赫曲波帕治疗同种异体造血干细胞移植后重组人血小板生成素抵抗性血小板减少症的有效性和安全性

IF 3.4 3区医学 Q2 HEMATOLOGY

Research and Practice in Thrombosis and Haemostasis Pub Date : 2024-10-01 DOI:10.1016/j.rpth.2024.102578

Jing Ni , Jian Hong , Xinglin Liang , Jifei Dai , Zhangbiao Long , ChengXin Luan , Mingzhen Yang , Qingsheng Li

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引用次数: 0

摘要

背景异基因造血细胞移植后血小板减少是一个具有挑战性的临床问题。重组人血小板生成素（rhTPO）和血小板生成素受体激动剂越来越多地被用于治疗移植后血小板减少症。方法这项回顾性研究纳入了21例rhTPO耐药的移植后血小板减少症患者，他们在2021年8月至2022年7月期间接受了赫曲波帕治疗。主要终点是总反应率，包括部分反应和完全反应（CR）。我们还评估了赫曲波帕格疗效和不良事件的预测因素。结果赫曲波帕格的总体应答率为81%，CR率为62%。从开始使用赫曲博帕到出现应答和CR的中位时间分别为16天和31天。骨髓中巨核细胞的减少与赫曲波帕的 CR 负相关（P = 0.03）。所有患者都能很好地耐受赫曲波帕，不良反应没有明显增加。结论我们的研究结果表明，希曲波帕是促进移植后血小板减少症患者血小板恢复的有效治疗方案，即使是对 rhTPO 耐药的患者也不例外。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

Efficacy and safety of hetrombopag in the treatment of recombinant human thrombopoietin–resistant thrombocytopenia after allogeneic hematopoietic stem cell transplantation

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Efficacy and safety of hetrombopag in the treatment of recombinant human thrombopoietin–resistant thrombocytopenia after allogeneic hematopoietic stem cell transplantation

Background

Thrombocytopenia after allogeneic hematopoietic cell transplantation is a challenging clinical problem. Recombinant human thrombopoietin (rhTPO) and thrombopoietin receptor agonists are increasingly used in posttransplant thrombocytopenia. However, the use of hetrombopag in patients with posttransplant thrombocytopenia, especially in patients with resistance to rhTPO, has not yet been reported.

Objectives

The present study aimed to investigate the efficacy and safety of hetrombopag in patients with rhTPO-resistant posttransplant thrombocytopenia.

Methods

This retrospective study included 21 patients with rhTPO-resistant posttransplant thrombocytopenia who received hetrombopag from August 2021 to July 2022. The primary endpoint was the overall response rate, including partial response and complete response (CR). We also evaluated the predictors of hetrombopag efficacy and adverse events.

Results

The overall response rate to hetrombopag was 81%, and the CR rate was 62%. The median time from hetrombopag initiation to response and CR were 16 and 31 days, respectively. Decreased megakaryocytes in bone marrow negatively correlated with CR to hetrombopag (P = .03). All the patients tolerated hetrombopag well without any significant increase in adverse events. At the last follow-up, 71% of responders had discontinued hetrombopag and sustained their best response.

Conclusion

Our results suggested that hetrombopag is an effective treatment option to promote platelet recovery in patients with posttransplant thrombocytopenia, even in patients resistant to rhTPO.

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