SCMC APL-2010方案治疗44例儿童急性早幼粒细胞白血病的临床总结

Clinical effect of the SCMC APL-2010 regimen in treatment of acute promyelocytic leukemia in children: an analysis of 44 cases

目的 评价SCMC APL-2010方案治疗儿童急性早幼粒细胞白血病(APL)的疗效.方法 收集2010年4月至2016年7月采用SCMC APL-2010方案治疗的44例APL患儿的临床资料,采用Kaplan-Meier生存分析评估患儿的无事件生存(EFS)率和总生存(OS)率.结果 44例APL患儿中,42例(95%)经1个疗程达完全缓解(CR),1例经2个疗程达CR,总CR率为98%,9年EFS率为96%±3%,OS率为97.7%±2.2%.不良事件中,发生感染41例(93%),粒细胞减少29例(66%),分化综合征12例(27%,其中死亡1例),肝功能异常16例(36%),胃肠道不良反应12例(27%),QT间期延长7例(16%),睾丸炎1例(2%),无第二肿瘤发生.结论 SCMC APL-2010方案治疗儿童APL预后好,可获得长期生存,但治疗相关感染较多.

Objective To study the clinical effect of the SCMC APL-2010 regimen in the treatment of acute promyelocytic leukemia(APL)in children.Methods A retrospective analysis was performed for the clinical data of 44 children with APL who received treatment with the SCMC APL-2010 regimen between April 2010 and July 2016.The Kaplan-Meier survival analysis was used to evaluate event-free survival(EFS)rate and overall survival(OS)rate.Results Of the 44 children with APL,42(95%)achieved a complete remission(CR)after one course of treatment and 1 achieved CR after two courses of treatment,with an overall CR rate of 98%.The 9-year EFS and OS rates were 96%±3%and 97.7%±2.2%respectively.As for adverse events,41(93%)had infection,29(66%)had granulocyte reduction,12(27%,1 died)had differentiation syndrome,16(36%)had liver dysfunction,12(27%)had adverse gastrointestinal reactions,and 7(16%)had QT prolongation,1(2%)had orchitis,and no secondary neoplasm was observed.Conclusions Children with APL receiving the SCMC APL-2010 regimen have a good prognosis and can achieve a long-term survival,while treatment-related infection is commonly seen.