儿童急性髓系白血病（M4／M5亚型）临床疗效及预后因素分析

Analysis of prognostic factors in children with acute myeloid leukemia （ M4/M5 ）

目的探讨儿童急性髓系白血病M4／M5亚型的临床疗效及预后相关因素。方法回顾性分析2009年1月至2014年12月上海市儿童医院收治的M4／M5亚型患儿临床资料，进行远期疗效、预后及复发因素分析。结果收集符合诊断的46例患儿临床资料，其中38例接受2个疗程以上治疗并纳入统计，其中男22例，女16例；M419例，M519例；中位年龄5岁。5年总体生存（OS）率为（57．7±9．3）％，5年无事件生存（EFS）率为（47．2±8．9）％，M45年EFS率为（52．4±12．7）％，M55年EFS率为（45．4±11．9）％。按国际危险度分层比较：预后良好、预后中等和预后不良组5年EFS率分别为（77．2±12．4）％、（49．5±14．9）％和（25．0±19．8）％，差异有统计学意义（χ2=6．305，P=0．043）。单因素分析显示髓外浸润、染色体核型、诱导治疗第8天骨髓反应评估、治疗疗程为影响预后的因素（χ2=4．828、10．178、5．382、4．771，P=0．028、0．017、0．020、0．029）；多因素分析显示髓外浸润（HR=5．323，95％CI：1．620-17．490，P=0．006）、〈6个疗程治疗（HR=6．186，95％C1：1．726～22．176，P=0．005）是影响生存的独立危险因素。结论1．儿童髓系白血病M4／M5亚型强化疗和足疗程是提高总体疗效的关键。2．髓外浸润是影响生存和导致复发的主要危险因素。3．初始评估有高危因素伴预后不良患儿建议尽早行造血干细胞移植。

Objective To investigate the clinical efficacy and prognostic factors for M4/M5 subtypes in children with acute myeloid leukemia （AML）. Methods A retrospective analysis of the clinical data of M4/M5 subtypes in Shanghai Children＇s Hospital Affiliated to Shanghai Jiaotong University, from January 2009 to December 2014 was carried out. The long - term efficacy, prognosis and relapse factors were analyzed. Results The clinical data of 46 cases were collected, among which 38 cases were treated with more than 2 courses,including 22 male, 16 female, 19 cases M4 and 19 cases M5. The median age was 5 years. 5 - year overall survival （OS） rate and 5 - year event - free survival （EFS） rate were （57.7 ± 9.3 ） % and （47.2 ±8.9 ） % , and 5 - year EFS of M4 and M5 were （52.4 ±12.7 ） % and （45.4 ±11. 9 ）%. Compared with the international risk stratification： 5 - year EFS rate of favorable - risk, intermediate - risk and poor - risk were （77.2 ±12.4） % , （49.5 ±14.9 ） % and （ 25.0 ±19.8 ） % （χ2 = 6. 305, P = 0. 043 ）. Single factor analysis showed that extramedullary infiltration（χ2 = 4. 828, P = 0. 028 ）, Chromosome karyotype （χ2 = 10. 178,P = 0.017 ）, the eighth day assessment （χ2 = 5. 382, P = 0. 020） and course of treatment （χ2 = 4.771, P = 0. 029） were prognostic factors; multivariate analysis showed extramedullary infiltration （ HR = 5. 323,95% CI： 1. 620 - 17. 490 ,P = 0. 006 ） and less - than - 6 courses of treatment （ HR = 6. 186,95% （CI： 1. 726 - 22. 176, P = 0. 005 ） were the independent risk factors of affecting survival. Conclusions （ 1 ） Strengthening treatment and adequate courses of treatment are the critical to improve the overall curative effect in children with M4/M5 subtypes. （ 2 ） Extramedullary infiltration was the risk factor for survival and recurrence in M4/M5 subtypes. （3） It is suggested that the children who have the initial symptoms and molecular biology with poor prognostic factors choose hematopoietic stem cell transplantation as early as possible.