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Blood：鲁索替尼也不能改善预后不良的高危型羟基脲耐受性ET患者的预后

2019-10-05 MedSci MedSci原创

特发性血小板增多症鲁索替尼羟基脲非MPN驱动突变
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具有高血栓风险的特发性血小板增多症(ET)患者需要细胞还原治疗，特别是羟基尿。但很多患者对羟基尿（HC0RES/INT）有耐药性或不耐受，疾病进展风险增加。MAJIC-ET是一项随机的二期研究，将鲁索替尼(RUX)与HC-RES/INT ET中最佳治疗(BAT)进行比较，结果显示两组患者在血液缓解率或疾病进展率方面没有差异。额外的非MPN驱动突变(NDM)对HC-RES/INT ET患者并发症风险

具有高血栓风险的特发性血小板增多症(ET)患者需要细胞还原治疗，特别是羟基尿。但很多患者对羟基尿（HC0RES/INT）有耐药性或不耐受，疾病进展风险增加。


MAJIC-ET是一项随机的二期研究，将鲁索替尼(RUX)与HC-RES/INT ET中最佳治疗(BAT)进行比较，结果显示两组患者在血液缓解率或疾病进展率方面没有差异。额外的非MPN驱动突变(NDM)对HC-RES/INT ET患者并发症风险的影响尚不清楚。


鉴于NDM的存在可能会影响试验结果，现研究人员通过靶向髓系32基因测序将MAJIC-ET的主要分析扩展到对MAJIC-ET患者的NDM进行连续评估。


起始时，在30%的患者中检测到NDM，最常见的突变基因有TET2（11%）、TP53（6.4%）和SF3B1（6.4%）。NDM与4年无转化存活率差相关（TFS： 65.4% vs 82.8%）。特别是Tp53和剪切因子（SF、SF3B1、ZRSR2）与TFS降低有关，但TET2突变与其无关，鲁索替尼治疗也不能改善携带这些突变的患者的TFS。纵向分析发现19.3%的患者有新发突变，主要影响TET2、TP53、SF3B1。


综上所述，本研究首次报道了HC-RES/INT ET患者的综合突变分析，并强调了HC-RES/INT ET中NDM突变系列分析的临床/预后实用性，包括SF和TP53突变的重要性，依据这些突变可识别出具有高疾病转化风险的HC-RES/INT ET患者。


原始出处：


Jennifer M O'Sullivan， et al. The poor outcome in high molecular risk， hydroxycarbamide resistant/intolerant ET is not ameliorated by ruxolitinib. Blood 2019 ：blood.2019001861； doi： https：//doi.org/10.1182/blood.2019001861


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    2019-10-07 Smile2681

    #耐受性#

    0

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    2019-10-07 12498adam41暂无昵称

    #羟基脲#

    0

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    2019-10-07 caoliehu

    #预后不良#

    0