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Addition of eltrombopag to immunosuppressive therapy in patients with newly diagnosed aplastic anemia
Author(s) -
Assi Rita,
GarciaManero Guillermo,
Ravandi Farhad,
Borthakur Gautam,
Daver Naval G.,
Jabbour Elias,
Burger Jan,
Estrov Zeev,
Dinardo Courtney D.,
Alvarado Yesid,
Hendrickson Stephany,
Ferrajoli Alessandra,
Wierda William,
Cortes Jorge,
Kantarjian Hagop,
Kadia Tapan M.
Publication year - 2018
Publication title -
cancer
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 3.052
H-Index - 304
eISSN - 1097-0142
pISSN - 0008-543X
DOI - 10.1002/cncr.31658
Subject(s) - eltrombopag , medicine , aplastic anemia , gastroenterology , refractory (planetary science) , surgery , bone marrow , immune thrombocytopenia , platelet , physics , astrobiology
Background The immune‐mediated destruction of hematopoietic stem cells is implicated in the pathophysiology of aplastic anemia (AA). Immunosuppressive therapy (IST) using antithymocyte globulin and cyclosporine is successful in this setting. Eltrombopag is active in patients with refractory AA, presumably by increasing the bone marrow progenitors. Methods This phase 2 trial initially was designed to evaluate standard IST in newly diagnosed patients with severe AA and later was amended to add eltrombopag to simultaneously address immune destruction and stem cell depletion. The primary outcome was the overall response rate (ORR) at 3 months and 6 months. Results A total of 38 patients were enrolled: 17 (45%) received IST alone and 21 (55%) received additional eltrombopag. The ORR was 74%. Patients receiving IST plus eltrombopag had a similar ORR (76% vs 71%; P = .72), complete remission rate (38% vs 29%; P = .73), and median time to response (84 days vs 57 days; P = .30) compared with those receiving IST alone. The 2‐year overall survival rate in the IST group was 91% compared with 82% for those patients treated with IST plus eltrombopag ( P = .82). No cumulative toxicities were noted after the addition of eltrombopag. Conclusions The addition of eltrombopag to standard IST was well tolerated and resulted in similar responses.