New Drug Helps Patients With High Blood Pressure of the Lungs Breathe Easier

Key TakeawaysPulmonary arterial hypertension is a rare, incurable, progressive condition that causes fatigue and difficulty breathing. It’s a type of high blood pressure in the lungs.A recent clinical study showed that a new drug, sotatercept, can improve the quality of life for individuals with pulmonary arterial hypertension. The drug recently received FDA approval.Patients living with pulmonary arterial hypertension have treatment options, and they can find the support and resources needed to manage their condition.

Key Takeaways

Pulmonary arterial hypertension is a rare, incurable, progressive condition that causes fatigue and difficulty breathing. It’s a type of high blood pressure in the lungs.A recent clinical study showed that a new drug, sotatercept, can improve the quality of life for individuals with pulmonary arterial hypertension. The drug recently received FDA approval.Patients living with pulmonary arterial hypertension have treatment options, and they can find the support and resources needed to manage their condition.

Heather Kauffman, 49, noticed her health changing in the summer of 2017. She had frequent rapid heartbeats and shortness of breath for no apparent reason. Because she was only 43 with no prior health issues at the time, Kauffman attributed these symptoms to panic attacks.

“I can remember sitting in the recliner, short of breath with my heart racing and thinking, ‘I’m just stressed out, I guess,’” Kauffman told Verywell. “I didn’t do anything about it because I didn’t have time to go to the doctor.”

By December, Kauffman’s worsening symptoms prompted her to visit urgent care, where the staff sent her to the hospital. Aright heart catheterizationrevealed that she hadpulmonary arterial hypertension(PAH), a rare form ofpulmonary hypertension.

Pulmonary hypertensionis a general term for elevated blood pressure within the lungs. It can happen for many reasons, including heart disease involving the left side of the heart, chronic lung disease, blood clots in the lungs, or genetics.

In people with PAH, the walls of the arteries in the lungs become narrow and stiff. As a result, the right side of the heart, which pumps unoxygenated blood returning to the heart from the veins, must work harder to push blood to the lungs. Eventually, the extra stress causes the right side of the heart to enlarge and become less flexible, compromising its ability to move blood out of the heart and through the lungs, where it receives fresh oxygen before being recirculated to the body.

Only somewhere between 15 and 50 persons per million across the U.S. and Europe have PAH. The rarity means there is no cure.But with medications, patients can manage their symptoms and slow the disease’s progression.

Symptoms of Pulmonary Arterial HypertensionPAH can be difficult to diagnose because early signs mimic other diseases. Common symptoms include:Increasing shortness of breath, especially with exertionFatigueSwelling of the lower extremities, abdomen, and neckDizziness or faintingChest pain or palpitationsPersistent coughVoice hoarseness

Symptoms of Pulmonary Arterial Hypertension

PAH can be difficult to diagnose because early signs mimic other diseases. Common symptoms include:Increasing shortness of breath, especially with exertionFatigueSwelling of the lower extremities, abdomen, and neckDizziness or faintingChest pain or palpitationsPersistent coughVoice hoarseness

PAH can be difficult to diagnose because early signs mimic other diseases. Common symptoms include:

Exploring Every Treatment Option

After her diagnosis, Kauffman began taking the standard treatments for PAH, including oral medications and drugs that require a continuous infusion pump. She’d improve for a while, then decline again.

“I just kept continually getting worse, and I was already maxed out on the meds [I was taking],” she said.

Kauffman’s cardiologist, Vallerie McLaughlin, MD, a pulmonary hypertension specialist at the University of Michigan Health, said this is a common experience for PAH patients.

“PAH is often a progressive disease with a high mortality,” McLaughlin told Verywell. “Some patients improve on therapy and get back to work and a relatively normal lifestyle but will always be burdened with the side effects and expense of their therapies, as well as the risk of the disease progressing. Other patients do not respond as well and will have multiple hospitalizations and a limited life span. The goal is to diagnose patients early and escalate therapy to improve them to the low-risk status.”

McLaughlin encouraged Kauffman to join a clinical trial she was working on called STELLAR, which was investigating the use of a new injectable medication, sotatercept, for pulmonary arterial hypertension. McLaughlin said she’s seen incredible results from patients in phases 1 and 2 of the trial, and Kauffman agreed to enroll in August of 2021.

Even though the trial was a double-blind study and Kauffman had no way of knowing if she was receiving sotatercept or a placebo, she said she felt an immediate breakthrough in her condition.

Once the study was unblinded, Kauffman received confirmation that she had been receiving sotatercept.

Kauffmann continued to improve with each dose she received.

“I just kept getting better and better. I took on a new normal,” she said. “I was able to walk further. My daughter plays softball, and I could pull the wagon out to the field and carry my own chair. It doesn’t sound like much, but when you can’t do any of that, it’s huge to be able to do things for yourself again.”

The FDA’s approval of sotatercept was based on results from phase 3 of the STELLAR trial. Trial participants who received sotatercept could walk almost 41 meters (about 134 feet) further over six minutes after being on the drug for 24 weeks. They also noted improvements in eight of nine secondary endpoints, including functional class, cardiovascular function, and time to clinical worsening.

A New Type of Drug

“Sotatercept may have reverse remodeling effects, [which] is an important advancement in our field,” McLaughlin said.

Is Pulmonary Arterial Hypertension Genetic?McLaughlin said that in a small number of people (about 10–15%), PAH can have a genetic component. Researchers have recently discovered that thebone morphogenetic protein type 2 receptor(BMPR2) gene, which mainly regulates bone and cartilage growth, can be linked to PAH when mutations occur. The full extent of this process is unclear. However, researchers believe that BMPR2 mutations may promote cell overgrowth or cell death in the walls of the pulmonary arteries, accelerating the disease progression.While the news of her PAH diagnosis came as a shock to Kauffman, when her parents learned of her diagnosis, they told her that PAH had taken the life of her brother BJ, who died in 1982 at 5 years old. Kauffman said she was only 7 when her brother died, so she did not clearly remember what caused his death. “If you asked me, I would have said he died of a rare lung disease,” she said.

Is Pulmonary Arterial Hypertension Genetic?

McLaughlin said that in a small number of people (about 10–15%), PAH can have a genetic component. Researchers have recently discovered that thebone morphogenetic protein type 2 receptor(BMPR2) gene, which mainly regulates bone and cartilage growth, can be linked to PAH when mutations occur. The full extent of this process is unclear. However, researchers believe that BMPR2 mutations may promote cell overgrowth or cell death in the walls of the pulmonary arteries, accelerating the disease progression.While the news of her PAH diagnosis came as a shock to Kauffman, when her parents learned of her diagnosis, they told her that PAH had taken the life of her brother BJ, who died in 1982 at 5 years old. Kauffman said she was only 7 when her brother died, so she did not clearly remember what caused his death. “If you asked me, I would have said he died of a rare lung disease,” she said.

McLaughlin said that in a small number of people (about 10–15%), PAH can have a genetic component. Researchers have recently discovered that thebone morphogenetic protein type 2 receptor(BMPR2) gene, which mainly regulates bone and cartilage growth, can be linked to PAH when mutations occur. The full extent of this process is unclear. However, researchers believe that BMPR2 mutations may promote cell overgrowth or cell death in the walls of the pulmonary arteries, accelerating the disease progression.

While the news of her PAH diagnosis came as a shock to Kauffman, when her parents learned of her diagnosis, they told her that PAH had taken the life of her brother BJ, who died in 1982 at 5 years old. Kauffman said she was only 7 when her brother died, so she did not clearly remember what caused his death. “If you asked me, I would have said he died of a rare lung disease,” she said.

The most common potential side effects of Winrevair include a low platelet count, bleeding, and telangiectasis (red-colored veins appearing on the face).

Kauffman is still taking Winrevair. Her pulmonary artery pressures, a key indicator of PAH treatment success, are better on Winrevair than at any point since her diagnosis. She continues to take three other PAH medications she was on before starting Winrevair. However, she is on lower doses of those drugs than she was before starting Winrevair, which alleviates the side effects she experienced from those medications.

Kauffman advises those living with PAH to advocate for their own best treatment plan and to speak with other physicians if they feel their condition needs better management. Based on her success, she also encourages patients to look for clinical trials.

“A lot of us have lost hope because nothing has come along in a long time that changed much about treating PAH, and [Winrevair] is a huge change for our disease,” she said.

What This Means For YouWhile high blood pressure is common, high blood pressure in the lungs is not. If you or a loved one is living with this condition, consider asking a doctor about treatment innovations of clinical trials to help improve quality of life.

What This Means For You

While high blood pressure is common, high blood pressure in the lungs is not. If you or a loved one is living with this condition, consider asking a doctor about treatment innovations of clinical trials to help improve quality of life.

6 SourcesVerywell Health uses only high-quality sources, including peer-reviewed studies, to support the facts within our articles. Read oureditorial processto learn more about how we fact-check and keep our content accurate, reliable, and trustworthy.Pulmonary Hypertension Association.Types of pulmonary hypertension.Pulmonary Hypertension Association.About pulmonary hypertension: the five groups.Levine DJ.Pulmonary arterial hypertension: updates in epidemiology and evaluation of patients.Am J Manag Care. 2021;27(Suppl 3):S35-S41. doi:10.37765/ajmc.2021.88609American Lung Association.Pulmonary arterial hypertension symptoms and diagnosis.National Library of Medicine: DailyMed.Winrevair—sotatercept-csrk kit[drug label].Hoeper MM, Badesch DB, Ghofrani HA, et al.Phase 3 trial of sotatercept for treatment of pulmonary arterial hypertension.N Engl J Med. 2023;388(16):1478-1490. doi:10.1056/NEJMoa2213558

6 Sources

Verywell Health uses only high-quality sources, including peer-reviewed studies, to support the facts within our articles. Read oureditorial processto learn more about how we fact-check and keep our content accurate, reliable, and trustworthy.Pulmonary Hypertension Association.Types of pulmonary hypertension.Pulmonary Hypertension Association.About pulmonary hypertension: the five groups.Levine DJ.Pulmonary arterial hypertension: updates in epidemiology and evaluation of patients.Am J Manag Care. 2021;27(Suppl 3):S35-S41. doi:10.37765/ajmc.2021.88609American Lung Association.Pulmonary arterial hypertension symptoms and diagnosis.National Library of Medicine: DailyMed.Winrevair—sotatercept-csrk kit[drug label].Hoeper MM, Badesch DB, Ghofrani HA, et al.Phase 3 trial of sotatercept for treatment of pulmonary arterial hypertension.N Engl J Med. 2023;388(16):1478-1490. doi:10.1056/NEJMoa2213558

Verywell Health uses only high-quality sources, including peer-reviewed studies, to support the facts within our articles. Read oureditorial processto learn more about how we fact-check and keep our content accurate, reliable, and trustworthy.

Pulmonary Hypertension Association.Types of pulmonary hypertension.Pulmonary Hypertension Association.About pulmonary hypertension: the five groups.Levine DJ.Pulmonary arterial hypertension: updates in epidemiology and evaluation of patients.Am J Manag Care. 2021;27(Suppl 3):S35-S41. doi:10.37765/ajmc.2021.88609American Lung Association.Pulmonary arterial hypertension symptoms and diagnosis.National Library of Medicine: DailyMed.Winrevair—sotatercept-csrk kit[drug label].Hoeper MM, Badesch DB, Ghofrani HA, et al.Phase 3 trial of sotatercept for treatment of pulmonary arterial hypertension.N Engl J Med. 2023;388(16):1478-1490. doi:10.1056/NEJMoa2213558

Pulmonary Hypertension Association.Types of pulmonary hypertension.

Pulmonary Hypertension Association.About pulmonary hypertension: the five groups.

Levine DJ.Pulmonary arterial hypertension: updates in epidemiology and evaluation of patients.Am J Manag Care. 2021;27(Suppl 3):S35-S41. doi:10.37765/ajmc.2021.88609

American Lung Association.Pulmonary arterial hypertension symptoms and diagnosis.

National Library of Medicine: DailyMed.Winrevair—sotatercept-csrk kit[drug label].

Hoeper MM, Badesch DB, Ghofrani HA, et al.Phase 3 trial of sotatercept for treatment of pulmonary arterial hypertension.N Engl J Med. 2023;388(16):1478-1490. doi:10.1056/NEJMoa2213558

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