Objective: To evaluate the efficacy and safety of recombinant human thrombopoietin (rhTPO) in treatment of pediatric primary immune thrombocytopenia (ITP).
Methods: The clinical characteristics of 41 pediatric ITP patients who received rhTPO therapy from December 2006 to September 2014 were retrospectively analyzed (as rhTPO group). During the same time another 26 pediatric ITP patients who received vindesine combined with human immunoglobulin therapy were selected as control group. The treatment outcomes were evaluated.
Results: A total of 67 cases of pediatric ITP, 31 males and 36 females with a median age 10.0(1.6-17.0) years were enrolled, including 19 cases of newly disgnosed ITP, 18 cases of persistent ITP and 30 cases of chronic ITP. Of them, 43 cases of whom were severe ITP (PLT<10×10⁹/L). The total response rate had no statistically significant difference between the rhTPO group and the control group (68.29% vs 65.38%, P=0.806), neither in newly ITP, persistent and chronic ITP (P=0.320, P=0.763). In severe ITP patients, 17 of 30 cases (56.67%) achieved response with rhTPO therapy, while the control group was 61.54% (8/13) (P=0.766). The median maximum peak of platelet counts and the time of the platelet counts >30×10⁹/L and > 50×10⁹/L had no statistically significant differences in rhTPO group compared with the control group [52(7-608)×10⁹/L vs 40(3-152)×10⁹/L, P=0.05; 7(3-13) d vs 4(2-24) d, P=0.202; 7.5(4-15) d vs 5.5(4-23) d, P=0.557]. The mean platelet counts were 43(3-605)×10⁹/L in the rhTPO group, which were higher than the control group [32(-14-149)×10⁹/L, P=0.042]. No severe adverse effects were observed in both groups.
Conclusion: For pediatric ITP, rhTPO has a similar outcomes with vindesine combined with human immunoglobulin, and it is an effective and safe treatment option.
目的: 观察重组人血小板生成素（rhTPO）治疗儿童原发免疫性血小板减少症（ITP）的疗效及安全性。
方法: 回顾性分析2006年12月至2014年9月接受rhTPO治疗（rhTPO组）的41例儿童ITP患者临床资料，与同期接受长春地辛联合静脉丙种球蛋白治疗（对照组）的26例ITP患儿进行对比观察。
结果: 全部67例ITP患儿中男31例、女36例，中位年龄10.0(1.6~17.0）岁；新诊断ITP 19例，持续性ITP 18例，慢性ITP 30例；重症ITP(PLT<10×109/L) 43例。rhTPO组与对照组总有效率差异无统计学意义[68.29%（28/41）对65.38%（17/26），P=0.806]。rhTPO组与对照组新诊断ITP、持续性及慢性ITP患儿总有效率差异均无统计学意义（P值分别为0.320和0.763）。rhTPO组与对照组重症ITP患儿总有效率差异无统计学意义[56.67%（17/30）对61.54%（8/13），P=0.766]。rhTPO组与对照组治疗后PLT峰值、PLT>30×109/L时间、PLT>50×109/L时间差异均无统计学意义[52(7~608）×109/L对40(3~152）×109/L，P=0.05; 7(3~13) d对4(2~24) d，P=0.202; 7.5(4~15) d对5.5(4~23) d，P=0.557]。rhTPO组治疗后PLT升高值于对照组[43(3~605）×109/L对32（−14~149）×109/L，P=0.042]。两组患儿均无明显不良反应发生。
结论: rhTPO单药治疗儿童ITP的疗效与长春地辛联合静脉丙种球蛋白相当，安全性较好。