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.
CITATION STYLE
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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