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AJH:艾曲波帕治疗单倍体相同外周血干细胞移植后持续性血小板减少症患者

2021-11-21 MedSci原创 MedSci原创

持续性血小板减少症(PT)是异基因造血干细胞移植(allo-HSCT)后常见的并发症,对生存率产生显著的负面影响。已发表的数据表明,PT的机制可归因于血小板生成受损、外周破坏加速或两种之间的相互作用。

持续性血小板减少症(PT)是异基因造血干细胞移植(allo-HSCT)后常见的并发症,对生存率产生显著的负面影响。已发表的数据表明,PT的机制可归因于血小板生成受损、外周破坏加速或两种之间的相互作用。同种异体造血干细胞移植 (allo-HSCT) 后持续性血小板减少症 (PT) 与出血风险增加和生存率降低有关。但目前PT 的确切发病机制尚不清楚,其管理也很困难。

在这里,国外一研究团队进行了一项回顾性研究,以评估艾曲波帕 (EPAG) 在 34 名同种异体造血干细胞移植后 PT 患者中的疗效和安全性。

7 名患者患有长期孤立性血小板减少症 (PIT),27 名患者出现继发性血小板恢复失败 (SFPR)。对于大多数患者,初始剂量为每天25mg或50mg,然后根据患者血小板恢复的反应和耐受性调整至每天50-100mg的最大剂量。

图1 :a,总体缓解的累计发生率(72.1%,n=34)。b,累积完全缓解发生率(60.7%,n=34)(由EZR(日本埼玉市直知医科大学埼玉医学中心)创建)

图2:停药与停药1个月后血小板计数中位数(P=0.586)(由Prism 8创建(GraphPad Software, La Jolla, CA))

无输血支持的血小板恢复至至少20×109×/L和50×109/L的累积发生率(CI)分别为72.1%和60.7%。22名应答者中有19例(86.4%)能够减少用药;此外,停用EPAG1个月后血小板计数保持稳定。虽然有2例患者在治疗期间因头痛和恶心停用了EPAG,但没有患者出现3级或4级毒性。多变量分析发现,骨髓发育不全和巨核细胞(MKs)减少分别是总体反应(OR)和完全反应(CR)的危险因素。

图3 :a,骨髓增生患者(n=27)和骨髓发育不全患者(n=7)的总缓解累积发生率(P=0.0216)。b,巨核细胞充足患者(n=19)和巨核细胞不足患者(n=15)之间的累积完全缓解发生率(P=0.0363)(由EZR创建(日本埼玉医科大学埼玉医学中心))

     总的来说,在有效性和安全性方面,这是单倍体PBSCT后PT的一种很有前途的治疗方法。停用后可持续的血小板计数表明,可能没有必要持续暴露于EPAG。足够的巨核细胞和骨髓增生可能是一个有用的预测因子

 

原始出处:

Yan, F., Lu, N., Gu, Z. et al. Eltrombopag in the treatment of patients with persistent thrombocytopenia after haploidentical peripheral blood stem cell transplantation: a single-center experience. Ann Hematol (2021). https://doi.org/10.1007/s00277-021-04706-6

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    2021-11-21 ms6000001024089719

    好极了

    0

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