Abstract

THE EFFECTIVENESS AND VALUE OF SOTATERCEPT FOR PULMONARY ARTERIAL HYPERTENSION

B. Aswini Bai*

Abstract

Background: Pulmonary arterial hypertension is characterized by pulmonary vascular remodeling, cellular proliferation, and poor longterm outcomes. Dysfunctional bone morphogenetic protein pathway signaling is associated with both hereditary and idiopathic subtypes. Sotatercept, a novel fusion protein, binds activins and growth differentiation factors in the attempt to restore balance between growth-promoting and growth-inhibiting signaling pathways. Methods: In this 14-week multicenter trial, we randomly assigned 100 adults who were receiving background therapy for pulmonary arterial hypertension to receive subcutaneous sotatercept at a dose of 0.3 mg per kilogram of body weight every 3 weeks or 0.7 mg per kilogram every 3 weeks or placebo. The primary end point was the change from baseline to week 24 in pulmonary vascular resistance. Results: Baseline characteristics were similar among the three groups. The least-squares mean difference between the sotatercept 0.3-mg group and the placebo group in the change from baseline to week 14 in pulmonary vascular resistance was −145.8 dyn·sec·cm−5 (95% confidence interval [CI], −241.0 to −50.6; P=0.003). The least-squares mean difference between the sotatercept 0.7-mg group and the placebo group was −239.5 dyn·sec·cm−5 (95% CI, −329.3 to −149.7; P

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