重组人血小板生成素治疗41例儿童原发免疫性血小板减少症的疗效及安全性

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<10×10(9)/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 >30×10(9)/L and > 50×10(9)/L had no statistically significant differences in rhTPO group compared with the control group [52(7–608) ×10(9)/L vs 40(3–152) ×10(9)/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) ×10(9)/L in the rhTPO group, which were higher than the control group [32(−14–149) ×10(9)/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.