybe this year, if the weather and my health permit, we’ll take a little road trip to see them. My parents live in Seattle, but I can’t really visit them because of the altitude of a flight.

My youngest son was here for a year, and he was very helpful, but at the same time, he was a teenager, and I didn’t want him to be stuck in the house with me all summer. He ended up going back to California.

I’ve been working ever since I was 12. I don’t know what a hobby is. After I had kids, I went right back into the workplace.

I have a friend, and we’ll get together and have lunch once a month, and it’s nice. I do like to read. I like romance novels. I really got into talking with other people with PAH in my area. I want to look into a chapter here in Charlotte. Maybe I could do something with them.

My husband was and still is a very big part of my healing process. He always makes sure that my needs are taken care of, no matter what, even cooking dinner after working an eight-hour day. I really do appreciate my husband, and I thank God for him.

To the newly diagnosed, I would say, “It’s going to work out. Don’t get upset, because being upset starts a lot of things, like stress.” I still do that, but I really try not to.

Sometimes I think, ‘What if this gets turned off? What if I lose my house?’ I realized when I was doing that, it was making me worse.

I went through a point where I didn’t want to be bothered with anybody. I was mad. I thought, ‘Why can’t I work?’ I thought my life was done.

Now, I just take everything one day at a time. I don’t let anything bother me. I keep it moving. I want to be able to grab, absorb and use everything you give me to the best of my abilities.

I still call myself a newbie. To the newly diagnosed I would say, “Life still goes on. It’s just that you have to take it slow. It’s about you. Nobody else. That’s how it is.”

Posted in Diseases, Featured, Uncategorized | Tagged , , , , , , , , , , , , , , , | 8 Comments

Helpful Resources

Posted on by CVCinfo


Caring Voice Coalition has partnered with LegalShield to offer CVC patients discounted legal plan membership. For a small, flat monthly fee, CVC patients can become LegalShield members, with access to services including unlimited legal advice on any topic, living wills, estate planning, health care proxies, insurance coverage, and identity theft protection. With LegalShield, CVC patients have access to caring, top-quality attorneys from highly-rated law firms across the country at very low cost.

NeedyMeds, founded in 1997, is a non-profit information resource devoted to helping people in need find assistance programs to help them afford their medications and costs related to health care.

Patient Associations

Copayment Assistance Organizations

Government Agencies and Programs

Transplant Information

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Accessible Travel: Travel Therapy

Posted on by Charlie

Those with chronic conditions and limited mobility have more options than ever before for accessible travel. Eva Leonard talks to travelers with special needs and accessible travel experts about important considerations when planning a trip.

Canoe Coming into shore, Waikiki, Oahu, Hawaii.
Canoe coming into shore, Waikiki, Oahu, Hawaii. Photo by Hawaii Tourism Authority (HTA)/Tor Johnson

“Part of my job is to make vacation travel possible,” says Royal Caribbean International Manager, Access Compliance, Ron Pettit. “Many people with disabilities don’t think about traveling or taking a vacation as an option.

“They have challenges in everyday life; getting out of bed, getting out of the house, going to school, to work, going shopping, and going to the doctor. So sometimes, when they think of all their daily challenges, they think, ‘Oh my goodness—I don’t think I could go on a cruise.’

“Some folks are born with a disability, so they’ve learned how to adapt all along. But a lot of people have acquired a disability with age, or a medical issue, so they have to rethink and learn things all over again.”

Fear of the unknown can be a factor in deterring those with chronic illness from traveling, says Pettit, who has worked for the past 25 years to improve travel for the disabled, first for 17 years at Northwest Airlines, where he served as program manager for customers with disabilities, then joining Royal Caribbean in 2006.

“It’s those personal issues. ‘How do I go to the bathroom? How do I know about oxygen? How do I do these things?’ Because, while they have learned to adapt at home, or maybe when going out a little bit, the thought of going onto an airplane, or going onto a ship seems a little daunting. They ask, ‘How would I ever do this with my new limitations?’”

But many with disabilities are traveling. The U.S. Census reports that more than 38 million Americans live with disabilities, and, according to a 2005 study by the Open Doors Organization and the Travel Industry Association of America, U.S. adults with disabilities or reduced mobility spend around $13.6 billion on travel every year.

With the world’s population now at 7 billion, about ten percent require barrier-free and easily accessible facilities. “Global estimates [of people with disabilities] range from 600 million to 900 million,” says Lilian Muller, President of the European Network for Accessible Tourism.

To meet rising demand, accessible and barrier-free travel options have grown dramatically over the last two decades. Whether you opt for a leisurely Caribbean cruise, a scenic train ride through the Canadian Rockies, or something more distant and action-packed, careful research, planning and preparation can help you decide which getaway is best for you and allow you to fully enjoy the mood-boosting, stress-reducing rewards of travel.

Before you start planning your trip, check with your doctor to assess what you can do, and, depending on the type of trip you plan to take, consider working with a travel agent who understands your needs. Says Pettit, “The more information you can share about your ability and needs, the better.”

Make arrangements well in advance for wheelchair, scooter, and medical device and supply accessibility and rental. Says traveler Tracy Schutt, “Since being on oxygen 24/7, traveling has become challenging, but not impossible. Making trips to Jacksonville, Florida, to visit family requires preplanning with my home care company to have oxygen supplies waiting when I get there.”

Another traveler with pulmonary hypertension suggests sending IV medication and supplies ahead by overnight service and arranging for an oxygen concentrator, so that all are in place on arrival.

Traveler Milli Washock advises, “Have a sheet handy with all pertinent medical information, medication, supplies and emergency contacts. Being on [intravenous infusion] therapy and oxygen, I have written on top [in big, bold letters], ‘Do Not Stop Pump.’ And, she adds, for devices that must be charged or plugged in, “When traveling to a foreign country, be sure to have the proper electrical adapters.”

It’s also important to get as much information as possible in advance about the availability of services you might need en route and at your destination. For example, find out what airline, hotel, rail, or cruise staff can assist you with when traveling, and if there is a medical facility specializing in your condition at your destination. If you’re planning a cruise, ask what types of onboard medical services are available, and if there is a fee.

Scandic Hotels height-adjustable bed.
Scandic Hotels height-adjustable bed.

Travel writer Candy Harrington has been covering accessible travel for the past 16 years and has authored books on the topic, including, Barrier-Free Travel; A Nuts and Bolts Guide for Wheelers and Slow Walkers. She also writes the barrier-free travel blog www.BarrierFreeTravels.com.

Harrington advises travelers with special needs to be very specific about their needs and ask detailed questions when booking a hotel room.

“First and foremost,” she emphasizes, “you have to understand that there are many types of accessible rooms, so you have to ask for an accessible room with the features you need. Don’t just ask for an accessible or an ‘ADA-compliant’ room.

“If you need a roll-in shower, specify that, because all accessible rooms do not have roll-in showers—some have tub/shower combinations. If you need the toilet grab bars on a specific side, you need to specify that too.

“And if bed height is an issue for you, inquire about that also. Bed height is not covered under the Americans with Disabilities Act (ADA), and you could very well end up with a 32-inch-high bed, which would make transfers very challenging.

“Don’t assume that the accessible hotel room is going to have the exact same features that your own home has, because in most cases, it won’t. Ask a lot of questions to make sure you get the access features you need.”

Some hotels are more accessible than others, and good indicators of their commitment can often be found online. Benchmark Resorts & Hotels comprises 13 properties across the United States, and the company’s website promotes Benchmark’s commitment to ADA compliance, with detailed accessibility information for each property.

Benchmark’s Turtle Bay resort on Oahu’s North Shore features 15 ADA-compliant guest rooms and one ADA-compliant cottage. Other accessible features include the resort’s swimming pools, hot tubs, fitness center, spa, restaurants, lounges, and a wide-open door-less gateway entrance.

Magnus Berglund: Accessibility Director, Scandic Hotels
Magnus Berglund: Accessibility Director, Scandic Hotels. Photo by Karl Gabor for Scandic Hotels.

Stockholm-based Scandic Hotels, with nearly 230 properties throughout Northern Europe, has won awards for its hotels’ accessible features, such as a minimum of two cane holders attached to the front desk, carpet-free meeting rooms and height-adjustable beds. In consultation with disability organizations, hotel guests, and team members, Scandic Hotels drew up an accessibility standard in 2003 that works as a checklist and template for the hotels. The standard has grown over the years, and today it contains 110 checkpoints to follow. Eighty-one of these points are mandatory for all hotels, and for new hotels, all points must be considered.

Community spoke with Magnus Berglund, Scandic’s accessibility director, about his work with Scandic and how his service dog, Dixi, helps him throughout the day.

How did you first become involved with Scandic Hotels?
It started about ten years ago, when I was a cook at Scandic Hotels. Due to a muscle disease, I was on sick leave for five years. When I was able to start work again, I contacted my former employer, with my ideas on how the hotel chain could increase accessibility and use accessibility to gain competitive advantage. In 2003, I was appointed disability ambassador for Scandic Hotels.

What is a typical day like for you as Scandic’s director of accessibility?
I work with all departments at head office, on everything from new hotels to renovations. I consult with Scandic’s hotel designers on accessibility, and I’m also involved with employee education within Scandic. I travel a lot, often several times a week, when I visit any of our hotels or when I’m invited as a speaker around the world.

Dixi is a service dog that helps me with everything from getting my clothes to the bed in the morning to picking up things that I drop. She also carries my computer bag when I’m flying, and she follows me on all my trips. Basically, she follows me everywhere I go in my daily work, at the office, at conferences, when I’m invited as a speaker, and so on.

What are some of Scandic Hotels’ smart design features?
A really smart design feature is our cane holder at the reception desk. We also have our vibrating alarm clock that the guest puts under the pillow, so if you’re hearing-impaired, the clock will wake you up so you “hear” the fire alarm. We also have special bread for breakfast for guests who have gluten or lactose intolerance.

What are some of the barriers that travelers with disabilities encounter most often when they travel?
I think it’s extremely different [depending on] what kinds of special needs people have. The big challenge is to get the right information.

For more information on Scandic Hotels, go to www.scandichotels.com/Always-at-Scandic/Special-needs


A video on Turtle Bay’s website features paraplegic surfer Jess Billauer, founder of the Life Rolls on Foundation, dedicated to improving the quality of life for young people affected by spinal cord injury, as he easily wheels through the resort, surfs with an adaptive electric surfboard and describes the independence that accessibility brings.

ADA-compliant pool, Benchmark's Turtle Bay Resort, Oahu, Hawaii.
ADA-compliant pool, Benchmark’s Turtle Bay Resort, Oahu, Hawaii. Photo courtesy of Benchmark Hotels & Resorts.

“We do everything possible to make sure everything is accessible,” says Gary Harnist, vice president of construction and design for Benchmark. “ADA guest rooms at Turtle Bay have automatic doors. The peepholes are lower. Bathrooms have roll-in showers, and shower, temperature and lighting controls are at a reachable height. We want to make sure the balconies are accessible, so we have sliding glass doors and ramps.”

Harnist advises, “Ask questions before you arrive. Let us know what your needs are. Many times we’ve sent staff members to the store to buy a lower shower seat, or called a rental company to get the kind of wheelchair a guest needs to insure that their stay is perfect.” For more information about Benchmark Resorts & Hotels, go to www.benchmarkresortsandhotels.com/about/social_responsibility/ada_accessibility_compliance

Roll-in shower, Benchmark's Costa d'Este Beach Resort, Vero Beach, Florida.
Roll-in shower, Benchmark’s Costa d’Este Beach Resort, Vero Beach, Florida. Photo courtesy of Benchmark Hotels & Resorts.

As with hotels and resorts, when planning a trip to a theme park, checking in advance for clarity on accessibility and disability policies can be a good idea. Walt Disney World and Disneyland Resorts made headlines in October when it replaced its Guest Assistance Card (GAS) program with a Disability Access Service Card (DAS).

A Disney Parks blog post by Thomas Smith, social media director, Disney Parks, explains that guests with disabilities can now “request a DAS at Guest Relations and receive a return time for attractions based on the current wait time.” Prior to the change, which Smith said was prompted by abuse of the program, guests with disabilities had been able to go directly to the front of the lines for Disney attractions.

For more information on Walt Disney World and Disneyland Resorts, go to http://disneyparks.disney.go.com

A recent study by the Open Doors Organization and the U.S. Travel Association found that 12 percent of Americans with disabilities have taken a cruise in the last five years.

“Cruises are great for people who need to move slowly and take their time. You can be as active as you want and do everything, or you can do as little as you want, have a nice spot in the lounge and watch the sea,” says Royal Caribbean’s Pettit. “We’ve designed our cruise ships to be very accessible, so there are elevators, ramps, and platform lifts. We have options all over the ship for guests with limited mobility.”

Travel agents can be helpful in sorting out accessible cruise options. Says Pettit, “If you’ve never cruised before, you don’t know the questions to ask, and that’s why we recommend using a travel agent.

“There are travel agents who specialize in accessible cruises and in specific conditions. Some specialize in dialysis cruises, autism cruises, deaf cruises, blind cruises, and disability in general, and not just group cruises. They deal with individuals or families. They specialize, may have the disability themselves, and know the questions to ask. They can help walk you through making an informed decision about the right cruise line and cruise for you.”

Says traveler Milli Washock, “A few years ago, I went on a weekend cruise, with a wheelchair, room air concentrator, oxygen tanks, a portable concentrator and a BIPAP, and did quite well. The cruise line, Carnival, went out of their way to bring me [my oxygen] tanks wherever I was and made sure everything was taken care of in the cabin and with shows and dining. They even had distilled bottled water. We had a great time.”

Oasis of the Seas
The Oasis of the Seas, Labadee, Haiti. Photo courtesy of Royal Caribbean.

For those cruising for the first time, Washock suggests, “Book a room with open air, a window and/or a balcony, and liberally use hand sanitizer everywhere.”

Says Pettit, “Oxygen has changed over the years. What works well for a lot of people now is the new portable oxygen concentrator, what I call’ the magic box.’ It takes ambient air and turns it into breathable oxygen on demand.

“This works well for many people who require oxygen therapy. It may not work for everyone. Some people need continuous air flow. They may need a flow rate that’s higher than what the portable concentrators can give. But for many, many people, portable oxygen concentrators have been revolutionary.

“Portable oxygen concentrators allow passengers to use the same equipment in the plane, on the ground, and on the cruise ships. We have power outlets in our staterooms, so passengers can charge overnight, and they can bring extra batteries as well.

“Technology and the ability of our staff to assist our guests go a long way to help [those with special needs] think about cruising as a possibility. We provide our staff with sensitivity training and technical training, primarily for wheelchair assistance and assisting guests on and off the ship. That’s the number one request that we get.

“We provide training about different types of disabilities and how to communicate with different types of guests. We focus primarily on people with mobility, hearing, and visual disabilities, but we do talk about guests with cognitive or developmental disabilities.

“From an identification perspective, there’s always the question of dignity in providing too much information. The more information we have, the more we can assist you. If we don’t understand your condition, we may provide inappropriate support. If we know ahead of time, it makes things easier.”

Royal Caribbean's Quantum of the Seas accessible balcony stateroom.
Royal Caribbean’s Quantum of the Seas accessible balcony stateroom. Photo courtesy of Royal Carribean.

In February, Royal Caribbean was the first cruise line to be named as autism-friendly by Autism on the Seas, a agency that develops cruise vacation services for those with children with special needs.

Says Pettit, “Our products and services are accessible for guests with autism and other developmental disabilities. We have priority check in, boarding, departure, and special dietary offerings like gluten-free items. We offer modifications to our youth program onboard, like dropping down an age group if the child would be more comfortable, based on ability.

“We offer autism-friendly movies: The sound is not so loud, the lighting is a little bit lighter, and the kids are encouraged to get up and walk around during the movies. We added a social story to help families dealing with autism prepare for their cruise. Often children with autism need a little structure and preparation, [so we let them know that] when you get to the ship, this is what you’re going to see and this is what you’re going to do, so they can prepare. And really, it’s about the entire family and not just children. Many of our features are used by teens and adults with autism.

“Everybody is different. Their needs are going to be different. We can work with our guests and our travel agents to see if we can help accommodate that need. A lot of our business continues to be booked through travel agents. For many people, it’s their first time, or they may have special needs. You may need a little additional help from an expert. It makes for a much better cruise experience.”

Royal Caribbean’s Access Department is a resource center for guests and travel agents. Staff can answer questions about accommodations for guests with disabilities and can be reached at 866-592-7225 (phone); 954-628-9708 (local); 954-628-9622 (fax) or email special_needs@rccl.com. Or for more information, go to www.RoyalCaribbean.com/AccessibleSeas

Pettit says that cruises that involve the United States are often preferable for those in wheelchairs or those who need to move slowly. “Alaska, Hawaii, New England, and Western Pacific coastal cruises are all great options, simply because they involve U.S. ports of call, and, generally, when you’re within the U.S., you have a much better sense of accessibility.

“There are curb cuts. There are accessible restrooms and facilities. Whenever you get outside of the U.S., while there are accessibility regulations in place, they may not always meet the same level as in the U.S.”

“The Caribbean has mixed levels of accessibility. We’ve seen progress over the years in many of our ports of call. People tend to gravitate to the Eastern Caribbean itineraries more because more of the ports of call are docked—that means you can roll on and roll off the ship with ease. When you get to the Western Caribbean, you have more ports that are tender,” says Pettit.

“We sail to more than 300 ports of call around the world, and about one third of those ports are tender. What that means is that our ships cannot dock at a pier. They have to anchor in the harbor, and so we transfer guests onto a smaller vessel, usually called a tender. They take that tender to the port and get off there. That process may pose some challenges for guests in wheelchairs and those who have difficulty walking.

“Our larger ships, like Oasis of the Seas and Allure of the Seas, are great ships for guests in wheelchairs and those with difficulty walking, because they never have to tender. They always dock.”

Royal Caribbean Allure at Port Everglades.
Royal Caribbean Allure at Port Everglades. Photo courtesy of Royal Caribbean.

Bob Curley, Caribbean travel expert for About.com, notes that the U.S. Virgin Islands must comply with the ADA and that “Barbados has also made a pretty concerted effort to be compliant, as have Aruba and St. Maarten. Every cruise port in Jamaica is a non-tender port, and St. Thomas also has a dock.”

Says Pettit, “Europe is becoming increasingly accessible, although it’s mixed. A lot of our ships sail there in the summer. There are cobblestone streets, and there may not always be curb cuts. The buildings are older, so they might not always have the wider doors and accessible restrooms. It does require that our guests and our travel agents research the different ports of call to see which ones are more suited than others.

“The Mediterranean is increasingly becoming more accessible, and the more northern and the western you go, it gets more accessible. When you look at bigger ports, especially those that have hosted the Olympics, because they hold the Paralympics, they have increased accessibility over the years.

“Athens and Barcelona are recent examples, so they may have more accessible taxis and motor coaches and overall facilities for people in wheelchairs. You go to some of the smaller ports, like Santorini, and the island ports, and there are mixed levels of accessibility.

“The challenge we get with different ports of call is usually with accessible vehicles; if a guest has some ability, or a caregiver who can assist them into a regular taxi, or they can go up a few steps into the motor coach, more options become available.”

To improve accessibility for guests with more limited mobility, Pettit says that, in Europe, Royal Caribbean has created Easy Tours—a modified version of the cruise line’s panoramic city tours.

“It’s a narrated ride throughout the city on a bus, with a couple of opportunities to get off to look around. These have a motor coach with a lift, or, more often in Europe, a van with a ramp in the back. These are in about 80 ports and are a great option for guests in wheelchairs or scooters, who have limited capability.”

Rail travel has its advantages for those with chronic conditions and medical devices, and train trips throughout the U.S. and Canada can be a good place to start.

Says traveler Milli Washock, “I always used to find a plug at the gate [at the airport] for last-minute charging, but I don’t fly anymore. Traveling [domestically] by train is easier in that there is a plug by every seat. It takes longer, but you have a bigger selection on dining and with movies if you plug in your laptop or device.”

With input from disability advocacy organizations, over the past five years, Amtrak has made accessibility improvements at more than 200 stations. All Amtrak trains have accessible seating and restrooms, and all long-distance trains have accessible bedrooms. Amtrak also offers a discount to passengers with disabilities and their companions.

For more information, go to www.amtrak.com/accessible-travel-services.

Other countries have also made recent improvements in rail travel accessibility. Australia, for example, offers a host of services and special deals to meet the needs of travelers with disabilities, including accessible services in most of its trains. For more information, go to www.australiaforall.com

But accessible international rail travel might be even closer than you think. “We are more accessible than flying or taking the bus,” says Jacques Gagnon, senior manager, media and community relations for Via Rail Canada, Canada’s national passenger rail service.

“When you take Via Rail, you can enjoy traveling while looking out the window and seeing scenic portraits of cities, prairies, and the Rockies, while being comforted by having a quiet time.

(Top) Indian Pacific train journey across Australia. (Left) Via Rail Canada, Toronto-Vancouver route. (Bottom) Via Rail Canada, Montreal-Halifax route. (Right) Via Rail Canada, Montreal-Halifax route.
(Top) Indian Pacific train journey across Australia. Photo © Great Southern Rail. (Left) Via Rail Canada, Toronto-Vancouver route. (Bottom) Via Rail Canada, Montreal-Halifax route. (Right) Via Rail Canada, Montreal-Halifax route. Via Rail photos by Bianca Courtemanche.

“It’s spacious. We have invested in making cars accessible, roomy, and user-friendly for people with limited mobility and special conditions. We also cater to gluten-free and other special dietary needs, with advance notice.

“Canadian laws and regulations provide ample services for people with limited mobility. It’s part of the fabric of Canada to provide accessibility to its citizens and to travelers.

“At various stations we have lifts to allow people with wheelchairs to board the train. We have a dedicated area where the person will anchor his or her wheelchair safely.

“We also allow someone, or a service animal, to accompany that person and ride at no additional cost. We ask to receive 24-hour, or ideally, 48-hour advance notice that someone with a specific condition will be boarding the train so the attendants can recognize and attend to their needs.”

For those interested in viewing gorgeous vistas while riding the rails, Gagnon says, “There are two long-haul routes—one is The Ocean—a 22-hour journey from Montreal to Halifax. The train travels along shorelines of the Atlantic Ocean and has a panoramic car.

“The other one is The Canadian, between Toronto and Vancouver, through the Canadian Rockies and the prairies. It‘s a very scenic three-and-a-half-day journey. The track goes across terrain where there are no highways and roads. It’s very unique—even in the summer time, the Rockies are covered with snow. It’s a very elevated, beautiful terrain, with views of the Pacific Ocean.” For more information on Via Rail, go to https://www.viarail.ca/en/travel-info/special-needs/accessibility

Getting as much information as possible and alerting the airline to your needs in advance, as well as allowing time for delays, are critical when traveling by air. Be sure to factor in potential traffic issues, lengthy distances between gates, crowds, long lines and flight delays when connecting. And don’t be hesitant to ask for assistance, such as wheelchairs or electric carts, experienced travelers advise.

One flyer says, “My husband has Huntington’s disease, and for him, we found going on the plane with assistance has given him more time to get to his seat without people pushing and being impatient. Also, we request wheelchair assistance at all airports.

“If you’ve ever been to Jamaica, Miami, or Atlanta airports, then you know it’s like walking a mile from one point to another. Imagine if you only have half an hour to get from one gate to the other. Always ask for assistance when needed. Most of the people who work at airports are only to happy to help.”

Traveler Keti Galanos says, “Panic attacks are commonplace for those of us afflicted. I am at the near norm readings for PAH, yet will take medication for the next year. I request a wheelchair, as I cannot run between gates in the airport, and I always panic and am out of breath.”

Traveler Cheryl Kneal recalls, “I traveled to Chicago from Orlando over Christmas. I had my portable oxygen concentrator and had the airline take me to the gate for departure, and then, when we arrived, to where I was picked up, to conserve energy.”

Kneal adds that, to deal with stress and panic attacks about running out of oxygen, she meditated frequently during the two-hour flight.

For those with limited mobility, addressing needs step-by-step ahead of time is key to alleviating stress and making travel as hassle-free as possible. When making your airline reservation, let the agent know if you’ll be traveling with a wheelchair or scooter, if you’ll need one at the airport, and if you’ll need to transfer to one of the airline’s aisle wheelchairs (a narrow wheelchair designed to fit aircraft aisles) to help you board or deplane. Also ask if you can use the aisle wheelchair during the flight to get to the bathroom.

Ask to keep your wheelchair until you get to the gate, check it there, and have it returned to you at the gate on arrival. (Depending on the type of wheelchair you have and space available, it will either be stored in the cabin or in the baggage hold during the flight.)

Flight attendants can help passengers use the aisle wheelchair to get to the restroom, but not in the restroom, and many onboard restrooms are not wheelchair accessible. Flight attendants are also not required to lift or carry passengers. Some fliers with limited mobility limit fluid intake before and during a flight to prevent the need to use the restroom, however doing so can present the risk of dehydration and other medical problems.

Flying can also be unpredictable. Air traffic and weather delays can mean the plane is stuck on the runway before takeoff or circling for an extra hour before landing. If you have limited mobility, it might be advisable to consider flying with someone who can help you in the restroom, or catheterization, rather than limiting fluids.

Candy Harrington advises those who have concerns about navigating airport security in wheelchairs and with medication and medical devices to check the TSA guidelines beforehand.

“Although the TSA is exempt from the ADA and the ACAA, they have developed specific guidelines for dealing with disabled passengers. They list these guidelines on their website, so it’s a good idea to familiarize yourself with them, so you will know what to expect.”

Traveler Ruth Cozad says that she was pleasantly surprised with her TSA experience. “We went to Hawaii with both oxygen and CPAP machines and had an amazing trip. Going through security was my big worry, and it went so smoothly.” For more information on TSA guidelines, go to www.tsa.gov/traveler-information/travelers-disabilities-and-medical-conditions or call the TSA Cares hotline at 855-787-2227 with any access-related questions prior to travel.

For some travelers with chronic conditions, especially those traveling with oxygen, driving can provide the most stress-free journey, and help them slowly ease into travel. Taking short trips from one central location can also help to conserve energy and get the most out of a trip.

Cozad suggests, “My advice would be to stay in one location and take day trips, instead of moving each day. We often take driving trips that last several weeks, and those are easy enough, with my husband unloading and reloading oxygen and CPAP equipment, plus our luggage.”

Milli Washock says, “A lot of people are using the Inogen One portable oxygen device, and it’s nice to go on a plane or a ship. I find it great for long-distance auto travel.”

A potential problem, though, says Washock, is that the device does not have a HEPA filter. She recalls that once, while she was dining out, “a man was smoking a cigar in the nearby bar, and suddenly I was ‘smoking a cigar’ and could not breathe.”

In this case, Washock found that traveling by car had benefits. “Thankfully, I had an oxygen E tank in the car and did not have to permanently abandon my dinner, but it gave me something to think about.”

Despite such challenges, Washock says, “It is good to travel if you can. Just because our bodies do not cooperate the way we want does not mean our brains and lives have to shut down. And, she notes, travel, no matter the distance, can be enhanced by state of mind. “Take it slowly, and enjoy the world around you, whether traveling to your porch or across the world. Each day is a new day.”


See Part 2 of our accessible travel story, highlighting some of Washington, D.C.’s top accessible attractions!

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Video Testimonials

Posted on by CVCinfo

David Haygood, a patient with Alpha-1 Antitrypsin Deficiency tells about his experience with Caring Voice Coalition.

Cheryl Staveley, parent and caregiver of Meghan Sullivan, a Huntington’s Disease patient, tells her story.

Kindra Cansler, caretaker for her son Justin, an Infantile Spasms patient, tells her story.

Joyce Scannell, a Narcolepsy patient, tells her story.

Beverly McGregor, a Pulmonary Hypertension patient, tells her story.

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Supported Diseases

Posted on by CVCinfo

CVC Disease Funds

Alpha-1 Antitrypsin Deficiency (FUND CLOSED)
Chronic Granulomatous Disease
Complex Partial Seizures
Cutaneous Lymphoma
Factor XIII Deficiency
Gaucher’s Disease
Huntington’s Disease
Idiopathic Pulmonary Fibrosis
Infantile Spasms
Inflammatory Eye Disease
Lennox-Gastaut Syndrome
Nephrotic Syndrome
Orthostatic Hypotension
Pulmonary Hypertension
Severe Chronic Pain
Steven-Johnson Syndrome
Thalassemia Syndromes

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We Can Be Heroes: Patricia George, M.D.

Posted on by Charlie

Patricia George, M.D.Patricia George, M.D., spoke with Community about her work in pulmonary transplant medicine, her HIV-PAH research, and her motivation as a member of PHA’s four-woman Team PHenomenal Hope in the nine-day 2014 Race Across America.

Going into medical school, I had passion for and experience in immunology research, so transplantation was something in which I was always interested. And like many who go into pulmonary medicine, I was initially drawn to it through my medical school and residency rotations in the medical intensive care unit. I enjoyed pulmonary physiology, and the work and pace of critical care medicine.

In addition, as a medical student, I met a patient with cystic fibrosis awaiting a lung transplant in the medical intensive care unit. I got to know her and her mom, and some of her life story, and wanted to be able to help people like her with lung disease. So that led me to pulmonary medicine and pulmonary transplant medicine.

My research involves looking at the mechanisms of HIV-associated pulmonary arterial hypertension (HIV-PAH). Pulmonary arterial hypertension is quite rare, however in patients with HIV, it affects at least 0.5 percent, and perhaps more, according to recent studies. That’s at least one in [every] 200 patients!

Advances in HIV care have changed the landscape for people with HIV, and many now label HIV a chronic disease. So medical complications, like HIV-PAH, become extremely important to study and hopefully help people live longer and live better.

Team PHenomenal Hope came together with people passionate about cycling and raising awareness. As an avid cyclist, it had long been a dream to someday race in the Race Across America (RAAM).

Stacie Truszkowski, one of my close friends in the cycling community, also shared this dream. So, in 2011 and 2012, Stacie and I reached out to our endurance cycling friends whom we thought might be crazy—er—passionate—enough to do this as well, and in 2012, with the addition of Anne-Marie Alderson, Ryanne Palermo, and Kate Bennett as our crew chief, our four-woman cycling team was born.

We organized this Pittsburgh-based team, met with our friends at PHA, as well as our earliest sponsors, and formed Team PHenomenal Hope. Later that summer, we added to this group Greta Daniels, assistant crew chief and alternate racer, and Sara Harper, alternate racer and crew.

Our mission is to dedicate our training and racing to those who live with pulmonary hypertension, to raise public awareness about the disease, and to raise funds to find a cure.

For more articles like this, click here to subscribe to COMMUNITY magazine!I started biking during pulmonary fellowship. I wanted to get back in shape, and a new women’s cycling team called Steel City Endurance was forming. I joined them in the inaugural year, and became totally enamored with biking and bike racing and met a lot of really neat people.

I enjoy being outside, escaping the stresses of my sometimes hectic lifestyle. As for endurance cycling, I enjoy pushing my body and mind to some sort of limit. It allows you to lose yourself in the present—how you’re feeling at that time.

We’re working with our team coach, who’s helping coordinate our training schedules so that they build and peak at the right time. Training is about consistency—getting the workouts in, getting stronger every day (except rest days). Eating healthy and getting enough sleep are crucial too.

As a team, we’re racing the whole race as a relay. To make the time cut-off and make it to the finish line as fast as we can, we divide up the ride into 20-to-30-minute segments.

On a four-person team, typically two riders will be out on the road, trading places in 20-to-30-minute pulls (one riding, the other in a support vehicle leapfrogging ahead for the exchange to happen). This pair of the four-woman team will ride for four to six hours, while the other pair rests, eats, sleeps, and recovers. It goes 24/7, from the time the gun goes off until we cross the finish line.

From what I hear, mental toughness will be one of the biggest challenges during RAAM. Those who have done it say that, at about day four or five, the sleep deprivation kicks in, and the reality of the Midwest flatlands also hits you. I know there is beauty in rolling plains, but at that point in the race, it may be tough to see it.

During RAAM, the crew is the essential group of people that will get us from Oceanside, California, to Annapolis, Maryland. The crew chief, Kate Bennett, is in charge of coordinating the drivers, navigators, medics, mechanics, nutrition, making sure we’re on course, and that people— including crew—are getting enough sleep, food, etc. A race with this relay between four racers, moving across the country with an RV, two support vehicles, and 13 crewmembers is quite an undertaking.

The greatest source of inspiration is the PH community. When I think about how hard it may be to be on the bike, mentally or physically, I think about what my patients go through on a daily basis.

Team PHenomenal Hope members Ryanne Palermo, Patty George, Stacie Truszkowski, Greta Daniels and Kate Bennett.
Team PHenomenal Hope members Ryanne Palermo, Patty George, Stacie Truszkowski, Greta Daniels and Kate Bennett.

I get to choose to ride my bike, to push myself through discomfort. My patients don’t have such a choice. They wake up and live with pulmonary hypertension every day, and face whatever that day may bring, and many do so with such grace. So when I’m feeling less than motivated, I often think of people I know living with PH, and it motivates me to get this job done.

Likewise, in my practice, I am regularly reminded of the need for a cure. I often evaluate patients with pulmonary hypertension in need of a lung transplant. For this group of patients, they often no longer are responding to medications. It is a reminder that, while we have come so far, and many patients do respond to medical therapies, we still need a cure.

In my job, I also conduct PH research, and know firsthand how important funding is to exploring the frontiers in science. It makes it all the more important to me that Team PHenomenal Hope is raising money for PHA to fund grants and help other scientists have funds needed to find a cure.

We have something truly special with our partnership with the Pulmonary Hypertension Association. PHA launched a Race of Our Lives campaign, and we have been amazed how people in the community have organized their own Unity events, walking, riding their bikes, doing whatever they can to raise awareness about PH and join us in raising funds to find a cure.

Team PHenomenal Hope is bigger than four of us on bikes, or the 17 of us crossing the country. This is actually a huge team that spans coast-to-coast.

Pulmonary hypertension is a rare disease that can affect anyone, from children to adults, men and women, and people of all races and ethnic backgrounds. Initially, it is often misdiagnosed as another pulmonary condition, taking on average over a year to make the correct diagnosis and get the proper treatment.

Although it is a rare disease, it is important for doctors to at least think about pulmonary hypertension in their differential diagnosis when faced with a patient with shortness of breath, because without considering it, the diagnosis won’t be made.

Fortunately there are many medical treatments on the market, changing the prognosis for many who have this disease; however there still are people who do not respond to therapy, and to date there is no cure. Team PHenomenal Hope is working with PHA to do something to try to change that.

 Team members Palermo, George, Truszkowski, and Anne-Marie Alderson at University of Pittsburgh Medical Center.
Team members Palermo, George, Truszkowski, and Anne-Marie Alderson at University of Pittsburgh Medical Center.


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High Notes

Posted on by Charlie

New York-based singer, songwriter and musician Chloe Temtchine has won rave reviews and awards for her solo work and collaborations with other top music industry artists. On March 29, 2014, Temtchine’s new song, “Be Brave,” will be released on iTunes, with fifty percent of the proceeds benefiting PHA. Diagnosed with pulmonary hypertension in 2013, Temtchine performs with her oxygen tank, which she has dubbed “Steve Martin,” alongside her. Community spoke with Temtchine about life, music and pulmonary hypertension.

Chloe TemtchineWhen did you start singing and songwriting? Who and which styles are some of your biggest influences?

I started singing at about the age of six. My father used to take me to a Baptist church in Harlem, on Sundays, where I listened to gospel music for hours. That’s where it all began.

I’ve always had very eclectic taste in music: from artists like Edy Phenomene (French dancehall), to Smokie Norful and Kim Burrell (gospel), to Stevie Wonder and Sam Cooke (R&B/soul), to James Vincent McMorrow and Ray Lamontagne (singers/songwriters), to Eric Reed (jazz). The list could go on forever.

When and how were you first diagnosed with pulmonary hypertension? What were your initial symptoms?

In March 2013, after my cardiologist reviewed an echo and listened to my heart, I was diagnosed with severe pulmonary hypertension and was rushed to the ER. The shortness of breath, lung pain, and fatigue that had started five years previously had become progressively worse, and I had reached a point where I could barely move. Getting to the bathroom was a major accomplishment! Then my heart started beating out of my chest. And then, together with the continued chest pain that accompanied every breath, I suddenly put on 10 pounds of water weight overnight.

Album Cover for Chloe Temtchine's 'Be Brave'
Chloe Temtchine’s “Be Brave” album cover

What is “Be Brave” about?

After being diagnosed, I found myself spending so much time trying not to die that I had forgotten to live. “Be Brave” is about making the choice to live.

Every time I went to the hospital, I came home feeling that there was very little hope. I realized that if I didn’t shift my consciousness quickly, my condition would continue to get worse.

I’m a big believer that our minds play a huge role in the state of our health. Keeping my mind in the right place is so important to me that I decided to write a song about it. I wanted to remind myself, and anyone else going through a similar experience, that any challenge had the potential to be an opportunity.

What inspires you in songwriting?

I’m much more comfortable expressing myself through song. Music captures the way I feel in a way that words without melody can’t seem to do.

For more articles like this, click here to subscribe to COMMUNITY magazine!What have you learned in dealing with pulmonary hypertension? What have been some of your biggest challenges, and how have you dealt with them?

Although I would do just about anything not to have pulmonary hypertension, I’ve understood through this process that there are many lessons I’m meant to be learning. I am very grateful for the perspective it has given me with regard to my life.

My biggest challenge was seeing a future when I was told that it was highly unlikely that I would have one. This continues to be my biggest challenge.

What do you think is most important for the newly diagnosed to know about pulmonary hypertension?

For the newly diagnosed, I would say: Surround yourself with positive people who instill hope in you, eat a very healthy diet (plant-based, if possible), go to pulmonary therapy, focus on other people’s success stories, and most importantly, believe that it is possible to get better no matter what you’ve been told.

How does being a singer, musician and songwriter help you deal with pulmonary hypertension?

I think that being inspired and passionate in general is helpful to anyone. I also think that it’s very important to express yourself in order to keep yourself in balance. Through music, I’m able to get out all of the things that I would otherwise potentially keep in.

What is next for you in terms of treatment for PH?

I’m not totally sure at the moment. Because there is a belief that I may have pulmonary veno-occlusive disease in addition to PH, my doctor is going very slowly with the medications, which I’m very grateful for. Lung transplantation has been suggested, and although I’m thankful that it exists as an option, my goal is to stay away from it, if at all possible.

What’s next for you in music?

My next goal in music is to finish writing the album I began when I got out of the critical care unit and to perform as much as possible.

What do you enjoy most about music?

I love that music has always had the ability to completely alter the way I feel. It has helped me be hopeful during very difficult times. I love the idea of creating something that could not only alter my own state, but that also has the potential to alter the state of someone else who may be in need of some state-altering!


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Newsmaker Q&A with Thomas L. Spray, M.D.

Posted on by Charlie

Thomas L. Spray, M.D., is chief of the Division of Cardiothoracic Surgery at The Children’s Hospital of Philadelphia, director of the hospital’s Thoracic Organ Transplantation Program, and Professor of Surgery at the University of Pennsylvania School of Medicine. Community recently interviewed Dr. Spray about pediatric lung transplantation.

What are the most important considerations for parents when making decisions about pediatric lung transplants?

The fundamental issue here is that, lung transplantation is, unlike heart transplantation, not associated with as good long-term survival. In some ways it’s somewhat palliative. Five-year survival for lung transplant is about 50 percent. And despite lung transplants being around now for about 25 years, there hasn’t been a lot of improvement in survival.

The lung is an immunocompetent organ, unlike most other organ transplants. It has the disadvantages of being connected to the outside, so it’s always at risk of infection. Also, there are lymph nodes in the lungs, which probably make them more susceptible to rejection and latent chronic rejection, which is where most lung transplants eventually fail; something called bronchiolitis obliterans—scarring of the small airways of the lungs—which is thought to largely be a chronic rejection problem.

Having said that, there are isolated patients who do extremely well and do well over the long term, but if you just take the averages, the average survival is about five years. So that’s the disadvantage.

On the other hand, for many patients, especially pediatric patients, their survival is going to be measured in a matter of a year or less if they don’t get a lung transplant. So it’s successful, but it’s not perfect by any means. It’s generally very much palliative for children as well.

To what degree should the child be involved in the decision?

To what degree the child should be involved in the decision is somewhat age-dependent. When you have a lung transplant, you’re basically exchanging one disease for another one, because you then have to be on medicines to prevent rejection, and you have a certain amount of medical surveillance necessary.

Unfortunately lung transplantation is still a procedure that has a significant risk associated with it. The lungs cannot work sometimes. The outcome cannot be positive if you get a viral infection of the transplant early on—it can destroy the lungs. And that’s very hard for anyone to survive.

I can remember some patients though, with cystic fibrosis, after lung transplant, even when the lungs went bad, and they ultimately died, having said during the time they were alive, that it was worth everything just to breathe normally for a change. So you have to put it in perspective.

When you get into the teenage years, I think it’s appropriate for the child to be involved in the decision. I think it’s especially important for teenagers, who unfortunately have a fairly high risk of not being compliant with medical management, and then they reject their organs and die.

Just being an adolescent is a risk factor for transplant, it appears. It’s a difficult time. That’s why when you’re talking teenagers, it’s very important that they be involved in the decision process, so that they recognize that they have to be involved in the medical management. They have to take their medicines faithfully. They have to be seen frequently. And if they aren’t willing to do that, then it’s kind of foolish to go down that pathway.

What have been some of the advances in pediatric lung transplantation?

Patient room at The Children's Hospital of Philadelphia

Patient room at The Children’s Hospital of Philadelphia

Most of the advances in transplantation, in terms of survival, over the last 15 years, have been at the early period after transplant. In other words, we’ve been able to transplant more complex patients, who’ve had multiple previous operations, who were sicker waiting for transplant.

We’ve evolved to the point where it’s now possible to support patients on an assist device called ECMO while waiting for transplant and still have them able to be up and around and maintain their ability to breathe, so that it makes it easier after the transplant. So there are some advances, but there haven’t been any major advances in immunosuppression.

We have experience now transplanting newborns all the way up to adults. We’ve had experience with many kinds of complex congenital heart disease and lung transplants. We have experience in pulmonary hypertension. Those are the more common causes in children.

I think it’s important to note that in the pediatric world, the most common indications for transplant are considered the higher risk indications in the adult world. For example, in adult lung transplant, the most common indication is emphysema. Emphysema is a more straightforward condition to treat with lung transplant than any of the diseases that we see in children, and emphysema is virtually unheard of in children, so pediatric transplant by its very nature is a higher risk population.

What percentage of pediatric transplant patients have pulmonary hypertension?

At The Children’s Hospital of Philadelphia, at least 50 percent or more have congenital heart disease or pulmonary hypertension, and relatively fewer have cystic fibrosis. Part of that is because the management of cystic fibrosis has improved over the years such that there are relativity fewer children who require transplantation for cystic fibrosis than in the past. So, most people with cystic fibrosis can get to adulthood before they have enough lung deterioration to require transplant, and therefore get transplanted in adult centers now.

When we first started doing lung transplant in about 1990, when I was in St. Louis, cystic fibrosis was a common indication, because children under 18 with severe cystic fibrosis would come to St. Louis for transplant. In Philadelphia, we see less of that and more pulmonary hypertension and congenital heart disease.

What drew you to the lung transplantation field?

I started out as a congenital heart surgeon. Congenital heart surgeons all have to be adult heart surgeons first. I did adult and congenital heart surgery at St. Louis Children’s Hospital. I was recruited to come here to take over from William Norwood, a very well known surgeon, who went to Europe to start a new program.

When I came to Philadelphia, there wasn’t a lung transplant program in this area. So I started a pediatric lung transplant program when I came in 1994, having started the program in St. Louis in 1990.

I think my interest in lung transplant came from patients I saw in St. Louis who had no real, good option for repair of their heart, because they didn’t have good lungs to push blood into. There are certain types of congenital heart disease where that’s the case, where you could repair the heart defect if you had pulmonary arteries to pump blood into.

But children who don’t have that became progressively more cyanotic. I saw some of these children and I thought, ‘If we could just put new lungs in, we could fix the heart.’ So that’s what got me interested initially, and then that expanded to all the other potential reasons for lung transplant, of which there are many.

What are some of the differences in quality of life after a successful pediatric lung transplant?

Art therapy at The Children's Hospital of Philadelphia

Art therapy at The Childrens Hospital of Philadelphia

Quality of life depends on the patient’s condition before the transplant, but the majority in the pediatric population are extremely debilitated. They often have cystic fibrosis. They’re chronically infected. They have poor lung function. They have very little exercise tolerance. Patients with PH may have heart failure also. So the quality of life prior to transplant is very poor.

The waiting times are so long for lung transplant that most children deteriorate significantly while waiting in the hospital. They’re sometimes waiting in the hospital for a year or more.

So the quality of life after transplant, while vastly improved, takes a while for them to recuperate. I think what people often don’t realize is that if you’re sick for months and months prior to lung transplantation, there’s a lot of rehabilitation necessary. Even after a successful lung transplant, it’s not like patients are going home in a week. Many of them have to stay in the hospital for months while they’re literally recuperating and rehabilitating themselves from being chronically ill for the previous several years.

How much interaction do your patients have with the Child Life, Education and Creative Arts Therapy department at The Children’s Hospital of Philadelphia?

They have a great deal of interaction, especially while waiting. Children who are sick and in the hospital and literally waiting for sometimes months and months for organs to become available need that kind of interaction. They need to have play.

They need to have creative interactions, and that’s why the Child Life Department is so important for all the patients in the hospital, but even more important for those who are chronically in the hospital for long periods of time. They need to have that kind of stimulation. They need to be able to be involved in arts and crafts to keep them occupied, literally, to help them develop while they’re waiting.

What are some ways that families can cope with being on a waiting list?

Everyone, I think, does that differently. I think it’s always difficult for families to recognize that a waiting list is exactly that. You have no idea when organs might become available.

There are times when organs are available, and we tentatively accept them, and then they deteriorate to the point that we can’t use them. So the families have situations where their hopes are raised that they’ll have a transplant, and then it falls through, and then they’re back waiting again. So it’s a difficult thing for the families.

I think the families need to recognize that donor organs are hard to come by; they’re very scarce. The organ system is as fair and open as people can make it. It’s constantly trying to be as completely open and straightforward as possible and to not disadvantage anybody and make everybody on an equal playing field. But these are difficult things sometimes.

Transplantation in some ways is kind of a fundamentally flawed strategy, because you have to have a tragedy to have a miracle. Someone has to die for organs to be available, and as a physician and surgeon, I don’t want anyone to die. In a way, I don’t want there to be more donors.

On the other hand, I think the donors who are available should be used as maximally as possible, because there are so many children who need the organs. It’s a constant battle to try to use everyorgan you possibly can. But then some of them have problems and don’t work. That’s unfortunately the chance you take.

What programs are in place at The Children’s Hospital of Philadelphia to help families afford the cost of pediatric lung transplants?

Art therapy at The Children's Hospital of Philadelphia

Art Therapy at The Children’s Hospital of Philadelphia

There’s a whole process before someone is listed for transplantation that involves evaluating their financial situation. The hospital has many programs to try to get them into programs that will provide coverage. Because it makes little sense to do a lung transplant if you have no coverage for medications after the transplant, which happens in some crazy insurance arrangements.

The hospital sometimes will help families get Medicaid or some other government program that will at least provide them with follow-up medications and follow-up care. There’s a whole financial counseling group that works with families in relation to transplant, because you have to recognize it’s not a one-time deal. It’s like a new disease, if you will, transplantation.

What else is important to stress about how pediatric lung transplants are different from adult transplants?

The diseases are different, and they’re more complex. Many of the [pediatric] patients have had previous surgeries, which complicate the transplant significantly, in terms of bleeding and other issues.

I’ve said many times that the hardest part of transplantation is not putting the new organs in; it’s getting the old ones out. That can be extremely difficult due to the scarring and inflammation and previous infections. It can sometimes be extremely difficult just to get the old organs out without damaging other important structures. They can all be stuck together in the chest.

That’s one major challenge with lung transplant, especially in children. It’s not as common in adults. Most of the adults with emphysema have not had significant previous surgeries; they don’t have a lot of extra blood vessels.

Many of the children who need transplant are also ‘blue.’ They’re cyanotic. They don’t have normal oxygen levels. That stimulates development of blood vessels in the chest that can be very difficult to control at the time of transplant, and bleeding is much more of an issue.

Then of course we’re dealing with different sizes. In adults, they’re using mostly adult lungs and some teenage lungs. But in pediatric transplantation, we have to list patients in a very discrete size range, because we do transplants in patients all the way from newborns up to adult-size teenagers. So we have to have the ability to put very small lungs in small children.

We do occasionally use lobes or parts of lungs from adults in small children, which is something the adult world rarely, if ever does, because we have to deal with this wide size range.

All photos courtesy of The Children’s Hospital of Philadelphia

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Donate Now

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Give Financially

Caring Voice Coalition has established a safe, secure way for you to make an on-line donation. By donating to Caring Voice Coalition, you are making a difference in the life of a patient. Caring Voice Coalition and our donors have a common goal – to help as many patients as possible. Caring Voice Coalition is a qualified 501(c)(3) tax-exempt organization and donations are tax-deductible to the full extent of the law. Our Federal EIN is 26-0058446.

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Newsmaker Q&A: Rino Aldrighetti, President of PHA

Posted on by CVCinfo

Rino Aldrighetti was hired as the first part-time professional staff member of the Pulmonary Hypertension Association. In the years since, he’s assumed the title of President of the organization, enhanced PHA’s scope, built a full-time staff, and advocated tirelessly to increase awareness about pulmonary hypertension. Here, Community talks to Rino about where PHA has been, and where it’s going.

Describe the journey in becoming President of PHA.

In 1998, seven years after PHA’s founding, the organization’s volunteer leadership decided to build a staff. Their first step was to advertise for a part-time executive director. They put an ad in the Chronicle of Philanthropy, running it once.

At the time they were doing this, I was a non-profit consultant. I had just finished a five-year project for one of my larger clients and was getting ready to do what I always did when I was preparing to think through a new direction, begin a three-day retreat. The day before I was to begin, I picked up the Chronicle of Philanthropy and saw a brief two-line ad: “Small rare disease association looking for part-time executive director”.

I responded and was invited to meet with an extraordinary person, Bonnie Dukart, who was PHA’s president. Bonnie, who had been diagnosed with PH shortly after graduating from college, explained to me that the goal was to bring on a person who could increase PHA’s budget so that the organization could do more. Following that meeting, I had the opportunity to meet the board and quickly became aware that I was in the presence of heroes.

I soon accepted the board’s offer and became PHA’s first (part-time) professional staff person in 1999. By 2001, we had increased PHA’s income from $132,000 to $1.1 million and built a small part-time staff. The board then invited me to lead a new full-time staff. The work has always been a privilege.

What are some of the challenges you face when trying to increase awareness or support a rare disease like pulmonary hypertension?

Our greatest challenge in raising awareness about PH is our relatively small numbers. A rare disease in the U.S. is defined as one with 200,000 or fewer patients. PH has 20,000 to 30,000 diagnosed patients.

Given that reality, each person’s decision to make a difference is of enormous importance. At PHA, our mantra is that any person whose life is touched by PH has the right to fight back as much or as little as health and interest allow. Over and over again we have seen what the ability to influence positive change means in people’s lives. We may have 30,000 patients living with this disease, but each has family members and friends, neighbors and medical professionals who care and are ready to help. Harnessing that energy with a strong community is what makes a real difference.

What are some of your goals for PHA for 2013?

In this economic and political environment, organizations that cannot adapt will ultimately decline. At PHA, we have been working hard over the past year to introduce new ways to support our research and patient-serving programs. In December, we opened PHA’s first three chapters – in New York, Chicago and San Francisco. This is a pilot for us to build a professional events structure that will develop a larger population of supporters for our core programming in communities around the country. If we are successful in our first year, we will implement a five-year program to expand the network. It’s an ambitious effort to not only assure our sustainability, but to make sure that our ability to serve the needs of our community is not limited by funding restrictions.

PHA’s early diagnosis program, Sometimes It’s PH Campaign, was launched at our 2012 International Conference and has generated early excitement in the U.S. and other nations. It came about following research indicating that despite all the increased visibility for PH during the past 20 years, the time from onset of symptoms to point of diagnosis has not reduced. This may not have been a problem two decades ago when there were no treatments; however, today with nine treatments and more on the way, getting patients diagnosed so that they can take advantage of these treatments is hugely important.

Why should a PH patient join PHA?

PHA is more than an organization. It’s a community, a place where people understand and help each other get through the challenges of this difficult disease. The strength of the community has value for the individuals who choose to join. It also has value for the whole, for all who live with the disease through our collective ability to develop public awareness, drive advocacy and sustain helping networks. These are things we can only do together and that will create opportunities for better tomorrows for everyone.

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Seeking Support

Posted on by CVCinfo

Whether you’re newly diagnosed with a condition, or have been living with it for years, getting the support you need is crucial to maintaining your best healthy life, both physically and mentally.  There are tons of ways to participate in support groups, whether it be through an actual physical get-together, online meetings, or even actively participating in message boards.

The benefits of support groups are obvious – being in an environment in which other people understand you and can share helpful advice and motivation is always going to be an aid, however it’s important to see how this engagement can benefit your personal relationships.  It can be truly difficult for someone to give the support they want to a person with a chronic illness, because they don’t truly understand or know what to say.  And it can be equally hard for the person affected to really share in words what is going on in their minds and with their bodies.  Going to an outside source of support can create a better bond between the caregiver and the person who is ill.

Often times, people are hesitant to go to support groups because they feel what they are going through is too personal or intimate to share with a room full of strangers.  So, if you’re feeling hesitant, think about the benefits of just going and listening.  It can be more beneficial than you may believe to just be silent for an hour and soak up what others are saying.  And if going to a support session seems like you are relinquishing some of your independence, just remember that everyone needs a helping hand sometimes, just don’t be afraid to take it.

Below are some great resources for support groups from some of our friends in the rare and chronic disease community.  Also, don’t hesitate to ask your doctor or medical professional where to look.

Pulmonary Hypertension Association
Pulmonary Hypertension Central
MD Junction Pulmonary Hypertension Online Support Group
Daily Strength Pulmonary Hypertension Online Support Group
Pulmonary Hypertension South Africa

Pulmonary Fibrosis Foundation
Daily Strength Pulmonary Fibrosis Support 
Coalition for Pulmonary Fibrosis
MD Junction Pulmonary Fibrosis Online Support Group

Narcolepsy Network
Daily Strength Narcolepsy Online Support Groups
Narcolepsy Online Support Groups

Huntington’s Disease Society of America
Daily Strength Huntington’s Disease Online Support Groups
Huntington’s Disease Lighthouse Families Online Support Groups
Huntington’s Disease Advocacy Center Message Boards 

Huntington’s Disease Youth Organization 

Infantile Spasms Center
The Infantile Spasms Community Board

Alpha-1 Association
Daily Strength Alpha-1 Online Support Groups

Are there support groups which you have found helpful?  Please share with us!

Posted in Caregivers, Diseases, Featured, Media Center, Uncategorized | Tagged , , , , , , | 3 Comments

Cindy’s Story

Posted on by CVCinfo

I have had a long history of coronary heart disease so it is not uncommon for me to experience angina on a regular basis. However, in November 2009 it became stronger than usual and my doctor ordered first a stress test, then a heart catheterization. The doctor preforming the cath found no real changes in the left side, so he decided to do a right sided cath and check the pulmonary artery pressure, which was elevated but not too high. He told me that I was begining to show signs of pulmonary hypertension.

I was not familiar with this and when I went to the follow up with my doctor he put me on a new medication which I assumed was for the PAH. It wasn’t, so in September 2010 I had to go to the ER due to severe angina again. Another heart cath was done including a right sided one to further assess the pulmonary pressure. It had gone from mild to severe in less than a year with a pressure of 80.

I discussed this with my doctor and asked why the new medication had not worked and I was then told that the medication he put me on was not for the pulmonary hypertension. I asked why he had not addressed the PH and he shrugged and left the room. It was at that time that I found a new cardiologist as well as a pulmonologist who put me on PH meds. I was on the lower dose for a year and a half and there were episodes of shortness of breath and I asked about possibly increasing the dosage, but the doctor did not want to do that. I had planned a trip out of the country for vacation and the doctor did a HAST test and said I was fine to fly without oxygen.

He was wrong. I thought I was going to die the enitre trip. I became very sick and really could not participate in my own vacation. By the time I got home I was even sicker. I went to the doctor to explain what had happened and he said it was not from the PH and that I should talk to my cardiologist. It was at that point that I looked for a PH specialist that my insurance accepted and found one in my general area. He is still in the process of adjusting my medications and has done two cardiac caths, one right side the other left side. I have still not been able to return to what I was able to do before the trip and the doctor isn’t sure if I will ever be able to.

When I was put on the PH meds, I had no idea how expensive the medication was and when they told me what my co-pay was I said, “Well they might as well shoot me because there is no way that I can afford that as I am on disability.” It was then that the speciality pharmacy referred me to Caring Voice. They made everything so easy and within only a couple of days had me approved for the balance of that year as well as the following year. I cannot thank them enough for making such a difficult time easier as I don’t have to fight to get my medication.

So that’s my story so far. I am still working on getting back to where I was before the trip and I am determined that I will do so. I will just have to take baby steps, but I know myself, and I will get there.

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Jewell’s Story

Posted on by CVCinfo

I would have to say my situation started back in the ’90s. I was experiencing a shortness of breath and would be sick with bronchitis a lot. I decided to go see an allergist and he put me on shots, I took them for about 10 years. It did help me but I still had the shortness of breath. My internal medicine doctor back then decided to do a chest x-ray and when she saw the results she decided to send me to a Pulmonary Specialist.

They diagnosed me with mild asthma and also did a lung biopsy. They said I had scar tissue on my lungs but never really told me how bad it really was. I was treated by this doctor for a number of years with steroids, antibiotics and breathing treatments. As the years went on I became sicker more and more often. I would have periods in which when I would cough I would black out for a few seconds, however the doctor didn’t make a big deal about it.

Fast forward to 2010, I started to have a lot of weakness and fatigue. One day while driving home I began to choke on some water and I blacked out while driving. When I came to I was making my way over to the next lane on the interstate. I know that the Lord was with me that day because otherwise I would not be talking to you today. I didn’t go to the doctor until about a week or so later when I began having severe headaches and weakness. I thought it maybe was a toothache so I made an appointment with the Dentist.

On the day of the appointment,  I had to climb a flight of stairs and by the time I got up those stairs I was gasping for breath. My dentist told me he would treat me for my toothache but he said I needed to see a doctor becayse he believe I had something very serious going on, pointing out that my hands were blue!

I called my Internal Medicine doctor and she ordered tests on my heart and blood tests. They would not let me go home because of the severity of my condition. They also conducted a CT scan of my lungs. The doctors came in and told me I had Pulmonary Hypertension.

They immediately started me on medicine (this was in July of 2010) and then in October of that same year I started to see a pulmonologist who put me on medicines related specifically to Pulmonary Hypertension. I am now also on oxygen pretty much all the time and I am doing Pulmonary Rehabilitation to help build up strength and endurance for my body.

It has been very encouraging and inspiring to do. I would like to say it has affected my life in many ways. It has affected my physical relationship with my husband who does not always understand and who is also dealing with his own health issues. It has affected my daughter and grandchildren as they were very worried about me. But, I am a fighter and refuse to give up because I know my Lord Jesus Christ is my healer, my rock and my shield and He will heal me.

I would like to say to all who are battling this disease or anyone who has a loved one who is battling it, to always support them, love them and pray for them and know that the Lord is with you and with them. I hope that I have been able to help or encourage someone today.

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Lee Ann’s Story

Posted on by CVCinfo

It was the third week that my ankles had been swollen consistently. That Wednesday, May 16, 2012 my mother and husband convinced me that an ER trip was necessary. The swelling had come and gone over the past 3 months. As a 41 year old mother of two teenagers, an asthmatic and someone who was overweight, I chalked the swelling up to being heavy. The shortness of breath had been there for years and again, I chalked that up to being heavy. Then I started to notice chest pain with the swelling and shortness of breath. That was when I realized that this might be a little more serious than I thought.

At the ER, the initial thought was that I had had a heart attack. Then blood clots then eventually, the final diagnosis… Pulmonary Hypertension. The language was not new to me or my family as my niece was born two short years ago with PH.

After a whirl of tests and a week-long hospital stay, I was discharged with little knowledge other than what I had learned from my niece. A Cardiac Cath was done and with this I was introduced to my now Pulmonary Doctor whom I love. Now the diagnosis was worse. Pulmonary Hypertension and Pulmonary Veno-Occlusive Disease.

I was told that after 5 years I would have a 50% chance of living without a double lung transplant. My world just stopped. Here we were a family of four making good money and living a great life. I had to quit my job and file for Social Security Disability. I had to deliver the news to my husband and boys that I was going to die.

Here it is four months later; I have had an open lung biopsy and have been taking medications that make my symptoms worse. The money situation is so bad, we’re making enough barely struggle by, but not enough to live. I was approved for disability but have a $5000 out of pocket deductible with my husband’s insurance. Not to mention copays and script costs. Disability wont start until December 26, 2012 and Medicare wont start until May 2014.

Stress is bad, I find myself crying all the time. I try to put on a happy face but it is so hard to ask for help, watch my husband come home tired from work and do housework and cook because I cannot.

Everyone keeps saying it will be ok, my question is ok for who? I have not yet learned to deal with my illness and pray that soon I will see that I can survive.

Thank you Caring Voice Coalition for all you do everyday.

Posted in About Us, Diseases, Events, Featured, How We Help, Media Center | Tagged , , , , , | 8 Comments ← Previous post