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Off-Label Sotatercept Use in Pediatric Pulmonary Arterial Hypertension: A Single-Center Retrospective Hemodynamic

Dhruv Rao1, Shylah Haldeman2, Jeanne Carroll3

  • 1University of Arizona, Tucson, AZ, USA.

Pediatric Cardiology
|June 26, 2026
PubMed

Insights

Sotatercept showed promising results in a small group of pediatric patients with severe pulmonary arterial hypertension (PAH) who did not respond to other treatments. Further studies are needed to confirm these findings in children with PAH.

Area of Science:

  • Cardiology
  • Pulmonology
  • Pharmacology

Background:

  • Pulmonary arterial hypertension (PAH) significantly impacts pediatric health, with limited treatment options for severe cases.
  • Sotatercept, an activin signaling inhibitor, has shown efficacy in adult PAH, but pediatric data are scarce.
  • Current pediatric PAH management often involves maximal triple therapy, yet many patients remain symptomatic or hemodynamically unstable.

Purpose of the Study:

  • To evaluate the safety and efficacy of off-label sotatercept in pediatric patients with WHO Group 1 or Group 3 PAH.
  • To assess hemodynamic changes in pediatric PAH patients refractory to maximal background therapy after initiating sotatercept.
  • To explore the potential of sotatercept as a disease-modifying therapy in a pediatric population.

Main Methods:

  • Retrospective analysis of seven pediatric patients (median age 14) with advanced PAH treated with off-label sotatercept.
  • Patients received maximal background triple therapy prior to sotatercept initiation.
  • Right heart catheterization was performed before treatment and at ~24 weeks post-initiation to analyze hemodynamic parameters.

Main Results:

  • The majority of pediatric patients experienced reductions in pulmonary vascular resistance index (PVRI) and mean pulmonary artery pressure (mPAP).
  • Pulmonary capillary wedge pressure (PCWP) remained stable across all patients.
  • Observed hemodynamic improvements showed heterogeneity in magnitude among responders.

Conclusions:

  • Off-label sotatercept demonstrated favorable hemodynamic trends in a small cohort of pediatric PAH patients with advanced disease.
  • Findings support the biologic plausibility of sotatercept's efficacy in pediatric PAH, consistent with adult trial data.
  • Urgent need for prospective clinical trials to validate sotatercept's role in pediatric PAH treatment.

Keywords:
HemodynamicsOff-label therapyPediatric pulmonary arterial hypertensionPulmonary vascular resistanceSotatercept

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