{"database":"biostudies-literature","file_versions":[],"scores":null,"additional":{"omics_type":["Unknown"],"volume":["9(15)"],"submitter":["Shimamura A"],"pubmed_abstract":["<h4>Abstract</h4>Severe aplastic anemia (SAA) is a rare, life-threatening disease with acquired pancytopenia and hypocellular bone marrow. ESCALATE evaluated eltrombopag in combination with immunosuppressive therapy (IST) in pediatric patients (aged 1 to <18 years) with relapsed/refractory (R/R) or treatment-naïve SAA. The eltrombopag starting dose was 25 mg/d for patients aged 1 to <6 years and 50 mg/d for patients aged 6 to <18 years; dose modifications (maximum dose, 150 mg/d) were allowed to achieve a target platelet count of 50 × 109/L to 200 × 109/L. Eltrombopag was administered with cyclosporine A, with or without horse antithymocyte globulin, for 26 weeks and could be extended if clinically beneficial. Fifty-one patients were treated (R/R SAA, n = 14; treatment-naïve SAA, n = 37). "],"journal":["Blood advances"],"pagination":["3728-3738"],"full_dataset_link":["https://www.ebi.ac.uk/biostudies/studies/S-EPMC12305589"],"repository":["biostudies-literature"],"pubmed_title":["Eltrombopag in combination with immunosuppressive therapy in pediatric severe aplastic anemia: phase 2 ESCALATE trial."],"pmcid":["PMC12305589"],"pubmed_authors":["Pongtanakul B","Rothman JA","Walkovich K","Komvilaisak P","Ferrao A","Bhat R","Sirachainan N","Nakano TA","Samarasinghe S","Hanna R","Li CK","Wang Q","Maschan A","Bertuch AA","Wang W","Zubarovskaya L","Vlachos A","Wong TF","Bennett C","Farrar JE","Urban P","Shimamura A","Boklan J","Williams DA","Strahm B","Overholt K"],"additional_accession":[]},"is_claimable":false,"name":"Eltrombopag in combination with immunosuppressive therapy in pediatric severe aplastic anemia: phase 2 ESCALATE trial.","description":"<h4>Abstract</h4>Severe aplastic anemia (SAA) is a rare, life-threatening disease with acquired pancytopenia and hypocellular bone marrow. ESCALATE evaluated eltrombopag in combination with immunosuppressive therapy (IST) in pediatric patients (aged 1 to <18 years) with relapsed/refractory (R/R) or treatment-naïve SAA. The eltrombopag starting dose was 25 mg/d for patients aged 1 to <6 years and 50 mg/d for patients aged 6 to <18 years; dose modifications (maximum dose, 150 mg/d) were allowed to achieve a target platelet count of 50 × 109/L to 200 × 109/L. Eltrombopag was administered with cyclosporine A, with or without horse antithymocyte globulin, for 26 weeks and could be extended if clinically beneficial. Fifty-one patients were treated (R/R SAA, n = 14; treatment-naïve SAA, n = 37). ","dates":{"release":"2025-01-01T00:00:00Z","publication":"2025 Aug","modification":"2026-05-29T17:09:07.617Z","creation":"2026-04-08T05:29:02.405Z"},"accession":"S-EPMC12305589","cross_references":{"pubmed":["40315366"],"doi":["10.1182/bloodadvances.2024015102"]}}