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在白血病中进行血造干细胞移植

Caroline Sevin¹, Fanny Mochel²

¹AP-HP, Kremlin-Bicêtre University Hospital, Department of Neuropediatrics, Reference Center for Pediatric Leukodystrophies, Paris, France; INSERM U 1127, CNRS UMR 7225, Sorbonne Université, UPMC Univ Paris 06 UMR S 1127, Institut du Cerveau, ICM, Paris, France.

Handbook of clinical neurology

|September 25, 2024

概括

造血干细胞移植 (HSCT) 通过恢复微质功能,为白血病提供治疗效益,并且如果早期进行,可能会改善结果. 基因疗法是某些疾病的新兴替代方案.

科学领域:

神经学神经学
遗传学遗传学是一个
免疫学免疫学免疫学

背景情况:

白血病是影响髓的遗传性疾病,已经描述了50多种类型.
神经炎症是某些白血病的关键特征,影响疾病的进展.
造血干细胞移植 (HSCT) 通过解决髓缺陷和神经炎症,已经显示出治疗潜力.

研究的目的:

探索HSCT用于频繁白血病的理由,指示和结果.
讨论HSCT在大脑上白细胞缩 (CALD),超色白细胞缩 (MLD),克拉贝病 (KD) 和具有轴球体和色素质细胞 (ALSP) 的成人发病白脑病变中的作用.
审查CALD和MLD新兴的ex vivo基因疗法选择.

主要方法:

对针对特定白血病的HSCT现有文献的综述.
分析HSCT对疾病自然史和中枢神经系统 (CNS) 缺陷的影响.
讨论ex vivo基因疗法的原则和潜力.

主要成果:

HSCT可以修改CALD,MLD,KD和ALSP的自然史.
在改善中枢神经系统相关的缺陷方面,高血压神经治疗的早期干预至关重要.
HSCT恢复正常的微质功能,可能减少神经炎症.

结论:

对于精选的白血病,HSCT是一种可行的治疗选择,特别是在早期进行时.
活体基因疗法对CALD和MLD呈现出异构HSCT的一个有希望的替代方案.
了解神经炎症的作用是优化白血病中HSCT结果的关键.

关键词:

血造干细胞基因疗法血造干细胞基因疗法造血干细胞移植造血干细胞移植在白血病中,白血病缩症 (leukodystrophy) 是一种疾病.微质细胞中的微质细胞神经系统疾病神经系统疾病

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