Waukeshahealthinsurance.com-
–
When doctors told Katrina Barry she had a rare and serious condition called pulmonary arterial hypertension, or PAH, they warned her not to Google it.
Come on, she thought; Did you want a young woman who was bound for graduate school, survived a heart attack on a transatlantic flight, and now has heart surgery, to avoid seeing the situation that is trying to kill her?
There was some cool information waiting for her on the internet. PAH affects 500 to 1,000 Americans each year, most often women between the ages of 30 and 60. according to American Lung Association.
Barry, then 25, learned from her reading. That she is She had two to five years to live, depending on the severity of her illness.
Then her medical team gave her a lifeline: a first-of-its-kind experimental drug called sotarecept, which blocks a growth factor produced in excess in people with PAH, potentially altering the biology of the disease.
She is enrolled in a study to test the drug, which is injected under the skin every three weeks.
She began treatment in March 2020 at the start of the Covid-19 pandemic. After diligently traveling to the hospital wearing an N95 mask and two layers of clothing, Barry wrapped herself up and said she wouldn't catch Covid on the road. .
“I had to go to the hospital because I was dying if I didn't receive this medicine,” she said. “This was my only chance at salvation.”
Tuesday, the US Food and Drug Administration approved Barry says the drug, which she took for four years, saved her life and allowed her to resume many of the activities she enjoyed before her diagnosis.
Merck, which manufactured the drug, said it would sell it under the brand name Winrevair.
Barry, now 29, says she was not compensated for telling her story, although she is a Merck patient advisor.
It is Winrevair A biologic drug designed to capture and trap proteins called activins that are overproduced in PAH. These proteins make the walls of a person's blood vessels Keep growing and growing.
As the blood vessels narrow; The heart is forced To work harder to lift blood to the lungs. This stress eventually damages the heart, causing it to fail.
Without treatment, people with PAH usually live two to three years after their diagnosis. Treatment with a combination of drugs that dilate or relax blood vessels may improve this outlook, but they are not a cure.
“This drug has a very different mechanism of action, and it can restore some of the growth in the very small pulmonary arterioles where the disease starts,” said Dr. Valerie McLaughlin, a cardiologist at the University of Michigan. It helped to study sotatercept.
“This can attack the disease from a different angle and allow for reversible improvement,” she said, meaning patients can improve over time while on medication.
Dr. Panagis Galiasatos, MD, a pulmonologist and critical care physician at Johns Hopkins Bayview Medical Center and a spokesperson for the American Lung Association, said Winrevair is the first new drug to come along for patients with PAH in a long time.
In animal studies conducted before human trials, the drug appears to do more than treat symptoms. It appears to be able to stop the thickening of blood vessels and possibly prolong the lives of patients, but the benefits have not been proven in humans.
Instead, Galiatsatos said, the FDA approved the drug based on other signs of effectiveness, including whether patients could walk for six minutes before starting the drug, a clinical measure called the six-minute walk test.
In a study involving 323 people who took sotatercept or a placebo for six months, those who took the drug were able to walk 34 meters – half a city block – in six minutes longer than they could before starting treatment. In the placebo group, they only walked about 1 meter longer than they could when they started the study. Both groups were taking regular medications that help relax blood vessels to improve blood circulation.
At the end of the study, nine participants, or about 5%, died or their disease worsened in the sotasterep group, while 42 patients, or about 26%, died or worsened in the placebo group.
The FDA was willing to work quickly to approve the drug because of the disease Galiatsatos He said, it's a “ticking time bomb” for people. You don't have time to wait for long studies.
The study that led to the drug's approval It has been published Last year in the New England Journal of Medicine.
“Because so many of us enjoy this kind of medicine, we can offer our patients something that we believe is better than the current standard of care,” Galissatos said. Neither he nor the American Lung Association has any financial interest in the drug.
But Galiatsatos says as promising as the drug looks, there are still many unknowns, including whether the drug will benefit all PAH patients equally. Although the majority of infected people were African-American, only seven people in the clinical trial — representing 2% of participants — were black, he said.
With any new drug, “I try to hold my breath,” Galiatsatos said, adding, “Waiting for the real-world data to come out to see how well this works in different patients.”
In the year In July 2019, Barry rented an apartment in Greece for the summer, where she was excited to volunteer at a children's hospital. But while she was abroad, she was asking doctors for more information about shortness of breath More than two years suddenly worsened.
One morning she woke up out of breath and realized that the situation was dire. She knew she had to return home to Boston for help. She called her mother and arranged for her to fly back to the States.
She landed at the airport and He went straight to the hospital. There, doctors performed a CT scan and found dozens of what looked like blood clots in tiny vessels in the lungs. Her heart was severely damaged by a heart attack.
“The extent of the heart damage was not determined until the cardiologist performed the autopsy,” Barry said. “And every doctor on the way home said I shouldn't have survived that plane.”
Gentle surgery Gentle surgery was performed – the surgeons opened her chest, but her veins appeared to be clots and collapsed in several areas. The blood pressure in her heart was sky high, and that's when they finally found out what they were dealing with with PAH, she said.
Delayed diagnosis is common because the disease is rare and often affects young women, Barry said.
When doctors warmed up Barry's body to pull her out of surgery, her heart failed to restart. She spent a week in an induced coma on a heart-lung bypass machine. The medical team told her parents that she might need an immediate heart-lung transplant.
However, after a few days on a ventilator, her body began to recover and doctors were able to bring her out of the coma.
When she woke up, she learned of her diagnosis of pulmonary hypertension.
At 25, facing a shorter life than expected, Barry saw everything differently.
“Why should I save if I'm not going to retire?” she wondered. “Let me enjoy the life I have now.”
But she didn't even have much life left to enjoy.
She is attached to oxygen tanks and is forever questioning how much she needs for the times she wants to leave the house. There was also a large new medication pump that lived in a fanny pack that she could never take out, even to shower.
She receives a continuous flow of medication through a catheter inserted into a large vein in her body so that the pump can help keep her blood vessels open. If something happened to the pump, she had three hours to get to the nearest hospital for help, she was told.
Being alive was good and all, Barry thought, but what about getting dressed? How about going for a swim?
“The list includes everything I can't do,” she said.
Barry was diagnosed shortly after her surgery in October 2019, and in the spring of 2020 she had the opportunity to try sotarecept. The improvements weren't immediate, but after a few months, she couldn't even fold her laundry. Being able to do a little more. She was able to go for short walks and kayak a bit.
A year later, Barry said She was able to wean off her continuous oxygen therapy and use it only at night. She was finally able to stop using at night.
After 18 months, doctors were able to remove her central line and the huge, ever-present medical pump. Change from IV versions of her vasodilating drugs to pill forms.
“For me, that was life-changing,” says Barry.
Much improved, she was able to return to Greece in 2022 and scale the volcano. She went skiing last month.
Barry still has PAH. Her lungs burn and she becomes short of breath if she exerts herself too much.
“But it got better over time,” Barry said.
She enrolled in a master's degree program in public health, and she was able to wear a dress to her brother's wedding. She did not need the heart-lung transplant that doctors had thought.
“Honestly, this is a miracle drug,” he says of Sotarecept. “It sounds weird to say, but there's a lot of positives, but not a lot of negatives.”
She knows of at least one person in the PAH community who experienced bleeding while on the drug and had to stop taking it.
Among the risks are bleeding and dizziness
In clinical trials, people on sotarecept had more nosebleeds due to broken blood vessels in their noses and developed more spider veins under their skin. Experts say that the drug can stimulate the growth of new blood vessels.
Other side effects include dizziness and increased hemoglobin, molecule…