The PHA 2023 Southern California O2breathe Walk.
The Pulmonary Hypertension Association (PHA) 2023 Southern California O2breathe Walk in Long Beach. (Photo courtesy of PHA)

On May 5, 1981, a young girl in Spain died of pulmonary hypertension (PH)—the first victim of a toxic rapeseed oil scandal that eventually killed over 300 people and sickened about 20,000.

More than 30 years later, scientists and patient advocacy groups meeting in Madrid established May 5 as World PH Day to mark that sad anniversary.

This year, as COVID-19 recedes from the world’s headlines, patient advocacy groups in the United States, Canada, Europe, and elsewhere are marking World Pulmonary Hypertension Day 2023 under the banner “United to Cure PH.”

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PHA president and CEO Matt Granato. (Photo courtesy of PHA)

“This slogan recognizes the global strides that have improved the lives of those affected by PH,” said Matt Granato, president and CEO of the Pulmonary Hypertension Association (PHA). “Although the disease is caused by many, many different things and factors around the world, it affects everyone in very, very similar ways.”

Granato, who assumed leadership of the Washington, DC-based group in January 2021, was born and raised in Argentina. Only the third president in the PHA’s 32-year history, Granato, 47, has more than 2 decades of experience working for nonprofit and healthcare associations. Before coming to PHA, he led the Society for Maternal-Fetal Medicine, which focuses on high-risk pregnancies.

A Kitchen Table That Made PH History

The PHA was founded in 1991 around a kitchen table in Florida by 3 women who had PH and a nurse whose sister was among the founders. That table today occupies a proud place in the organization’s Washington headquarters.

Awards and plaques decorate the original dining table and chairs at which three Florida women founded the PHA in 1991; the furniture now resides at PHA headquarters in Washington, DC. (Photo by Larry Luxner)

“One of the things that attracted me to PHA was that, in addition to working with healthcare professionals that were making improvements in the lives of patients, I got to work directly with patients and caregivers,” said Granato, whose organization employs 33 people and has an annual budget of $9 million.

“We have a very extensive network of peer support through online or in‑person support groups, email, and telephone support lines,” he added.

“Our patient education is second to none. We provide webinars, digital and print publications, and video. We have networking through in‑person events, such as our biannual international PH conference, scientific sessions, and free regional education workshops.”

A Serious Subtype With High Mortality Rates

The World Health Organization has classified PH into 5 main types and at least a dozen subtypes. The most serious form is pulmonary arterial hypertension (PAH).

PAH is characterized by the narrowing and thickening of the pulmonary arteries, which increases blood pressure in the pulmonary arteries and the right ventricle of the heart. Over time, this can lead to right ventricular failure and death. Without treatment, patients generally live no more than 3 years after diagnosis. At present, the 5-year mortality rate for people with PAH is 43%.

In the organization’s recent survey of patients with PAH, 86% reported shortness of breath, 27% fatigue, 22% chest pain, 21% swelling, 15% fainting or light-headedness, and 13% palpitations.

A memorial quilt honoring patients who died of pulmonary hypertension decorates PHA headquarters in Washington, DC. (Photo by Larry Luxner)

According to an article published in the European Respiratory Review in 2019, the incidence of PAH ranges from 2.0 per million in the United States to 7.6 cases per million in Scotland. Women are 3-5 times more likely to develop the disease than men.

PAH is itself divided into subtypes, including heritable PAH, which is linked to specific genes.

“It can develop in relation to congenital heart disease, liver disease, or in some cases, HIV or some other connective tissue disease, such as scleroderma or lupus. It could also be as a result of stimulant use such as methamphetamine and certain diet pills that were popular in the ‘80s and ‘90s,” Granato said. “There’s also idiopathic PAH, which means it’s not clear what’s causing it.”

PHA Urges Support for Affordable Care, ‘Step Therapy’ Bills

The PHA plans to host virtual legislative visits throughout May to raise awareness and advocate on behalf of those with the disease. On Capitol Hill, the organization will ask lawmakers to protect access to affordable care by supporting the HELP Copays Act as well as reforms for supplemental oxygen access.

The PHA is also lobbying on behalf of the Safe Step Act of 2023, a bipartisan bill that places reasonable limits on the use of “step therapy,” which requires patients to try and fail on a lower-cost drug before being allowed to access the drug originally prescribed by their physicians.

The organization encourages patients and caregivers to help raise awareness on social media by using the #WorldPHDay2023 hashtag. It will also host 4 in-person events during the month of World PH Day:

The PHA’s 2023 Southern California O2breathe Walk in Long Beach. (Photo courtesy of PHA)
  • PH Community Workshop, May 6, in San Diego, California
  • Detroit O2breathe Walk, May 6, in Shelby Township, Michigan
  • Greater Washington DC O2breathe Walk, May 20, in Alexandria, Virginia
  • DeWitt Take a Breath for PH, May 20, in DeWitt, Michigan.

“We’ve come a long, long way. At the beginning, 30 years ago, when PHA was founded, patients would live 2-5 years at most. Now, we see people living 10, 15, 20, 30 years with the disease. It’s become more of a chronic illness,” Granato said. “However, you could still live with it for 30 years, but not enjoy your life. Your quality of life would not be there, especially people who require multiple therapies or oxygen.”

During the COVID-19 pandemic, many PAH patients had difficulty getting routine tests to monitor their condition. According to a study published in Respiratory Medicine, around half of research activities around PAH came to a halt, and 81% of patients surveyed said the pandemic had impeded research on their disease.

Janssen, Merck Seek FDA Approval for New PAH Therapies

Nevertheless, the estimated 30,000 Americans who have PAH can access 14 therapies approved by the US Food and Drug Administration (FDA). That compares to zero in 1991, when the PHA was established.

Two more PAH drugs could be headed to the market. Janssen Pharmaceuticals, a unit of Johnson & Johnson, announced on March 6, 2023, that an investigational once-a-day tablet combining 10 mg of macitentan and 40 mg of tadalafil “significantly improved pulmonary hemodynamics” vs macitentan or tadalafil alone in patients with PAH. The study involved 187 adults with PAH from across 148 sites in 19 countries.

Separately, Merck on October 10, 2022, announced positive top-line results from its “pivotal phase 3 STELLAR trial which evaluated the safety and efficacy of sotatercept, an investigational activin receptor type IIA-Fc (ActRIIA-Fc) fusion protein being evaluated as an add-on to stable background therapy to treat PAH.”

“The trial met its primary efficacy outcome measure, demonstrating a statistically significant and clinically meaningful improvement in 6-minute walk distance” from baseline at 24 weeks, according to Merck.

“This particular trial we’re excited about, because it’s a completely different pathway to treating PH. Usually, we’re treating the symptoms, but this would actually have the potential to reverse the damage caused by PH,” Granato said. “We’re looking forward to seeing the final results of this trial and FDA approval, and seeing how the drug performs in the market.”

PHA Europe Rallies Help for Ukrainian PAH Patients

On the other side of the Atlantic, PHA Europe is pursuing much the same goals as the US group. The nonprofit was launched in 2003 by Austrian businessman Gerald Fischer, whose daughter, Maleen, had been diagnosed with PAH 5 years earlier. At that time, not a single clinic or hospital in Europe was set up to treat children with the disease.

Ukrainians with pulmonary hypertension
Ukrainians with pulmonary hypertension wear T-shirts marking World PH Day at a March 2023 gathering in Lviv, Ukraine. (Photo courtesy of the Pulmonary Hypertension Ukraine Rare Disease Association (PHURDA))

“Her doctors told me that Maleen wouldn’t live to the age of 10, but she’s been on intravenous treatment via a permanent cannula for 24 years,” he said proudly. “She has a tube in her chest, and on May 24 she’s graduating from Harvard.”

Vienna-based PHA Europe operates on a €600,000 annual budget and encompasses 33 national patient associations in 27 countries. Maleen, now 28, is the charity’s social media manager.   

“General practitioners need to keep in mind that PH is not asthma or epilepsy,” Fischer said. “Through World PH Day, our awareness campaign targets the authorities and payers, so that patients will get access to very expensive treatments.”

These days, one of PHA Europe’s priorities is helping PH patients in war-torn Ukraine through the organization’s Ukrainian affiliates in Kyiv and Lviv.

Iryna Zhyvylo, MD (Photo courtesy of PHURDA)

Iryna Zhyvylo, MD, who fled her country following Russia’s February 2022 invasion, cared for about 400 of Ukraine’s 679 PH patients at Kyiv’s MD Strazhesko Institute of Cardiology. She said Poland has given Ukraine extensive humanitarian assistance, but that it’s not enough, given the country’s sporadic electricity supply and severely damaged infrastructure.

The lack of battery-powered oxygen concentrators isn’t the only problem. In Europe, 10 therapies are available for PAH patients, but in Ukraine, there’s only sildenafil, bosentan, ambrisentan, and iloprost.

“Pulmonary hypertension patients in America or Ukraine are equally short of air because their lungs are damaged. But the possibility of survival for these patients is very different,” said Oksana Skåra, who founded the Pulmonary Hypertension Ukraine Rare Disease Association (PHURDA) in 2013 and now lives in Norway. “Patients often start expensive treatments in Europe, but when they return to Ukraine, they do not have the opportunity to continue this therapy.”

Dr. Zhyvylo added that many of her former PH patients who relocated to Western Europe currently receive macitentan, selexipag, and riociguat; those in Poland may also access Revatio® (sildenafil). Ukraine’s Ministry of Health is exploring options for these patients, she said, warning that “otherwise, when they return, they could die.”