ALL-BFM95方案治疗儿童急性淋巴细胞白血病高危患儿的预后分析

目的 研究ALL-BFM 95 方案在治疗中国儿童急性淋巴细胞白血病(ALL)高危患儿的疗效及可行性。方法 回顾性分析2003 年7 月至2013 年8 月按ALL-BFM 95 方案进行化疗的47 例初治ALL 高危患儿的临床资料,其中单纯行化疗患儿27 例,化疗联合造血干细胞移植(HSCT)患儿20 例,采用Kaplan-Meier 法进行生存分析。结果 47 例患儿中,复发后死亡12 例(26%),治疗相关死亡5 例(11%),5 年预计无事件生存率(pEFS)为62%。单纯化疗患儿5 年pEFS 为52%,化疗联合HSCT 患儿5 年pEFS 为77%,差异有统计学意义(P=0.035);单纯强的松反应差的高危ALL 患儿5 年pEFS(80%)显著高于化疗后第15 天(60%)及第33 天(0)骨髓M3 状态的高危ALL 患儿(P<0.05)。结论 ALL-BFM 95 方案在治疗高危儿童ALL 中有良好的临床疗效,但复发仍是ALL 高危患儿死亡的主要原因;化疗联合HSCT 对高危ALL 患儿的疗效优于行单纯化疗;化疗后第15 天及第33 天骨髓M3 状态的高危ALL 患儿预后较差。

Outcome of childhood high-risk acute lymphoblastic leukemia treated with the ALLBFM 95 protocol

Objective To evaluate the effectiveness and the practicability of the Acute Lymphoblastic Leukemia Berlin-Frankfurt-Münster 95 (ALL-BFM 95) protocol in treating childhood high-risk acute lymphoblastic leukemia (HR-ALL). Methods A retrospective analysis of 47 children with newly diagnosed HR-ALL between July 2003 and August 2013 was performed. These children were treated by the ALL-BFM 95 protocol. Survival was evaluated by Kaplan Meier analysis and Log-Rank test. Results Relapse-related death occurred in 12 of 47 patients (26%), and 5 of 47 patients (11%) were treatment-related mortality. Five-year probability of event-free-survival (pEFS) was 62%. Children with hematopoietic stem cell transplantation (HSCT) after chemotherapy achieved significantly better pEFS than those with chemotherapy alone (77% vs 52%; P=0.035). The patients who were only poor responders to prednisone had a better outcome (5-year pEFS 80%) than the Days 15 and 33 bone marrow M3 subgroups (5- year pEFS 60% and 0 respectively). Conclusions ALL-BFM 95 protocol can improve the outcome of children with high-risk ALL. The major cause of death is attributed to relapse. Chemotherapy plus HSCT can produce a better outcome than chemotherapy alone. The Days 15 and 33 bone marrow M3 subgroups have a poor prognosis.