儿童急性早幼粒细胞白血病异常克隆与疗效研究  

Clinical study on therapeutic efficacy of acute promyelocytic leukemia children with abnormal clone

作　　者：李红[1,2] 蒋慧[1,2] 邵静波[1,2] 陆正华[1,2] 杨为群[1,2] 杨静薇[1,2] 张娜[1,2] 孟琳懿[1,2] 

机构地区：[1]上海市儿童医院 [2]上海交通大学附属儿童医院血液科,上海200040

出　　处：《临床血液学杂志》2012年第3期302-305,共4页Journal of Clinical Hematology

基　　金：上海交通大学医学院科技基金项目(No:JYY0914)

摘　　要：目的:回顾性评价不同治疗方法对儿童急性早幼粒细胞性白血病(APL)异常融合基因的临床意义,并评估疗效。方法:我院确诊初发APL患儿30例,男16例,女14例。根据不同时间采用不同治疗方案分成A、B两组。A组(1990—2000年):16例患儿以ATRA加化疗方案为主方案治疗。B组(2001—2010年):14例患儿以ATRA加As2O3加化疗为主方案治疗。治疗前PML/RARa融合基因阳性23例,其中A组12例,B组11例。染色体t(15;17)17例,其中A组10例,B组7例。根据临床表现、实验室检查与动态分子生物学分析,评价疗效。结果:A、B两组患儿诱导后均完全缓解。A组平均缓解时间(32.3±7.2)d,B组(25.9±8.3)d(P<0.05);A组PML/RARa融合基因平均第1次转阴时间(13.6±10.6)个月,B组(3.2±1.7)个月(P<0.01)。A组PML/RARa融合基因复转阳5/12例(41.7%),B组未见复转阳病例(P=0.037)。A组11/16例(68.8%)无病生存,5/16例(31.2%)死亡,B组所有14例患儿无病生存至今(P=0.045)。A组7/16例(43.8%)复发,复发中位时间27(4.5～51.0)个月;B组复发1/14例(7.1%)(P=0.039)。两组患儿心肝肾功能损害及维甲酸不良反应比较差异无统计学意义,B组患儿骨髓抑制发生率明显少于A组(P<0.01)。结论ATRA联合As2O3治疗组缩短临床缓解时间及PML/RARa融合基因首次转阴时间,持续下调融合基因,同时减少化疗相关死亡、降低复发率。ATRA和AS2O3为主方案治疗APL安全、有效,适用于儿童。Objective：Retrospective evaluations of two different chemotherapy regimens in children with acute promyelocytic leukemia（APL）were made to assess clinical significance of abnormal fusion gene and observe the therapeutic efficacy as while. Method：Thirty cases of newly diagnosed APL patients,including 16 males and 14 females,from 1990 to 2010 were divided into two groups-A and B,according to different chemotherapy regimens at different time.Sixteen children in group A diagnosed from 1990 to 2000 were treated with ARTA and chemotherapy,and fourteen children in group B diagnosed from 2001 to 2010 were treated with the main regimen consist of ATRA,As203 and chemotherapy.23 children expressed PML /RARa fusion gene positive with 12 children in group A and 11 children in group B.Statistics analysis was made on the SPSS13.0. Result：Both groups gained 100% CR rate.The CR time of group A was（32.3±7.2）days compared with（25.9±8.3）days of group B（P0.05）.The median time that PML/RARa fusion gene switched to negative in group A was（13.6±10.6）months compared with（3.2±1.7）months of group B（P0.01）.68.8 % Children in group A gained disease-free survival,and 31.2% died.All 14 children in group B were still disease-free survival（P0.05）.The relapse rate of group A was higher than group B,P=0.039.There was no significant difference in heart,liver and renal toxicity between two groups（P0.05）.But children in group A were much more likely to gain bone marrow suppression（P0.01）. Conclusion：The regimen combined ATRA with As2O3 could shorten CR time and the time of PML/RARa fusion gene switching to negative,reduce relapse rate and chemotherapy-related deaths.This regimen is safe and effective for children with APL.

关 键 词：白血病 早幼粒细胞性 急性 全反式维甲酸 三氧化二砷 儿童 

分 类 号：R733.71[医药卫生—肿瘤]