Lifesaving Compassionate-Use of Sotatercept in a 12-Year-old With Idiopathic Pulmonary Arterial Hypertension
Weronika Pelczar-Płachta, Rafał Surmacz, Waldemar Bobkowski
https://doi.org/10.1002/pul2.70293
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.
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