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一个新的预后风险模型,用于接受venetoclax加上低甲基化剂的耐药/复发性急性髓性白血病患者

Rabia Shahswar¹, Razif Gabdoulline¹, Katja Krueger¹

¹Department of Hematology, Hemostasis, Oncology, and Stem Cell Transplantation, Hannover Medical School, Hannover, Germany.

|January 8, 2025

概括

一个新的风险评分,VEN-PRS,有助于预测治疗低甲基化剂和venetoclax的复发/耐药AML患者的结果. 这一分数确定了有利,中等和不利的风险组,以指导治疗选择.

科学领域:

血液学血液学血液学
在瘤学瘤学.
遗传学遗传学是一个

背景情况:

复发性和耐药性急性髓性白血病 (R/R AML) 是一个重大的临床挑战.
低甲基化剂和venetoclax (HMA/VEN) 经常用于R/R AML的非标签,但结果预测因素仍然不清楚.

研究的目的:

开发和验证用HMA/VEN.治疗R/RAML患者的预后风险评分.
确定与这种患者群体的生存相关的临床和分子因素.

主要方法:

对240名成年R/RAML患者进行了回顾性观察性多中心研究.
多变量分析以确定生存的风险因素.
构建和验证一个三层风险模型 (VEN-PRS).

主要成果:

在VEN-PRS模型中,患者被分为有利的,中间的和不良的风险组,其总生存时间显著不同 (mOS分别为21.4个月,7.5个月和4.6个月).
关键的不良预后因素包括外骨髓性疾病,HMA预治疗和NF1,PTPN11,FLT3和TP53.3中的突变.
突变的SF3B1被确定为一个有利的预后因素.

结论:

开发的VEN-PRS是一种经过验证的临床分子工具,用于预测R/RAML的HMA/VEN治疗结果.
这种风险评分可以帮助选择高风险R/RAML患者的适当治疗策略.

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