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

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。

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

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.