Paul J. Critser, Usha S. Krishnan, Catherine M. Avitabile, Andrea L. Jones, Benjamin S. Frank, Dunbar D. Ivy, Emily P. Williams, Rachel T. Sullivan, Eric D. Austin, Dephine Yung, Stephanie S. Handler, Edward Kirkpatrick, Mary P. Mullen, Claire Parker, Megan Griffiths, Patrick D. Evers, Meredith J. O’Neil, Neelam D. Bhatt, Rachel Capote, Erika B. Rosenzweig, Nidhy P. Varghese
Cincinnati Children’s Hospital Medical Center and University of Cincinnati College of Medicine. Columbia University Irving Medical Center. Children’s Hospital of Philadelphia and University of Pennsylvania Perelman School of Medicine. University of Colorado School of Medicine. Children’s Healthcare of Atlanta Cardiology and Emory University School of Medicine. Vanderbilt University Medical Center and Monroe Carrell Jr Children’s Hospital. University of Washington School of Medicine. Medical College of Wisconsin. Boston Children’s Hospital and Harvard Medical School. University of California, San Francisco. University of Texas Southwestern Medical Center. Oregon Health and Sciences University. Texas Children’s Hospital and Baylor College of Medicine. Westchester Medical Center.
United States
Chest
Chest 2026;
DOI: 10.1016/j.chest.2026.09.096
Abstract
Background: Sotatercept, a ligand trap for TGF-β family members, has been shown to improve exercise capacity and decrease pulmonary vascular resistance (PVR) in adults with pulmonary arterial hypertension. Limited data exist describing the use of sotatercept in children with pulmonary hypertension (PH).
Research question: What is the safety and efficacy of sotatercept as add-on therapy in children with PH.
Study design and methods: This is a multi-center, retrospective study of children and young adults ≤ 21 years of age with pediatric PH treated by programs in the Pediatric PH Network and institutional registries. Demographic data and clinical characteristics before and after sotatercept initiation were collected.
Results: Sixty-six subjects received sotatercept as add-on therapy for a median duration of 323 days (Interquartile range 239, 500). Forty (60%) were female with median age 15.3 years (range 3.0 – 21.0) at initiation of sotatercept, and 61 with Group 1 (92%) PH. WHO functional class improved in 51% and did not worsen in any subjects (p < 0.0001). Subjects experienced significant improvement in six-minute walk distance (median 31 m, p=0.0003, n=40), NT-proBNP (median decrease 378 pg/ml, p< 0.0001, n=26), mean pulmonary artery pressure (median decrease 17 mmHg, p<0.0001, n=21), and PVR (median decrease 4.4 WUi, p=0.0011, n=21). Parenteral prostacyclin was weaned or discontinued in 13 of 38 patients. Adverse events occurred in 33 (50%) subjects with bleeding in 15 (23%). No patients had escalation of PH therapy, were hospitalized for clinical worsening, underwent lung transplant, or died.
Interpretation: Pediatric PH patients treated with sotatercept as add-on therapy demonstrated improvement in functional class and other PH disease indicators and was generally well tolerated.
Category
Medical Therapy. Efficacy or Lack of Efficacy
Medical Therapy. Adverse Effects or Lack of Adverse Effects
Age Focus: Pediatric Pulmonary Vascular Disease or Adult Pulmonary Vascular Disease
Fresh or Filed Publication: Fresh (PHresh). Less than 1-2 years since publication
Article Access
Free PDF File or Full Text Article Available Through PubMed or DOI: Yes