Why the study?
Sotatercept has shown efficacy in adults with PAH, but pediatric data remain limited to ongoing clinical trials.
Does off-label sotatercept improve hemodynamic parameters in pediatric patients with advanced pulmonary arterial hypertension refractory to triple therapy?
Comparison
Off-label sotatercept
Design
Single-center retrospective analysis
Follow-up
Approximately 24 weeks
Key result
Off-label sotatercept in 7 pediatric patients with advanced PAH refractory to triple therapy was associated with reductions in pulmonary vascular resistance index and mean pulmonary artery pressure.
Authors
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Should not yet change practice in pediatric PAH; leaves open efficacy and safety pending prospective trials.
Observational (n=7)
No
Does off-label sotatercept improve hemodynamic parameters in pediatric patients with advanced pulmonary arterial hypertension refractory to triple therapy?
Off-label sotatercept use in pediatric patients with advanced, refractory pulmonary arterial hypertension is associated with favorable hemodynamic improvements at 24 weeks, supporting the biologic plausibility for its use in this population.
Rao et al. (2026) conducted an observational in Pediatric Pulmonary Arterial Hypertension (n=7). Sotatercept was evaluated on Hemodynamic parameters including pulmonary vascular resistance index (PVRI), mean pulmonary artery pressure (mPAP), and pulmonary capillary wedge pressure (PCWP). Off-label sotatercept in 7 pediatric patients with advanced PAH refractory to triple therapy was associated with reductions in pulmonary vascular resistance index and mean pulmonary artery pressure.