TOPLINE

Sotatercept was associated with an improved World Health Organization (WHO) functional class, longer walk distances, and lower pulmonary artery pressure in children with pulmonary hypertension. It also showed a satisfactory safety profile and was generally well tolerated.

METHODOLOGY

• Researchers retrospectively analyzed registry data from multiple centers to assess the real-world efficacy and safety of sotatercept as add-on therapy in children with pulmonary hypertension.
• They included 66 patients aged 21 years or younger (median age at drug initiation, 15.3 years; 60% female) who received sotatercept as part of routine clinical care between March 2024 and December 2025. 
• Treatment began at a median of 0.3 mg/kg, and 86% of patients had their dose increased to 0.7 mg/kg for the second dose; the median treatment duration was 323 days.
• Researchers collected demographic data and information on WHO functional class, six-minute walk distance, laboratory values such as the levels of B-type natriuretic peptide and N-terminal Pro-BNP, echocardiographic metrics, and cardiac catheterization hemodynamics, when available, before and after sotatercept initiation.
• Adverse events, including nosebleeds, nasal congestion, and gastrointestinal bleeding, were also documented.

TAKEAWAY

• Among 57 patients with repeat assessments, 51% had an improvement in WHO functional class, and none had a worsening after a median of 150 days of sotatercept treatment. The odds of improvement were higher at the latest follow-up than before treatment (odds ratio, 15.4; 95% CI, 5.1-46.6).
• Six-minute walk distance increased by a median of 31 m (P = .0003). The levels of N-terminal pro-B-type natriuretic peptide fell by a median of 378 pg/mL (P < .0001).
• Among patients who underwent repeat cardiac catheterization after a median of 254 days, mean pulmonary artery pressure and indexed pulmonary vascular resistance improved significantly (P < .0001 and P = .0011, respectively). Echocardiograms also showed improvements in the measures of right ventricular pressure: the ratio of tricuspid regurgitation jet velocity to systolic blood pressure and the systolic left ventricular eccentricity index (P < .0001 for both).
• Adverse events occurred in 50% of patients, with bleeding, particularly nosebleeds, among the most common events. Five patients discontinued sotatercept; no patients died, underwent lung transplantation, or were hospitalized for clinical worsening.

IN PRACTICE

"These findings provide the largest supportive evidence base to date for sotatercept use in pediatric PH [pulmonary hypertension], encompassing patients with diverse etiologies and severity, and highlight its potential role in treatment of pulmonary vascular disease in this population," the authors wrote.

SOURCE

The study was led by Paul J. Critser, The Heart Institute, Cincinnati Children's Hospital Medical Center, University of Cincinnati College of Medicine, Cincinnati. It was published online on October 5, 2026, in CHEST.

LIMITATIONS

Because the study was retrospective, the researchers could not confirm that adding sotatercept to background therapy caused the improvements. As the data came from routine care, follow-up testing varied, and blood marker results were available for only a subset of patients.

DISCLOSURES

The study received no financial support. Some authors disclosed receiving grants from or having consulting relationships with various pharmaceutical companies.

This article was created using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication.