A new real-world study suggests sotatercept could be a meaningful option for kids with pulmonary hypertension. Among 66 pediatric patients, more than half showed improved heart-lung function, and none got worse. The drug was generally well tolerated, though half of patients experienced some adverse events, most commonly nosebleeds.
A retrospective, multi-center registry study published in CHEST offers the largest real-world evidence yet supporting sotatercept as an add-on therapy for pediatric pulmonary hypertension (PH). Researchers analyzed data from 66 patients aged 21 and under who received sotatercept alongside their existing treatment between March 2024 and December 2025. The results were encouraging: over half of patients with repeat assessments improved their WHO functional class, and none worsened.
Beyond functional status, patients walked farther, had lower biomarker levels indicating reduced cardiac stress, and showed measurable improvements in pulmonary artery pressure and right ventricular function on imaging. The drug was generally well tolerated, though adverse events occurred in half of patients, with nosebleeds being the most common complaint.
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Why it matters: Pulmonary hypertension in children is a serious, life-threatening condition with limited treatment options. This study provides the strongest real-world evidence to date that sotatercept may meaningfully improve outcomes in this vulnerable population, potentially expanding its role beyond adult care.
