Efficacy of eltrombopag for treatment of thrombocytopenia in the setting of allogeneic hematopoietic cell transplantation: a systematic review and meta-analysis.

Thrombocytopenia following an allogeneic hematopoietic cell transplant (allo-HCT) is a potentially serious complication, and the efficacy of eltrombopag, a thrombopoietin receptor agonist, in this context is unclear due to inconsistent findings. Additionally, other post-allograft outcomes in eltrombopag treated patients such as transfusion independence, overall survival (OS), and non-relapse mortality have not been systematically reviewed. The aim of this systematic review/meta-analysis (SR/MA) is to evaluate the efficacy of eltrombopag in allo-HCT-induced thrombocytopenia by analyzing data from 16 eligible studies. Pooled rate of response for platelets counts achieving >30 × 109/L and >50 × 109/L were 72% and 56%, respectively. When evaluating the composite endpoint of platelets response and transfusion independence, pooled rates for >30 × 109/L and >50 × 109/L plus transfusion independence were 47% and 56%, respectively. Pooled OS, bleeding-related mortality and mortality from GVHD/infection were 68%, 6%, and 19%, respectively. These findings show that eltrombopag is an effective treatment of allo-HCT-induced thrombocytopenia. Optimal dose and duration of treatment remain to be determined in a large prospective study. Results of this SR/MA suggest a beneficial effect of eltrombopag for thrombocytopenia after allo-HCT. These results could represent the benchmark to be used for future prospective and comparative studies to better understand the benefit of this intervention.