环孢素A治疗126例儿童非重型再生障碍性贫血疗效分析

Efficacy of cyclosporine A on treatment of 126 children with non-severe aplastic anemia

目的评价环孢素A（CSA）治疗儿童非重型再生障碍性贫血（NSAA）疗效。方法回顾性分析2005年1月至2014年6月径CSA口服治疗的126例NSAA患儿的临床资料。结果 126例患儿中男76例、女50例,中位年龄7岁11月（1岁11个月-14岁）,中位随访时间14.5个月（3-79个月）;非输注依赖型NSAA78例（61.9%）,输注依赖型NSAA48例（38.1%）。总有效率55.6%;CSA治疗输注依赖型NSAA有效率77.1%,非输注依赖型NSAA有效率42.3%,差异有统计学意义（χ^2=15.83,P=0.000）。CSA治疗后,14.1%非输注依赖型NSAA病例完全缓解,80.8%维持非输注依赖NSAA,5.2%进展为输注依赖NSAA或重型/极重型再生障碍性贫血（SAA/VSAA）;16.7%输注依赖型NSAA患儿完全缓解,60.4%好转为非输注依赖,23.0%维持输注依赖型NSAA或进展为SAA/VSAA。结论 CSA治疗可以延缓NSAA患儿的疾病进展,但CSA治疗完全缓解率低,尚需更多临床试验建立更有效的NSAA治疗方案。

Objective To evaluate the efficacy of cyclosporine A（CSA） in children with non-severe aplastic anemia（NSAA）. Methods A retrospective analysis of clinical data of 126 children with NSAA treated with CSA in our hospital between January 2005 and June 2014 was performed. Results Of the 126 cases, 76 were male, and 50 were female, with a median age of 7 years and 11 months old（1 year and 11 months-14 years old）, respectively. The median follow-up time was 14.5 months（3-79 months）. There were 78（61.9%） cases of transfusion-independent NSAA, and 48（38.1%） cases of transfusion-dependent NSAA. After receiving CSA treatment, 14.1% patients with transfusion-independent NSAA achieved complete remission, 80.8% cases kept transfusion-independent NSAA, and 5.2% children progressed to transfusion-dependent NSAA or severe aplastic anemia/very severe aplastic anemia（SAA/VSAA）. The response rate of CSA was 42.3%. 16.7% cases with transfusion-dependent NSAA achieved complete remission, 60.4% patients improved to transfusion-independent NSAA, and 23% children maintained transfusion-dependent NSAA or progressed to SAA/VSAA. The response rate of CSA in patients with transfusion-dependent NSAA was 77.1%. The overall response rate of CSA in 126 NSAA patients was 55.6%. Conclusions CSA treatment can markedly reduce the probability of progression in children with NSAA. The patients can obtain benefits from early treatment of CSA. However, the complete remission rate of CSA was low, further clinical trials are necessary to establish more effective regimens for NSAA.