Eltrombopag in combination with immunosuppressive therapy in pediatric severe aplastic anemia: phase 2 ESCALATE trial
9
Issued Date
2025-08-12
Resource Type
ISSN
24739529
eISSN
24739537
Scopus ID
2-s2.0-105012262873
Pubmed ID
40315366
Journal Title
Blood Advances
Volume
9
Issue
15
Start Page
3728
End Page
3738
Rights Holder(s)
SCOPUS
Bibliographic Citation
Blood Advances Vol.9 No.15 (2025) , 3728-3738
Suggested Citation
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. Eltrombopag in combination with immunosuppressive therapy in pediatric severe aplastic anemia: phase 2 ESCALATE trial. Blood Advances Vol.9 No.15 (2025) , 3728-3738. 3738. doi:10.1182/bloodadvances.2024015102 Retrieved from: https://repository.li.mahidol.ac.th/handle/123456789/111640
Title
Eltrombopag in combination with immunosuppressive therapy in pediatric severe aplastic anemia: phase 2 ESCALATE trial
Author(s)
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.
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.
Author's Affiliation
Harvard Medical School
Chinese University of Hong Kong
Northwestern University Feinberg School of Medicine
Cleveland Clinic Foundation
Emory University School of Medicine
Duke University Medical Center
Universitätsklinikum Freiburg
University of Arkansas for Medical Sciences
Novartis International AG
St. Jude Children's Research Hospital
Texas Children's Hospital
Great Ormond Street Hospital for Children NHS Foundation Trust
Siriraj Hospital
University of Colorado Department of Pediatrics
Pavlov University
Faculty of Medicine, Khon Kaen University
University of Arizona College of Medicine – Phoenix
Faculty of Medicine Ramathibodi Hospital, Mahidol University
C.S. Mott Children's Hospital
Unidade Local de Saúde Santa Maria, E.P.E
Cohen Children’s Medical Center
Dmitry Rogachev National Research Center of Pediatric Hematology, Oncology and Immunology
The Fetal Center at Riley Children’s Health
Chinese University of Hong Kong
Northwestern University Feinberg School of Medicine
Cleveland Clinic Foundation
Emory University School of Medicine
Duke University Medical Center
Universitätsklinikum Freiburg
University of Arkansas for Medical Sciences
Novartis International AG
St. Jude Children's Research Hospital
Texas Children's Hospital
Great Ormond Street Hospital for Children NHS Foundation Trust
Siriraj Hospital
University of Colorado Department of Pediatrics
Pavlov University
Faculty of Medicine, Khon Kaen University
University of Arizona College of Medicine – Phoenix
Faculty of Medicine Ramathibodi Hospital, Mahidol University
C.S. Mott Children's Hospital
Unidade Local de Saúde Santa Maria, E.P.E
Cohen Children’s Medical Center
Dmitry Rogachev National Research Center of Pediatric Hematology, Oncology and Immunology
The Fetal Center at Riley Children’s Health
Corresponding Author(s)
Other Contributor(s)
Abstract
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 × 10<sup>9</sup>/L to 200 × 10<sup>9</sup>/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.