Taga Takashi
Department of Pediatrics, Shiga University of Medical Science.
Rinsho Ketsueki. 2020;61(8):971-978. doi: 10.11406/rinketsu.61.971.
The event-free survival rate for childhood acute myeloid leukemia (AML) is approaching 60%, owing to the use of intensive chemotherapy, optimal indication of hematopoietic stem cell transplantation, and advances in supportive care. For further improvement, the development of more definitive risk stratification system and introduction of more effective treatment options are necessary. Recently, much attention has been drawn on minimal residual disease (MRD) that is considered a strong prognostic factor for children with AML. Recent studies from the United States and Europe have shown the prognostic impact of FCM-based MRD detection and it is already used for risk stratification in modern AML protocols. Myeloid leukemia in Down's syndrome (ML-DS) has unique characteristics, and ML-DS children are recently being treated separately from non-DS AML children with less intensive treatment. It has been shown that relapsed and refractory cases of ML-DS are difficult to treat; however, no universal prognostic factor has been found yet. In order to determine accurate methods for identifying a subgroup with poor prognosis, an attempt to analyze the role of MRD had been examined in the JPLSG AML-D11 study. In this study, the MRD by FCM and targeted deep sequencing for GATA1 after initial induction therapy were significant prognosis factors for predicting relapse.
由于采用了强化化疗、优化造血干细胞移植指征以及支持治疗方面的进展，儿童急性髓系白血病（AML）的无事件生存率接近60%。为了进一步提高生存率，有必要开发更明确的风险分层系统并引入更有效的治疗方案。最近，微小残留病（MRD）备受关注，它被认为是儿童AML的一个强有力的预后因素。美国和欧洲最近的研究表明了基于流式细胞术（FCM）的MRD检测对预后的影响，并且它已被用于现代AML方案的风险分层。唐氏综合征髓系白血病（ML-DS）具有独特的特征，最近ML-DS儿童与非DS AML儿童分开治疗，治疗强度较低。已表明ML-DS的复发和难治病例难以治疗；然而，尚未发现通用的预后因素。为了确定识别预后不良亚组的准确方法，在JPLSG AML-D11研究中尝试分析了MRD的作用。在这项研究中，初始诱导治疗后通过FCM检测的MRD以及针对GATA1的靶向深度测序是预测复发的重要预后因素。
