Zhou K, Liu C X, Li Y, Li J P, Fan H H, Zhang L, Jing L P, Peng G X, Ye L, Li Y, Song L, Zhao X, Yang W R, Wu Z J, Chen F, Zhang F K
Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Tianjin 300020, China.
Zhonghua Er Ke Za Zhi. 2017 Jul 2;55(7):523-528. doi: 10.3760/cma.j.issn.0578-1310.2017.07.011.
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. 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. 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 . the IST group was 6 . 3 patients (χ(2)=3.906, =0.048) at the 3rd month, 7 . 7 patients (χ(2)=1.568, =0.210) at the 6th month, and 13 . 14 patients (χ(2)=6.667, =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 . 2.9 U, =-3.523, =0.002; red blood cells transfusion: 0.8 U . 2.6 U, =-2.392, =0.026). No serious adverse effect related to rhTPO application was observed in the IST combined with rhTPO group. Application of rhTPO can improve the short-term therapeutic efficacy of IST for pediatric SAA, alleviate transfusion dependence, and has a good safety profile.
评估免疫抑制治疗（IST）联合重组人血小板生成素（rhTPO）治疗小儿重型再生障碍性贫血（SAA）的疗效及安全性。进行一项回顾性病例对照研究，分析2009年12月至2014年12月期间在中国医学科学院血液病医院贫血诊疗中心收治的45例初诊小儿SAA患者的临床资料。其中，15例患者采用IST联合rhTPO方案治疗，30例患者仅给予IST治疗。动态观察外周血常规变化特点及血液制品输注情况，并在治疗后3、6和12个月分别评估疗效。同时，记录rhTPO应用相关的不良反应。之后，采用非参数秩和检验比较两组的统计数据。45例小儿患者中，男26例，女19例，中位年龄为11岁（6 - 14岁）。联合组与IST组在第3个月时获得良好血液学反应（完全缓解（CR）加良好部分缓解（GPR））的患者数分别为6例、3例（χ(2)=3.906，P =0.048），第6个月时分别为7例、7例（χ(2)=1.568，P =0.210），第12个月时分别为13例、14例（χ(2)=6.667，P =0.01）。对于在第3个月获得良好血液学反应的患者，联合组在第10至12周期间的血小板输注量和红细胞输注量均少于IST组（血小板输注量：1.4 U对2.9 U，t = -3.523，P =0.002；红细胞输注量：0.8 U对2.6 U，t = -2.392，P =0.026）。IST联合rhTPO组未观察到与rhTPO应用相关的严重不良反应。rhTPO的应用可提高IST治疗小儿SAA的短期疗效，减轻输血依赖，且安全性良好。
