New therapy shows dramatic survival benefits in advanced pulmonary arterial hypertension

New therapy shows dramatic survival benefits in advanced pulmonary arterial hypertension

A relatively new therapy to treat pulmonary arterial hypertension in patients with mild to moderate disease has been found to be effective in preventing death in patients with advanced disease. The results were published on Wednesday, May 28thThe New England Journal of Medicineand could have “transformative effects” for patients, according to an editorial accompanying the study by Bradley Maron, MD, professor of medicine and director of the Hypertension Program at the University of Maryland School of Medicine.

When the U.S. Food and Drug Administration named the first-in-its-class drug last year, it was indicated only for people with mild pulmonary arterial hypertension to increase exercise capacity and prevent clinical worsening of the lung disease, which is rare but progressive and often leads to premature death. About 1,000 Americans are diagnosed with the condition each year, and women under 60 are at higher risk. The condition, caused by narrowing of small arteries throughout the lungs, triggers the heart to work harder and eventually lose its ability to pump blood effectively.

The clinical trial, called Zenith, was led by researchers in France and conducted at multiple clinical sites in the U.S. and internationally. There were 172 patients with advanced pulmonary arterial hypertension who were randomly assigned to receive an injection of sotatercept along with their usual treatments or to receive a placebo injection along with their usual treatments.

“The authors observed a 76 percent lower risk of a primary endpoint event [death from any cause, lung transplantation, or hospitalization] with sotatercept than with a placebo – an astonishing effect by a standard of uniquely relevant pulmonary arterial hypertension, since previous trials have typically shown comparatively modest results with weaker endpoints,” wrote Dr. Maron, who was also director of the University of Maryland Institute for Health Computing.

The trial was stopped early after it became clear that the sotatercept group had significant advantages over those taking a placebo: 50 percent of the placebo group were hospitalized during the study compared to 9 percent of the sotatercept group. Death occurred in 15 percent of those on a placebo, compared with 8 percent of those on sotatercept.

Vascular malformations and bleeding events occurred in some patients taking sotatercept, but this did not cause them to stop taking the drug. According to Dr. However, according to Maron, a better understanding of how these side effects might relate to patients' adherence to sotatercept in actual clinical practice is needed.

“Findings from the Zenith study,” he wrote, “provide an essential measure of optimism for patients with pulmonary arterial hypertension with limited or no option.”

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