重组人血小板生成素治疗特发性血小板减少性紫癜的临床疗效

目的 评价重组人血小板生成素（rhTPO）对糖皮质激素治疗无效的成人特发性血小板减少性紫癜（ITP）的有效性与安全性。方法 选择对糖皮质激素治疗无效的ITP患者74例，分为rhTPO+达那唑组(观察组)37例，达那唑组(对照组)37例。观察组皮下注射rhTPO 1.0μg/kg（300U/kg），1次/d， 共14d，停用rhTPO后再观察14d。对照组口服达那唑14d后，如血小板≤20×109/L可皮下注射rhTPO(1.0μg/kg)，1次/d， 14d ，停用rhTPO后观察14d。两组在整个试验阶段口服达那唑200mg/次，3次/d。比较两组前14d血小板计数的最高值和不同时间点血小板计数值的曲线下面积及有效率，比较对照组应用rhTPO前后的血小板计数及其差值。结果 观察组前14d血小板计数和升高值均显著高于对照组(P＜0.01）。14d内血小板计数值的曲线下面积中位数明显高于对照组（P＜0.001）;观察组rhTPO治疗14d内显效者（血小板计数升高至≥100×109/L）(16例、43.24%)明显高于对照组（3例、8.10%）（P＜0.01）。观察组良效者（血小板计数升高≥30×109/L）(9例、24.32%）与对照组(10例、27.03%）差异无统计学意义（P>0.01）。观察组近期有效observation率（显效+良效）明显高于对照组（P＜0.05）。对照组14d血小板升高未达有效的患者给予rhTPO治疗后血小板计数与使用rhTPO前相比明显升高，有效率达到62.50%。两组均无严重不良事件发生。结论 rhTPO治疗糖皮质激素治疗无效的ITP患者安全、有效。

Clinical efficacy and safety of rhTPO in the treatment of idiopathic thrombocytopenic purpura: a randomized controlled trial

Objective To investigate the efficacy and safety of recombinant human thrombopoietin (rhTPO) in the treatment of adult steroid-resistant idiopathic thrombocytopenic purpura(ITP). Methods 74 patients with steroid-resistant ITP were randomly divided into the observation group and the control group, 37 in each group. Patients in the observation group were treated with 1.0μg/kg·d of rhTPO, ih（300U/kg·d）and danazol, 600mg/d, three times a day, for 14d. Patients in the control group were treated with danazol, 600mg/d, three times a day, for 14d, and when the platelet count≤20×109/L after 14d, rhTPO, 1.0μg/kg·d, ih（300U/kg·d）, was added for 14d. In a 14d observation period, the increase of platelet and safety of rhTPO were analyzed. Results The mean platelet count and the increased number of platelets in the observation group were higher than those in the control group（P＜0.01）. The area under the curve of the platelet count in the observation group was larger than that in the control group（P=0.001）. The complete response rate(the platelet count≥100×109/L after treatment) was higher in the observation group(16 cases, 43.24%) than that in the control group(3 cases, 8.10%)（P＜0.01）. The good response(the platelet count≥30×109/L after treatment) had no difference in the observation group(9 cases, 24.32%) comparted with the control group(10 cases, 27.03%)（P>0.01）. The overall efficacy in the observation group was higher than that in the control group(67.56% vs 35.14%, P＜0.05）. In the control group, patients who had no response to danazol within 14d were treated with rhTPO, and the overall efficacy rate was 62.5%. rhTPO had no effect on platelet function and anti-platelet antibodies. No severe side effect was observation. Conclusion rhTPO is an effective and safe treatment method for adult steroid resistant ITP.