To report a retrospective hemodynamic analysis of pediatric patients with severe pulmonary arterial hypertension (PAH) who received off-label sotatercept following failure of maximal triple therapy.
Approach:
Study Design: Retrospective, observational study approved by the Institutional Review Board at Rady Children’s Hospital/UC San Diego.
Patient Selection: Included pediatric patients diagnosed with WHO Group 1 or Group 3 PAH, receiving maximal triple therapy, and showing persistent symptoms or adverse hemodynamics.
Sotatercept Administration: Sotatercept was initiated off-label with dosing following adult trial protocols, and patients were monitored for adverse effects.
Hemodynamic Assessment: Right heart catheterization data were collected at baseline and 24 weeks post-initiation, analyzing pulmonary vascular resistance index (PVRI), mean pulmonary artery pressure (mPAP), and pulmonary capillary wedge pressure (PCWP).
Statistical Analysis: Data presented as individual patient values and medians with ranges; no formal statistical testing was performed.
Key Findings:
Seven pediatric patients were included, with a median age of 14 years (range 4–19).
Six of seven patients showed a reduction in pulmonary vascular resistance index (PVRI) after 24 weeks of sotatercept treatment.
Mean pulmonary artery pressure (mPAP) decreased in the majority of patients, with one patient showing stable to mildly elevated mPAP.
Pulmonary capillary wedge pressure (PCWP) remained stable across all patients.
Interpretation:
The hemodynamic improvements observed in this small cohort suggest potential efficacy of sotatercept in pediatric PAH, mirroring findings from adult studies.
Limitations:
Small sample size limits generalizability.
Retrospective design may introduce bias.
Conclusion:
Sotatercept was well tolerated in pediatric patients with severe PAH who had failed maximal therapy.
