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Eltrombopag in children with severe aplastic anemia
Author(s) -
Lesmana Harry,
Jacobs Timothy,
Boals Michelle,
Gray Nathan,
Lewis Sara,
Ding Juan,
Kang Guolian,
Hale Melvanique,
Weiss Mitchell,
Reiss Ulrike,
Wang Winfred,
Wlodarski Marcin
Publication year - 2021
Publication title -
pediatric blood and cancer
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.116
H-Index - 105
eISSN - 1545-5017
pISSN - 1545-5009
DOI - 10.1002/pbc.29066
Subject(s) - eltrombopag , medicine , aplastic anemia , pediatrics , paroxysmal nocturnal hemoglobinuria , anemia , thrombopoietin receptor , retrospective cohort study , thrombopoietin , gastroenterology , surgery , bone marrow , haematopoiesis , platelet , genetics , stem cell , biology , immune thrombocytopenia
Background Immunosuppressive therapy with horse antithymocyte globulin and cyclosporine currently remains the standard therapy for children with severe aplastic anemia (SAA) who lack human leukocyte antigen (HLA)‐identical sibling. The thrombopoietin receptor agonist eltrombopag has been recently approved for SAA patients 2 years and older. However, there are limited data on its safety and efficacy in pediatric cohorts. Methods We conducted a retrospective study of patients ≤18 years old consecutively diagnosed with SAA between 2000 and 2018. Patients received either standard immunosuppressive therapy (IST‐Std) or IST with eltrombopag (IST‐Epag). The primary outcome was the objective response (OR), including partial and complete response (CR), at 6 and 12 months after starting therapy. Results We identified 16 patients receiving IST‐Std and nine IST‐Epag treatment (seven of nine as upfront therapy and two of seven after previously failed IST). The OR at 6 and 12 months in IST‐Std arm was 71% and 100%, with CR in 29% and 58%, respectively. Seven patients receiving upfront IST‐Epag had OR at 6 and 12 months, with two of seven (29%) achieving CR at 6 and 12 months. Two patients who previously failed standard IST did not respond to eltrombopag. No significant differences were observed in both cohorts with regard to infections. One IST‐Epag‐treated patient developed transient grade 3 transaminitis. Finally, no changes in paroxysmal nocturnal hemoglobinuria (PNH) clone size and cytogenetic abnormalities were seen in either cohort. Conclusion The addition of eltrombopag to standard IST was well tolerated and resulted in satisfactory hematological response at 6 and 12 months in this single‐institution experience. A larger cohort with longer follow‐up is required to assess response durability.