{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,30]],"date-time":"2026-09-30T13:25:49Z","timestamp":1790774749156,"version":"4.1.0"},"reference-count":14,"publisher":"Springer Science and Business Media LLC","license":[{"start":{"date-parts":[[2026,6,26]],"date-time":"2026-06-26T00:00:00Z","timestamp":1782432000000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0"},{"start":{"date-parts":[[2026,6,26]],"date-time":"2026-06-26T00:00:00Z","timestamp":1782432000000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0"}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["Pediatr Cardiol"],"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:p>Sotatercept, a first-in-class activin signaling inhibitor, has demonstrated disease-modifying efficacy in adults with pulmonary arterial hypertension (PAH). Pediatric data remain limited to ongoing clinical trials. We conducted an IRB-approved, single-center retrospective analysis of pediatric patients with WHO Group 1 or Group 3 PAH treated with off-label sotatercept at Rady Children\u2019s Hospital \/ UC San Diego. All patients had received maximal background triple therapy yet demonstrated persistent symptoms or adverse hemodynamics. Right heart catheterization was performed before sotatercept initiation and at approximately 24 weeks thereafter. Hemodynamic parameters were analyzed descriptively. Seven pediatric patients (median age 14 years; range 4\u201319) were included. Diagnoses comprised idiopathic PAH, heritable PAH, and PAH associated with congenital heart disease or congenital diaphragmatic hernia. Following sotatercept initiation, the majority of patients demonstrated reductions in pulmonary vascular resistance index (PVRI) and mean pulmonary artery pressure (mPAP), with heterogeneity in magnitude of response. Pulmonary capillary wedge pressure (PCWP) remained stable in all patients. In this small, highly selected pediatric cohort with advanced PAH refractory to triple therapy, off-label sotatercept was associated with favorable hemodynamic trends at 24 weeks. These findings support biologic plausibility suggested by adult randomized trials and underscore the urgent need for prospective pediatric studies.<\/jats:p>","DOI":"10.1007\/s00246-026-04349-2","type":"journal-article","created":{"date-parts":[[2026,6,26]],"date-time":"2026-06-26T03:32:18Z","timestamp":1782444738000},"update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":2,"title":["Off-Label Sotatercept Use in Pediatric Pulmonary Arterial Hypertension: A Single-Center Retrospective Hemodynamic Study"],"prefix":"10.1007","author":[{"given":"Dhruv","family":"Rao","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Shylah","family":"Haldeman","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jeanne","family":"Carroll","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rohit","family":"Rao","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2026,6,26]]},"reference":[{"key":"4349_CR1","doi-asserted-by":"publisher","first-page":"1204","DOI":"10.1056\/NEJMoa2024277","volume":"384","author":"M Humbert","year":"2021","unstructured":"Humbert M, McLaughlin V, Gibbs JSR et al (2021) Sotatercept for the treatment of pulmonary arterial hypertension. N Engl J Med 384:1204\u20131215. https:\/\/doi.org\/10.1056\/NEJMoa2024277","journal-title":"N Engl J Med"},{"key":"4349_CR2","doi-asserted-by":"publisher","unstructured":"Doggrell SA (2023) Is sotatercept, which traps activins and growth differentiation factors, a new dawn in treating pulmonary arterial hypertension (PAH)? Expert Opin Biol Ther 23(7):589\u2013593. https:\/\/doi.org\/10.1080\/14712598.2023.2221784","DOI":"10.1080\/14712598.2023.2221784"},{"key":"4349_CR3","doi-asserted-by":"publisher","first-page":"1234","DOI":"10.3390\/jcm14155177","volume":"14","author":"C Dagher","year":"2025","unstructured":"Dagher C, Akiki M, Swanson K et al (2025) Sotatercept in connective tissue disease-associated pulmonary arterial hypertension with interstitial lung disease. J Clin Med 14:1234. https:\/\/doi.org\/10.3390\/jcm14155177","journal-title":"J Clin Med"},{"key":"4349_CR4","doi-asserted-by":"publisher","first-page":"2402102","DOI":"10.1183\/13993003.01424-2024","volume":"65","author":"MM Hoeper","year":"2025","unstructured":"Hoeper MM, Gomberg-Maitland M, Badesch DB et al (2025) Efficacy and safety of sotatercept in pooled PULSAR and STELLAR analyses. Eur Respir J 65:2402102. https:\/\/doi.org\/10.1183\/13993003.01424-2024","journal-title":"Eur Respir J"},{"key":"4349_CR5","doi-asserted-by":"publisher","first-page":"1478","DOI":"10.1056\/NEJMoa2213558","volume":"388","author":"MM Hoeper","year":"2023","unstructured":"Hoeper MM, Badesch DB, Ghofrani HA et al (2023) Phase 3 trial of sotatercept for pulmonary arterial hypertension. N Engl J Med 388:1478\u20131490. https:\/\/doi.org\/10.1056\/NEJMoa2213558","journal-title":"N Engl J Med"},{"key":"4349_CR6","doi-asserted-by":"publisher","first-page":"1007","DOI":"10.1002\/clc.24173","volume":"47","author":"A Nasrollahizadeh","year":"2023","unstructured":"Nasrollahizadeh A, Soleimani H, Hashemi S, Hosseini K (2023) Navigating the sotatercept landscape: a meta-analysis of clinical outcomes. Clin Cardiol 47:1007\u20131016. https:\/\/doi.org\/10.1002\/clc.24173","journal-title":"Clin Cardiol"},{"key":"4349_CR7","doi-asserted-by":"publisher","first-page":"1","DOI":"10.1080\/17476348.2025.2530201","volume":"19","author":"M Ershed","year":"2025","unstructured":"Ershed M, Da Silva A, Cavalcante D et al (2025) Efficacy and safety of sotatercept in pulmonary arterial hypertension: a systematic review and meta-analysis. Expert Rev Respir Med 19:1\u201310. https:\/\/doi.org\/10.1080\/17476348.2025.2530201","journal-title":"Expert Rev Respir Med"},{"key":"4349_CR8","doi-asserted-by":"publisher","first-page":"107520","DOI":"10.1016\/j.vph.2025.107520","volume":"155","author":"M Abdulelah","year":"2025","unstructured":"Abdulelah M, Ezenna C, Jenil-Franco A et al (2025) Clinical outcomes of sotatercept in pulmonary arterial hypertension. Vasc Pharmacol 155:107520. https:\/\/doi.org\/10.1016\/j.vph.2025.107520","journal-title":"Vasc Pharmacol"},{"key":"4349_CR9","doi-asserted-by":"publisher","first-page":"145","DOI":"10.1513\/AnnalsATS.202311-942OC","volume":"21","author":"T Pitre","year":"2024","unstructured":"Pitre T, Desai K, Mah J, Humbert M (2024) Comparative effectiveness of sotatercept for pulmonary arterial hypertension. Ann Am Thorac Soc 21:145\u2013153. https:\/\/doi.org\/10.1513\/AnnalsATS.202311-942OC","journal-title":"Ann Am Thorac Soc"},{"key":"4349_CR10","doi-asserted-by":"publisher","first-page":"e002873","DOI":"10.1136\/openhrt-2025-003636","volume":"12","author":"Z Cao","year":"2025","unstructured":"Cao Z, Xiao Q, Li X, Zhang H, Zhang M (2025) Postmarketing analysis of sotatercept: identifying serious unlabelled adverse events. Open Heart 12:e002873. https:\/\/doi.org\/10.1136\/openhrt-2025-003636","journal-title":"Open Heart"},{"key":"4349_CR11","doi-asserted-by":"publisher","first-page":"e63","DOI":"10.1016\/S2352-3026(18)30002-4","volume":"5","author":"R Komrokji","year":"2018","unstructured":"Komrokji R, Garcia-Manero G, Ad\u00e8s L et al (2018) Sotatercept in lower-risk myelodysplastic syndromes. Lancet Haematol 5:e63\u2013e72. https:\/\/doi.org\/10.1016\/S2352-3026(18)30002-4","journal-title":"Lancet Haematol"},{"key":"4349_CR12","doi-asserted-by":"publisher","first-page":"477","DOI":"10.3324\/haematol.2018.198887","volume":"104","author":"MD Cappellini","year":"2019","unstructured":"Cappellini MD, Porter J, Origa R et al (2019) Sotatercept, a novel transforming growth factor \u03b2 ligand trap, improves anemia in \u03b2-thalassemia. Haematologica 104:477\u2013484. https:\/\/doi.org\/10.3324\/haematol.2018.198887","journal-title":"Haematologica"},{"key":"4349_CR13","doi-asserted-by":"publisher","first-page":"1585","DOI":"10.1016\/j.ekir.2019.08.001","volume":"4","author":"DW Coyne","year":"2019","unstructured":"Coyne DW, Singh H, Smith W et al (2019) Three times weekly epoetin alfa and safety data for sotatercept: final results of a phase 2 study. 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Support Care Cancer 24:1517\u20131525. https:\/\/doi.org\/10.1007\/s00520-015-2929-9","journal-title":"Support Care Cancer"}],"container-title":["Pediatric Cardiology"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1007\/s00246-026-04349-2.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/article\/10.1007\/s00246-026-04349-2","content-type":"text\/html","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1007\/s00246-026-04349-2.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,8,10]],"date-time":"2026-08-10T14:13:14Z","timestamp":1786371194000},"score":1,"resource":{"primary":{"URL":"https:\/\/link.springer.com\/10.1007\/s00246-026-04349-2"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2026,6,26]]},"references-count":14,"alternative-id":["4349"],"URL":"https:\/\/doi.org\/10.1007\/s00246-026-04349-2","relation":{"has-preprint":[{"id-type":"doi","id":"10.21203\/rs.3.rs-9261199\/v1","asserted-by":"object"}]},"ISSN":["0172-0643","1432-1971"],"issn-type":[{"value":"0172-0643","type":"print"},{"value":"1432-1971","type":"electronic"}],"subject":[],"published":{"date-parts":[[2026,6,26]]},"assertion":[{"value":"29 March 2026","order":1,"name":"received","label":"Received","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"10 June 2026","order":2,"name":"accepted","label":"Accepted","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"26 June 2026","order":3,"name":"first_online","label":"First Online","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"10 August 2026","order":5,"name":"change_date","label":"Change Date","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"Update","order":6,"name":"change_type","label":"Change Type","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"Wrong DOIs listing in references has been corrected.","order":7,"name":"change_details","label":"Change Details","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"The authors declare no competing interests.","order":1,"name":"Ethics","label":"Conflict of interest","group":{"name":"EthicsHeading","label":"Declarations"}},{"value":"This study was approved by the Institutional Review Board of Rady Children\u2019s Hospital \/ UC San Diego. All procedures were conducted in accordance with the Declaration of Helsinki.","order":2,"name":"Ethics","label":"Ethical Approval","group":{"name":"EthicsHeading","label":"Declarations"}},{"value":"Informed consent for off-label sotatercept use was obtained from parents or legal guardians of all patients.","order":3,"name":"Ethics","label":"Informed Consent","group":{"name":"EthicsHeading","label":"Declarations"}}]}}