Addition of Eltrombopag to Immunosuppressive Therapy in Patients with Newly Diagnosed Aplastic Anemia

Overview

Journal Cancer

Publisher Wiley

Specialty Oncology

Date 2018 Oct 12

PMID 30307606

Citations 17

Authors

Rita Assi

Guillermo Garcia-Manero

Farhad Ravandi

Gautam Borthakur

Naval G Daver

Elias Jabbour

Jan Burger

Zeev Estrov

Courtney D DiNardo

Yesid Alvarado

Stephany Hendrickson

Alessandra Ferrajoli

William Wierda

Jorge Cortes

Hagop Kantarjian

Tapan M Kadia

Affiliations

Soon will be listed here.

Abstract

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.

Citing Articles

A Systematic Review and Meta-Analysis of Eltrombopag Efficacy Combined With Immunosuppressive Drugs in Treatment of Severe Aplastic Anemia.

Illango J, Seffah K, Naveen N, Paudel Y, Patel A, Pachchipulusu V Cureus. 2024; 16(8):e65970.

PMID: 39221321 PMC: 11365712. DOI: 10.7759/cureus.65970.

Modified Delphi panel consensus recommendations for management of severe aplastic anemia.

Babushok D, DeZern A, de Castro C, Rogers Z, Beenhouwer D, Broder M Blood Adv. 2024; 8(15):3946-3960.

PMID: 38669341 PMC: 11331724. DOI: 10.1182/bloodadvances.2023011642.

Efficacy and safety of immunosuppressive therapy combined with eltrombopag for severe aplastic anemia: a systematic review and meta-analysis.

Zhang Y, Li J, Li X, Geng Q, Xie Y, Zhang G Syst Rev. 2024; 13(1):101.

PMID: 38576005 PMC: 10993616. DOI: 10.1186/s13643-024-02515-2.

Progress in medical therapy in aplastic anemia: why it took so long?.

Scheinberg P Int J Hematol. 2024; 119(3):248-254.

PMID: 38403842 DOI: 10.1007/s12185-024-03713-3.

Multicenter evaluation of the addition of eltrombopag to immunosuppressive therapy for adults with severe aplastic anemia.

Shinn L, Benitez L, Perissinotti A, Reid J, Buhlinger K, van Deventer H Int J Hematol. 2023; 118(6):682-689.

PMID: 37882977 DOI: 10.1007/s12185-023-03670-3.

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