Thomas Xavier
Department of Clinical Hematology, Hospices Civils de Lyon Service d'Hématologie Clinique Centre Hospitalier Lyon Sud, Pierre-Bénite, 69495-cedex, France.
Curr Oncol Rep. 2025 Apr 28. doi: 10.1007/s11912-025-01678-y.
Despite differences in the various classification systems of acute myeloid leukemia (AML), rare entities can be identified according to clinical, biological or morphological characteristics. Uncommon AML defined on specific morphological criteria and/or genetic abnormalities were considered if occurring with a frequency of ≤ 5% in adult patients with AML.
Most of uncommon AML are characterized by a poor outcome with the standard treatment approaches. During the last decade, several therapeutic drugs with promising investigational approaches have been used in therapeutic regimens in both frontline and relapsed/refractory AML and represent a positive potential benefit for some rare entities displaying specific molecular lesions. Several rare subtypes can be identified in adult patients with AML. In this descriptive review, we assess the available information for these rare entities and summarized treatments that could be proposed especially according to their genetic characterization.
尽管急性髓系白血病(AML)的各种分类系统存在差异,但仍可根据临床、生物学或形态学特征识别出罕见类型。如果在成年AML患者中出现频率≤5%,则考虑根据特定形态学标准和/或基因异常定义的不常见AML。
大多数不常见AML的标准治疗方案预后较差。在过去十年中,几种具有前景的研究性治疗药物已被用于一线和复发/难治性AML的治疗方案中,对于一些表现出特定分子病变的罕见类型具有积极的潜在益处。成年AML患者中可识别出几种罕见亚型。在本描述性综述中,我们评估了这些罕见类型的现有信息,并总结了根据其基因特征可提出的治疗方法。