Sotatercept rescue therapy led to rapid clinical recovery, improving WHO functional class from IV to II and normalizing NT-proBNP levels in a 12-year-old girl with refractory end-stage IPAH.
Does sotatercept improve clinical and hemodynamic outcomes in pediatric patients with end-stage idiopathic pulmonary arterial hypertension refractory to conventional therapy?
Sotatercept may serve as an effective, life-saving rescue therapy for pediatric patients with severe, refractory idiopathic pulmonary arterial hypertension.
ABSTRACT Pediatric idiopathic pulmonary arterial hypertension (IPAH) refractory to maximal medical therapy is associated with high morbidity and mortality, and therapeutic options remain limited. We describe a 12‐year‐old girl with end‐stage IPAH who developed acute decompensated right heart failure despite triple combination therapy, including high‐dose prostacyclin. Escalation to intravenous epoprostenol failed to result in clinical or hemodynamic improvement. Following multidisciplinary discussion, ethical approval, and informed consent, sotatercept was initiated under compassionate use. Sotatercept therapy resulted in rapid and marked clinical recovery, including improvement in World Health Organization functional class (WHO FC) from IV to II, substantial increase in 6‐min walk distance, normalization of NT‐proBNP levels, and significant echocardiographic and invasive hemodynamic improvement. This case highlights the potential role of sotatercept as a rescue therapy in pediatric IPAH refractory to conventional treatment.
Pelczar‐Płachta et al. (2026) studied this question. Sotatercept rescue therapy led to rapid clinical recovery, improving WHO functional class from IV to II and normalizing NT-proBNP levels in a 12-year-old girl with refractory end-stage IPAH.