CELL INTELLIGENCE · 肿瘤细胞治疗研究
肿瘤细胞治疗研究
英文原题:A Review of Childhood Acute Myeloid Leukemia: Diagnosis and Novel Treatment.
A Review of Childhood Acute Myeloid Leukemia: Diagnosis and Novel Treatment.
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急性髓系白血病(AML)是儿童中第二常见的血液系统恶性肿瘤。儿童AML发病率远低于急性淋巴细胞白血病(ALL),因此属于儿童罕见病。遗传异常在AML分类、管理及预后预测中的作用比以往更加重要。世界卫生组织(WHO)、国际共识分类(ICC)及欧洲白血病网(ELN)等疾病分类和风险分组标准已于2022年修订,这些新信息将在未来数年逐步应用于儿童AML。本综述强调儿童AML中已知的重要遗传亚型,包括核心结合因子AML(CBF AML)、KMT2A重排AML(KMT2A/11q23重排)、伴体细胞突变的正常核型AML、不平衡细胞遗传学异常AML、NUP98 11p15/NUP09重排AML,以及急性早幼粒细胞白血病(APL);成人与儿童AML中各类遗传异常的发生频率有所不同。本文还回顾当前风险分层、儿童AML管理流程,以及靶向治疗、免疫治疗和嵌合抗原受体(CAR)T细胞疗法等新型治疗方式,最后讨论AML造血干细胞移植(HSCT)的适应证。
Acute myeloid leukemia (AML) is the second most common hematologic malignancy in children. The incidence of childhood AML is much lower than acute lymphoblastic leukemia (ALL), which makes childhood AML a rare disease in children. The role of genetic abnormalities in AML classification, management, and prognosis prediction is much more important than before. Disease classifications and risk group classifications, such as the WHO classification, the international consensus classification (ICC), and the European LeukemiaNet (ELN) classification, were revised in 2022. The application of the new information in childhood AML will be upcoming in the next few years.
The frequency of each genetic abnormality in adult and childhood AML is different; therefore, in this review, we emphasize well-known genetic subtypes in childhood AML, including core-binding factor AML (CBF AML), KMT2Ar ( KMT2A /11q23 rearrangement) AML, normal karyotype AML with somatic mutations, unbalanced cytogenetic abnormalities AML, NUP98 11p15/NUP09 rearrangement AML, and acute promyelocytic leukemia (APL).
Current risk group classification, the management algorithm in childhood AML, and novel treatment modalities such as targeted therapy, immune therapy, and chimeric antigen receptor (CAR) T-cell therapy are reviewed.
Finally, the indications of hematopoietic stem cell transplantation (HSCT) in AML are discussed.
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