Shimamura A.Maschan A.Bennett C.Samarasinghe S.Farrar J.E.Li C.K.Sirachainan N.Pongtanakul B.Komvilaisak P.Zubarovskaya L.Rothman J.A.Walkovich K.Nakano T.A.Bertuch A.A.Ferrao A.Bhat R.Hanna R.Overholt K.Boklan J.Wong T.F.Wang Q.Urban P.Strahm B.Wang W.Vlachos A.Williams D.A.Mahidol University2025-08-152025-08-152025-08-12Blood Advances Vol.9 No.15 (2025) , 3728-373824739529https://repository.li.mahidol.ac.th/handle/123456789/111640Severe 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). Data were analyzed overall and as 2 cohorts: R/R and treatment-naïve cohorts. The overall response rate (ORR; per North American Pediatric Aplastic Anemia Consortium criteria) at 26 weeks was 54.9% in both cohorts combined and 71.4% and 48.6% in the R/R and treatment-naïve cohorts, respectively; most responders had sustained responses after discontinuing eltrombopag. Among baseline transfusion-dependent patients, 66.7% and 76.7% achieved red blood cell and platelet transfusion independence, respectively, with rates of 70% and 80% for the R/R cohort and 65.6% and 75.8% for the treatment-naïve cohort, respectively.MedicineArticleSCOPUS10.1182/bloodadvances.20240151022-s2.0-1050122628732473953740315366