Objective: To evaluate the therapeutic efficacy and safety of immunosuppressive therapy (IST) combined with recombinant human thrombopoietin (rhTPO) for severe aplastic anemia (SAA) in pediatric patients. Method: A retrospective case-control study was conducted and the clinical data of 45 pediatric patients with de novo SAA admitted to the Anemia Diagnosis and Treatment Center of Chinese Academy of Medical Sciences & Blood Disease Hospital during the period from December 2009 to December 2014 were analyzed. Among them, 15 patients were treated with the regimen of IST together with rhTPO and 30 patients were given IST treatment only. The variation characteristics of the peripheral blood routine as well as the transfusion of blood products was dynamically observed, and the therapeutic efficacy was assessed respectively after 3, 6 and 12 months after the treatment. In the meantime, adverse effects related to rhTPO application were recorded. Thereafter, the statistics of the two groups were compared by non-parametric rank sum test. Result: Among 45 pediatric patients, there were 26 male and 19 female, and the median age was 11 years (6-14). The number of patients received good hematological response(complete remission (CR) plus good partial response (GPR)) in the combinatory group versus vs. the IST group was 6 vs. 3 patients (χ(2)=3.906, P=0.048) at the 3rd month, 7 vs. 7 patients (χ(2)=1.568, P=0.210) at the 6th month, and 13 vs. 14 patients (χ(2)=6.667, P=0.01) at the 12th month respectively. For those achieved good hematological response at the 3rd month, the amount of platelets transfusion and red blood cells transfusion of the combined group were both less than that of the IST group during the period from the 10th to the 12th weeks (platelets transfusion: 1.4 U vs. 2.9 U, t=-3.523, P=0.002; red blood cells transfusion: 0.8 U vs. 2.6 U, t=-2.392, P=0.026). No serious adverse effect related to rhTPO application was observed in the IST combined with rhTPO group. Conclusion: Application of rhTPO can improve the short-term therapeutic efficacy of IST for pediatric SAA, alleviate transfusion dependence, and has a good safety profile.
目的： 评价联合应用免疫抑制治疗(IST)和重组人血小板生成素(rhTPO)对儿童重型再生障碍性贫血(SAA)的效果和安全性。 方法： 回顾性病例对照研究，分析2009年12月至2014年12月中国医学科学院血液病医院贫血诊疗中心收治的45例SAA患儿资料，其中接受IST联合rhTPO方案者15例，单纯IST方案者30例，动态观察治疗后3、6、12个月的外周血常规变化和血制品输注情况并评价疗效，记录应用rhTPO患儿的不良反应，采用非参数秩和检验法对比两组资料。 结果： 45例患儿中男26例，女19例，中位诊断年龄11(10～13)岁，rhTPO联合IST组和IST组在3、6、12个月时获得良好血液学反应[完全缓解(CR)＋良好部分治疗反应(GPR)]的例数分别为6、7、13例和3、7、14例(χ(2)＝3.906、1.568、6.667, P＝0.048、0.210、0.010)。3个月时有效者，rhTPO联合IST组第10～12周血小板和红细胞的输注量均较IST组少(血小板1.4 U比2.9 U, t＝－3.523，P＝0.002；红细胞0.8 U比2.6 U，t＝－2.392，P＝0.026)，应用rhTPO患儿未发生相关严重不良反应。 结论： 应用rhTPO可以提高SAA患儿IST治疗的近期疗效，减轻输血依赖，其安全性较好。.
Keywords: Anemia, aplastic; Child; Immunosuppression; Thrombopoiesis.