治疗三个小儿AML共表达NUP98-NSD1,FLT3-ITD和WT1的治疗方法
Li Liu1,2, Qi Nie3, Zugang Xiao4
1Department of Hematology, The Affiliated Children's Hospital of Kunming Medical University, Kunming Medical University, Kunming, China.
BMC pediatrics
|July 27, 2024
概括
患有NUP98-NSD1,FLT3-ITD和WT1突变的儿科急性髓性白血病 (AML) 显示化学疗法反应不佳. 血造干细胞移植 (Haplo-HSCT) 可能会改善这些高风险AML患者的治疗结果.
科学领域:
- 血液学 血液学 血液学
- 儿科瘤学 儿科瘤学
- 分子遗传学 分子遗传学
背景情况:
- 急性髓性白血病 (AML) 是一种异质性癌症.
- 识别高危基因突变对于治疗分层至关重要.
- NUP98-NSD1,FLT3-ITD和WT1突变的共同表达代表了儿童罕见且激烈的AML亚型.
研究的目的:
- 调查患有同时存在NUP98-NSD1,FLT3-ITD和WT1突变的儿科AML患者的临床过程和治疗反应.
- 评估标准化疗和替代治疗在这种特定的AML亚组中的疗效.
主要方法:
- 在2020-2023年期间诊断出三名儿科AML患者的回顾性分析.
- 使用下一代测序 (NGS) 和定量聚合酶链反应 (qPCR) 检测高风险突变的骨髓查.
- 根据中国儿童白血病组 (CCLG) -AML-2019协议进行治疗,包括诱导化疗和救援方案.
主要成果:
- 这三名患者都呈现了NUP98外因子12-NSD1外因子6融合,FLT3-ITD和WT1突变.
- 标准诱导 (DAH/IAH) 和复发性/耐药性 (C+HAG) 化疗方案未能达到缓解.
- 一名患者在Haploidentical Hematopoietic干细胞移植 (Haplo-HSCT) 后实现了完整的分子缓解,而两名未移植的患者因疾病而死亡.
结论:
- 患有共同表达NUP98-NSD1,FLT3-ITD和WT1突变的儿科AML患者对常规化疗有抗性.
- 哈普洛同一性造血干细胞移植 (Haplo-HSCT),可能有量身定制的移植前和移植后策略,为这个高危人群提供了一个有前途的治疗途径.
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