Evaluation of eltrombopag in patients with aplastic anemia in real-world experience

Highlights • The use of eltrombopag was expanded to cover AA in Japan in August 2017. • The overall response rate was 55% (6/11). • Four patients had recovery of all three cell lineages, and two patients had only neutrophil recovery. • Stage at the initial assessment, the neutrophil-to-lymphocyte ratio and platelet counts were significantly different between the responders and non-responders.
Background Although eltrombopag has recently been approved for treating AA, the effects of its clinical use remain unknown. Methods We retrospectively analyzed 11 patients with AA, who had been treated with eltrombopag from August 2017 to May 2018. Results Overall response rate was 55%. There was tri-lineage recovery in four patients and platelet recovery in two. The reactive time was within 8 weeks after treatment initiation. Stage at the initial assessment, the neutrophil-to-lymphocyte ratio and platelet counts were significantly different between the responders and non-responders. Conclusion Eltrombopag is a promising agent for treating patients with any degree of AA.