Blood:艾曲波帕(EPAG)与难治性重度再生障碍性贫血(rSAA)

2019-04-18 MedSci MedSci原创

基于43位患者治疗12周(50-150mg)的效果,艾曲波帕(EPAG)获得FDA批准用于治疗难治性重度再生障碍性贫血(rSAA)。反应动力学表明延长EPAG(150mg)的给药时间可加速反应速度,提高缓解率。研究人员招募了40位rSAA患者,EPAG 150mg/日,主要评估指标是24周时的缓解率。24周时,有20位(50%)患者获得缓解,其中5位(25%)在12周时被认为是无反应者。15/19

基于43位患者治疗12周(50-150mg)的效果,艾曲波帕(EPAG)获得FDA批准用于治疗难治性重度再生障碍性贫血(rSAA)。反应动力学表明延长EPAG(150mg)的给药时间可加速反应速度,提高缓解率。

研究人员招募了40位rSAA患者,EPAG 150mg/日,主要评估指标是24周时的缓解率。

24周时,有20位(50%)患者获得缓解,其中5位(25%)在12周时被认为是无反应者。15/19位继续EPAG治疗的患者因反应强烈而停药,其中5位因复发而再次采用EPAG治疗,复发的5位患者对EPAG再治疗均有反应。

为分析克隆进展的风险,研究人员将两次的研究结合起来,共有83位rSAA患者的长期随访数据。16位(19%)患者出现异常核型,大部分发生于EPAG治疗的前6个月。

在治疗前、主要评估结点、和克隆进化及最后一次随访时对患者进行深度靶向测序/全外显子测序。细胞遗传进化与突变状态无关,EPAG对髓系癌基因的突变等位基因片段无明显影响。

总而言之,EPAG(150mg/日)治疗12周无反应的rSAA患者,延长治疗至24周,或可获得缓解。EPAG可能可促进已存在的异常核型的休眠克隆扩增。

原始出处:

Thomas Winkler, et al.Eltrombopag for refractory severe aplastic anemia: dosing, duration, long term outcomes and clonal evolution.Blood 2019 :blood.2019000478; doi: https://doi.org/10.1182/blood.2019000478 

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    2020-03-23 朱睿睿
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    2019-04-20 gwc384
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    2019-04-20 freve
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    2019-04-18 orangesking

    0

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