A recent study, published in the New England Journal of Medicine, looked at the effects of sotatercept for pulmonary arterial hypertension (PAH) within the first year after diagnosis.
PAH affects the heart and lungs. It is characterized by abnormally high blood pressure in the pulmonary artery. Symptoms include shortness of breath during exercise and fainting spells. The symptoms tend to get worse over time and may include dizziness, swelling of the ankles or legs, chest pain, and a racing pulse. Some cases of PAH are due to genetic changes in the BMPR2 gene. Most cases of PAH occur in individuals with no family history of the disorder. PAH can also occur secondary to underlying conditions such as connective tissue diseases, HIV infection, chronic hemolytic anemia, and congenital heart disease, or can be induced by certain drugs and toxins.
Sotatercept is an activin-signaling inhibitor that reduces morbidity and mortality among patients with long-standing pulmonary arterial hypertension. It is currently approved by the U.S. Food and Drug Administration (FDA) for the treatment of adults with PAH (PAH, WHO Group 1).
The HYPERION (NCT04811092) clinical trial was a phase 3 randomized, double-blind, placebo-controlled, study evaluating sotatercept when added to background PAH therapy in newly diagnosed intermediate- or high-risk PAH participants. This trial enrolled adult patients with WHO functional class II or III PAH who had received their diagnosis less than one year earlier, had an intermediate or high risk of death, and were receiving double or triple background therapy.
Primary endpoints included clinical worsening, a composite of death from any cause, unplanned hospitalization lasting at least 24 hours for worsening of pulmonary arterial hypertension, atrial septostomy, lung transplantation, or deterioration in performance in exercise testing due to pulmonary arterial hypertension, assessed in a time-to-first-event analysis.
The trial was stopped early due to loss of clinical balance after the reporting of positive results from previous sotatercept trials. A total of 320 patients were included and the median duration of follow-up was 13.2 months. At least one primary endpoint event occurred in 10.6% of patients in the sotatercept group and 36.9% in the placebo group.
Deterioration in performance in exercise testing due to PAH occurred in 5% of patients in the sotatercept group and in 28.8% of patients in the placebo group. Additionally, unplanned hospitalization for worsening of pulmonary arterial hypertension occurred in 1.9% and 8.8%, respectively, and death from any cause occurred in 4.4% and 3.8%. No cases of atrial septostomy or lung transplantation occurred. The most common adverse events with sotatercept were epistaxis and telangiectasia.
Overall, among adults with PAH who had received the diagnosis less than 1 year earlier, the addition of sotatercept to background therapy resulted in a lower risk of clinical worsening than placebo.
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