在患有异常HOX/MEIS1表达异常的儿科急性髓性白血病的治疗向
Kristian L Juul-Dam1, Neerav N Shukla2, Todd M Cooper3
1Department of Pediatrics and Adolescent Medicine, Aarhus University Hospital, Aarhus, Denmark.
European journal of medical genetics
|January 4, 2024
概括
针对性治疗,如脑膜抑制剂,对具有特定基因变异的儿童急性髓性白血病 (AML) 是有前途的. 这些药物破坏关键的蛋白质复合体,导致白血病细胞死亡,为难以治疗的病例提供了新的希望.
科学领域:
- 儿科瘤学 儿科瘤学
- 分子生物学分子生物学
- 癌症遗传学 癌症遗传学
背景情况:
- 儿童急性髓性白血病 (AML) 在约三分之一的患者中仍然是致命的,原因是化学抵抗,复发和治疗毒性.
- 特定的遗传亚型,包括具有HOXA/HOXB和MEIS1上调的亚型 (在30%的儿科AML中发现),存在治疗挑战.
- 异常的HOXA/MEIS1表达与KMT2A-r,NUP98-r和NPM1c基因型有关,表明分子脆弱性.
结论:
- 梅因抑制剂代表了对特定儿童AML基因型的有希望的向治疗,其特点是HOXA/MEIS1上调.
- 临床试验,例如由PedAL/EUPAL协调的临床试验,对于评估儿科患者的脑膜抑制剂至关重要.
- 结合治疗涉及脑膜抑制剂可能会增强抗白血病效果,并改善儿童AML治疗的安全性.
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