Shimada Akira
Department of Pediatric Hematology/Oncology, Okayama University Hospital.
Rinsho Ketsueki. 2017;58(8):983-990. doi: 10.11406/rinketsu.58.983.
Annually, it is estimated that approximately 150-200 children aged 0-16 years are diagnosed with acute myeloid leukemia (AML). In Japan, clinical studies with ANLL91, AML99, CCLSG-AML9805, and JPLSG-AML05 protocols were performed historically, and the risk stratification with a combination of chemotherapy and hematopoietic stem cell transplantation resulted in the improvement of clinical outcomes. Regarding the onset of pediatric AML at the molecular level, mutations in FLT3-ITD or KIT (Class I mutation) showed a poor prognosis, but the ratio of mutations in Class III-V genes was smaller than that in adult AML. In contrast, several pediatric AML cases are complicated due to chromosome fragility syndrome or congenital bone marrow failure syndrome. To improve the clinical outcomes, clinical application of next generation sequencing may allow for personalized therapy in each patient in the future.
据估计，每年约有150 - 200名0至16岁的儿童被诊断出患有急性髓系白血病（AML）。在日本，过去曾开展过针对ANLL91、AML99、CCLSG - AML9805和JPLSG - AML05方案的临床研究，化疗与造血干细胞移植相结合的风险分层使临床疗效得到了改善。关于儿童AML在分子水平上的发病情况，FLT3 - ITD或KIT突变（I类突变）预后较差，但III - V类基因突变的比例低于成人AML。相反，一些儿童AML病例因染色体脆性综合征或先天性骨髓衰竭综合征而病情复杂。为了改善临床疗效，下一代测序技术的临床应用未来可能会实现对每位患者的个性化治疗。
