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

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-corner 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.