唐氏综合征和白血病:了解疾病生物学和当前的治疗选择
Sonali P Barwe1, E Anders Kolb1, Anilkumar Gopalakrishnapillai1
1Lisa Dean Moseley Institute for Cancer and Blood Disorders, Nemours Children's Health, Wilmington, Delaware, 19803, USA.
Blood reviews
|November 28, 2023
概括
患有唐氏综合征 (DS) 的儿童患白血病的风险更高. 这篇评论涵盖了当前的治疗方法,化学疗法毒性等挑战,以及DS白血病的新分子标,为更好的结果提供了希望.
科学领域:
- 儿科瘤学 儿科瘤学
- 遗传学 遗传学 是一个
- 血液学 血液学 血液学
背景情况:
- 患有唐氏综合征 (DS) 的儿童患急性白血病的风险显著增加 (10-20倍),主要是髓性白血病 (ML-DS).
- 虽然ML-DS通常有良好的预后,但复发病例与糟糕的结果有关.
- 与非DS ALL相比,DS (DS-ALL) 中的急性淋巴细胞白血病预后不佳,复发率高.
研究的目的:
- 对唐氏综合征儿童白血病的当前治疗策略进行审查.
- 讨论在DS中白血病发病和进展的机制.
- 突出新型分子标,提供对DS白血病有前途的临床前数据.
主要方法:
- 对DS白血病当前治疗策略和结果的文献综述.
- 对DS白血病的突变谱和病理机制研究的分析.
- 对新型分子标的临床前数据的检查.
主要成果:
- 对DS白血病突变格局的理解得到了改善.
- 动物,胚胎干细胞和诱导多能干细胞模型已经阐明了疾病机制.
- 与化疗相关的毒性在治疗DS白血病方面带来了重大挑战.
结论:
- 尽管面临挑战,但了解DS白血病的进展正在为针对性治疗铺平道路.
- 新型分子标在临床前环境中表现有前途,并可能转化为改善临床结果.
- 针对性治疗的进一步研究对于改善DS白血病的预后至关重要,特别是在复发或高风险病例中.
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