Eltrombopag in the management of aplastic anaemia: real-world experience in a non-trial setting

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Abstract

Objective: The thrombopoietin mimetic eltrombopag has been used in clinical trials for the frontline and salvage treatment of aplastic anaemia (AA). Eltrombopag was investigated in AA patients on a non-trial all-comer basis. Methods: Consecutive newly diagnosed and relapsed/refractory AA patients were treated with eltrombopag. Results: In a 4.5-year period, 20 consecutive AA patients (newly diagnosed, N = 10; relapsed/refractory, N = 10) at a median age of 47 (22–84) years were treated with eltrombopag. For newly diagnosed patients, the frontline use of eltrombopag (concomitant medications: anti-thymocyte globulin, ATG, and ciclosporin, N = 4; ciclosporin, N = 5; nil, N = 1) at a median maximum dose of 150 (50–300) mg/day led to an overall response rate (ORR) of 90% (trilineage: 60%; neutrophil: 20%; platelet: 10%). After a median follow-up of 47 (14–179) weeks, responses were maintained in all cases. In relapsed/refractory patients, eltrombopag at a median maximum dose of 150 (50–300) mg/day led to an ORR of 50% (trilineage: 40%; neutrophil: 10%), with responses maintained after a median follow-up of 115 (53–253) weeks. Adverse effects included reversible skin pigmentation (observed in all patients taking eltrombopag at ≥150 mg/day), dyspepsia, and liver function derangement. Conclusion: In a routine haematological practice, the use of eltrombopag in AA patients was feasible, safe, and associated with very favourable responses.

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APA

Hwang, Y. Y., Gill, H., Chan, T. S. Y., Leung, G. M. K., Cheung, C. Y. M., & Kwong, Y. L. (2018). Eltrombopag in the management of aplastic anaemia: real-world experience in a non-trial setting. Hematology, 23(7), 399–404. https://doi.org/10.1080/10245332.2017.1422306

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