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<p>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 &gt;30 × 10<sup>9</sup>/L and &gt;50 × 10<sup>9</sup>/L were 72% and 56%, respectively. When evaluating the composite endpoint of platelets response and transfusion independence, pooled rates for &gt;30 × 10<sup>9</sup>/L and &gt;50 × 10<sup>9</sup>/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&#xa0;to better understand the benefit of this intervention.</p>

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

  • 期刊论文

  • 发表日期：

    2025年12月5日

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  • Arni Kumar,
  • Arin Singh,
  • Tea Reljic,
  • Madiha Iqbal,
  • Razan Mohty,
  • Hemant Murthy,
  • Taiga Nishihori,
  • Ricardo Parrondo,
  • Ernesto Ayala,
  • Vivek Roy,
  • Mahmoud Aljurf,
  • Mohamed A. Kharfan-Dabaja

摘要

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.