Objective: To evaluate the efficacy of recombinant human thrombopoietin (rhTPO) and related factors which influencing the therapeutic effect in adults with severe immune thrombocytopenia (ITP).
Methods: The efficacy of rhTPO in 92 hospitalized adult patients [35 males and 57 females, median age as 34 (18-65) years] with severe ITP, including 7 cases of newly diagnosed ITP, 29 cases of persistent ITP and 56 cases of chronic ITP from May 2012 to May 2014 was retrospectively investigated. All patients received subcutaneous rhTPO, the injected dosage was 300 U·kg⁻¹·d⁻¹ for 14 days, platelet counts were recorded and followed-up for a week.
Results: The overall response rate of rhTPO treatment was 60.9%. The overall response rates in newly diagnosed, persistent and chronic ITP were 71.4%, 62.1% and 58.9% respectively. The median platelet counts on fourth，seventh, fourteenth days of treatment, and the seventh day of withdrawal were 27(5-49), 65(16-138), 133(28-208) and 67(15-134)×10⁹/L, respectively. The median time was 6(5-7) days when platelet counts reached 100×10⁹/L, the median peak time was 11(5-17) days, the median maximum peak of platelet counts was 194(132-274)×10⁹/L in patients who reached CR after treatment. Related factors which affected therapeutic effect were analyzed in patients who reached CR after treatment, and indicated that sex, age, disease stage, express of platelet membrane glycoprotein (GP) and relative number of CD19+ B, CD3+CD4+ T, CD3+CD8+ T lymphocyte in blood samples did not influence the probability of complete response (P>0.05). A few patients with fever, muscle aches, fatigue or dizziness could be self-recovery without special intervention.
Conclusion: Severe ITP in adults treated by rhTPO had satisfactory therapeutic effect and safety.
目的: 评价重组人血小板生成素（rhTPO）治疗成人重型原发免疫性血小板减少症（ITP)的疗效及安全性。
方法: 对2012年5月至2014年5月期间收治的92例成人重型ITP患者的临床资料进行回顾性分析。其中新诊断ITP 7例、持续性ITP 29例、慢性ITP 56例，男35例、女57例，中位年龄34 (18~65）岁。全部患者接受rhTPO 300 U·kg−1·d−1×14 d治疗，停药后观察7 d。
结果: 全部92例患者rhTPO治疗的总反应率为60.9%(56/92)，新诊断ITP、持续性ITP、慢性ITP患者总反应率分别为71.4%、62.1%、58.9%。所有患者治疗第4、7、14天与停药第7天中位PLT分别为27(5~49)、65(16~138)、133 (28~208)、67(15~134)×109/L。获完全反应的患者PLT达到100×109/L的中位时间为6(5~7)d, PLT达峰值中位时间为11(5~17)d，中位PLT峰值为194(132~274)×109/L。患者性别、年龄、疾病分期、血小板膜糖蛋白特异性抗体表达及外周血CD19+B、CD3+CD4+T、CD3+CD8+T淋巴细胞数量均与疗效无相关性(P>0.05)。少数患者出现低热、肌肉酸痛、乏力、头晕，均自行恢复。
结论: rhTPO治疗成人重型ITP具有较好的疗效和安全性。