PAH is a condition that causes narrowing of blood vessels in the lungs, causing impaired blood flow throughout the lungs.

People living with PAH develop high blood pressure in the pulmonary arteries, which have to transport oxygen-starved blood from the heart to the lungs. According to the National Heart, Lung, and Blood Institute.

People with PAH find their heart working too hard to pump blood to the lungs. This can have a negative impact on your overall health. If left untreated, this condition can be fatal.

The condition is extremely rare. Only 500 to 1,000 people are diagnosed with PAH each year in the United States.

When asked to explain how PAH differs from other forms of pulmonary hypertension, Dr. Christine Hyland, a respiratory medicine specialist at the Cleveland Clinic who also participated in the sotatercept clinical trial, said that We explained that there are various overlapping features between pulmonary hypertension. A group that falls under the broader category of pulmonary hypertension. PAHs are classified as “Group 1”.

Dr. Hyland told Healthline that “Group 2” pulmonary hypertension is the most common type, known as “postcapillary,” where there is increased pressure on both the right and left sides of the heart. Ta. PAH, along with Group 3 and Group 4, is considered the precapillary form of pulmonary hypertension, which means “pressure on the left and right sides of the heart is normal.”

All groups have pulmonary vascular abnormalities, and PAH is a “diagnosis of exclusion”, meaning that doctors cancel the association with other groups (group 3 is the result of pulmonary disease such as chronic hypoxia). (Group 4 is blood-borne, etc.) As two examples, a blood clot that dislodges elsewhere in the body, lodges in the lungs, and then does not dissolve would lead to a PAH diagnosis.

“In PAH, the lesions are confined to the pulmonary arterioles and there are abnormalities in all layers of the blood vessels, resulting in vasculopathy, that is, thickening and narrowing of the blood vessels, which increases the resistance of the pulmonary vascular bed. , symptoms such as “pulmonary hypertension, or increased pressure in the lungs,” Hyland added. “This eventually culminates in the right side of the heart losing its ability to pump blood to the lungs.”

Dr. Hyland explains that one of the challenges that arises from this condition is that PAH can be misdiagnosed or underdiagnosed because the symptoms can mimic other conditions. Did.

She said diagnosis is often delayed for more than two years.

Hyland said that while living with PAH, patients often unconsciously “slow down” in response to symptoms. This means that doctors may miss this symptom entirely unless someone does something unusual, such as going on a difficult hike.

“If a patient is short of breath at rest, they have a very advanced disease,” Professor Hyland explained. “As is often the case, patients are usually diagnosed with asthma, COPD, left heart failure, obesity, and poor health.”

As the disease progresses, it puts more strain on the right side of the heart, and symptoms can include fatigue, chest pain, swelling of the stomach and legs, dizziness and fainting, he said. Hyland noted that syncope could be “a sign that the right side of the heart is really suffering.”

Kaufman said she thought her symptoms might be tied to anxiety, as she recalled the time just before her diagnosis, when her husband had just been deployed to the Middle East. She felt her shortness of breath and her heart pounded.

She noticed that just going to work seemed to use up a lot of oxygen, and she sometimes even had to sit in her car and take a break before heading to the office.

She received emergency treatment and was eventually admitted to the ER. Three days later she was diagnosed with PAH.

She calls it “fate” and “a series of lucky events,” given the reality that most people wait two to three years to receive a diagnosis.

Winrevair will be available at specialty pharmacies in the U.S. by the end of April, according to the release.

When asked how important it was for this new treatment to be available on the market, McLaughlin, the doctor who enrolled Kaufman in the trial, said it was exciting to have this new drug. All of the past 30 years have targeted “the same three pathways,” and “this is the first drug that actually has the potential to reverse remodel the disease,” she explained.

“I think this is a big step forward. It’s really exciting to start addressing the fundamental molecular questions of this disease and develop drugs that may have antiproliferative and reverse modeling capabilities,” McLaughlin added. Ta. “The breadth of positive results in the test was incredible. We’re very excited about what that actually shows, further progress, right?” We’re learning more and more, and it’s really great to see companies investing in our field and developing medicines with new mechanisms of action. ”

She said there are “many other clinical trials” currently underway and she expects more PAH drugs to become available in the near future.

Dr. Hyland pointed out that epoprostenol, the first FDA-approved PAH drug, was not available until 1996. That means there were countless others, like Kaufman’s brother BJ, living with the disease without a cure. Before the first drug was approved, Hyland said, the expected survival time for patients with PAH was less than three years.

“Since then, available treatments for PAH have focused on vasodilation, but most of these drugs have pleiotropic effects. “We’re focusing on either enhancing the nitric oxide pathway or blocking the endothelin pathway,” Hyland added. “Patients usually need to receive combination therapy. Patients often also need to be given diuretics and oxygen. Pulmonary rehabilitation can also be very beneficial.”

Professor Hyland said although these are encouraging times, survival rates for PAH are estimated to be only 86% at one year, 67% at three years and just 54% at five years, according to French registries. He added that . Hyland emphasized that this reality for people diagnosed with PAH means “additional and more effective treatments are needed.”

Kaufman said she has noticed a “night and day” difference in managing her symptoms since taking the medication. She remembers going to get her first shot in August 2021, but she just learned that what she received was the drug, not the placebo in her study. was.

“That night, my breathing immediately changed. It wasn’t dramatic, but my mood changed, and the next day I was sleeping about 22 hours out of 24,” she says. recalled. “With each shot, things were getting better and better.”

Kaufman said that after taking the medication, she was able to walk further and do more things. Her daughter plays travel softball and she was able to go with her daughter on the weekends and participate with other parents and families.

However, Kaufman emphasized that this is not a cure. She is also taking other medications that have side effects that cause extreme leg pain, which she said is causing more discomfort than PAH at this time.

“I don’t think I’m going to be here anymore. I’m looking forward to things,” Ms. Kaufman said of her past fears that she wouldn’t have many more years with her family. “My daughter is almost 16 and it’s just like that. I’m just so hopeful. I just feel better.”