[Allogeneic transplantation as a therapeutic option for atypical chronic myeloid leukemia].

Hidehiro Itonaga
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Abstract

Atypical chronic myeloid leukemia (aCML) is a rare disease classified as a myelodysplastic/myeloproliferative neoplasm (MDS/MPN). Recent advances in gene mutational profiling have clarified the characteristics of aCML as a disease entity relative to other MDS/MPNs. Although some studies suggest the efficacy of DNA demethylating agents and tyrosine kinase inhibitors, data about these agents are limited due to the small number of patients. Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is only therapeutic option that can provide durable remission for aCML and other MDS/MPNs. Retrospective studies from Europe and Japan revealed the clinical results of allo-HSCT for aCML. This review summarizes the pathogenesis of aCML and the development of allo-HSCT and other therapeutic options.

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[异基因移植作为非典型慢性髓性白血病的一种治疗方案]。
非典型慢性髓性白血病(aCML)是一种罕见疾病,被归类为骨髓增生异常/骨髓增生性肿瘤(MDS/MPN)。基因突变分析的最新进展阐明了 aCML 作为一种疾病实体相对于其他 MDS/MPN 的特征。尽管一些研究表明 DNA 去甲基化药物和酪氨酸激酶抑制剂具有疗效,但由于患者人数较少,有关这些药物的数据十分有限。异基因造血干细胞移植(allo-HSCT)是唯一能为 aCML 和其他 MDS/MPNs 带来持久缓解的治疗方案。欧洲和日本的回顾性研究揭示了异基因造血干细胞移植治疗 aCML 的临床效果。本综述概述了 aCML 的发病机制以及 allo-HSCT 和其他治疗方案的发展。
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