儿童急性早幼粒细胞白血病76例随访报告

目的 探讨儿童急性早幼粒细胞白血病(APL)的治疗及预后,并评估砷剂在儿童APL中的疗效.方法 对76例初治APL患儿进行疗效及预后相关因素分析.采用SPSS10.0软件进行统计学分析.结果 本组76例APL患儿,6例早期死亡.其余70例患儿依据诱导方案的不同分成3组:1组44例,单用全反式维甲酸(ATRA);2组7例,单用三氧化二砷(As_2O_3);3组19例,联合应用ATRA和As_2O_3.1组的完全缓解率为100%,2组+3组的完全缓解率为100%.6例临床复发,2例分子生物学复发,复发部位均为骨髓.5年累计复发率为13.8%,5年累积无事件生存(EFS)率、无病生存(DFS)率、总生存(OS)率分别为79.5%、86.3%和90.5%.在1组与2组+3组之间的5年EFS、DFS没有明显差异.初诊时白细胞计数可能是影响儿童预后最主要的因素.结论 ATRA治疗儿童APL疗效好.砷剂治疗儿童APL的疗效亦较好,耐受性好.砷剂可以用于不能耐受ATRA副作用的患儿,亦可用于初治和复发的儿童APL.

Retrospective analysis of 76 children with acute promyelocytic leukemia

Objective There are very limited data on childhood acute promyelocytic leukemia (APL), especially childhood APL treated with arsenic trioxide (As_2O_3). Method Between January 1999 and December 2007,76 children (< 18 years) with newly diagnosed APL were included. Results Six eases failed to achieve hematological complete remission (CR) due to early death. Three groups of patients were identified according to the different induction regimen. Forty-four patients were given all-trans retinoic acid (ATRA) alone (groupl, G1), 7 patients were given As_2O_3 alone (group 2, G2), 19 patients(group 3, G3) were treated with combination of ATRA and As_2O_3. The CR rate of G1 was 100%. The overall CR rate of G2+G3 was 100%. Five children underwent hematologic relapse and 2 developed molecular relapse. The 5-year cumulative incidence of relapse was 13.8%, whereas event-free (EFS), disease-free (DFS) and overall survival rates were 79.5%, 86.3% and 90.5%, respectively. The 5-year estimate of EFS and DFS between G1 and G2+G3 had no significant difference. Conclusion As_2O_3 is an effective and well tolerable therapy for children with APL and it may be used in those who not only cannot bear side effects of ATRA but also the newly diagnosed and relapsed APL.