重组人血小板生成素联合大剂量地塞米松治疗初治原发性免疫性血小板减少症的临床分析

A clinical analysis of treatment with recombinant human thrombopoietin combined with large doses of dexamethasone in primary immune thrombocytopenia

目的 观察重组人血小板生成素（rhTPO）联合大剂量地塞米松治疗初治原发性免疫性血小板减少症（ITP）的有效性和安全性.方法 本研究为前瞻性、随机对照临床研究.2013年6月至2015年2月新疆医科大学第一附属医院收治初治ITP患者59例,随机分为试验组（30例）和对照组（29例）.试验组采用rhTP0（300U·kg-1 ·d-1,第1～l4天,皮下注射,若PLT升高绝对值＞50×10^9/L则停用rhTPO）联合大剂量地塞米松（40 mg,第1～4天,静脉点滴）治疗;对照组仅使用大剂量地塞米松治疗（剂量用法同前）.比较两组的治疗效果,同时观察不良反应.结果 试验组的短期（15 d内）和中期（3个月）总有效率均高于对照组[83.3％ （25/30）比51.7％ （15/29）,76.7％（23/30）比20.7％ （6/29）,P值均＜0.01].试验组与对照组PLT达到100×109/L的中位时间分别为6.0d和6.8d.试验组患者rhTPO使用天数为（6.1±1.7）d.两组的不良反应均较轻微,试验组与rhTP0相关的不良事件发生率为6.7％（2/30）,主要表现为膝关节疼痛、乏力不适.结论 与单用大剂量地塞米松相比,rhTPO联合大剂量地塞米松治疗初治ITP在短期、中期疗效上均有明显优势,且不良反应较小,为ITP患者提供了新的治疗方法.

Objective To study the efficacy and safety of recombinant human thrombopoietin （rhTPO） combined with dexamethasone as front line regimen in patients with primary immune thrombocytopenia （ITP）.Methods This study was a prospective,randomized,controlled trial.A total of 59 primary ITP patients were enrolled at the First Affiliated Hospital,Xinjiang Medical University from June 2013 to February 2015.All subjects were randomized into study group （30 cases） and control group （29 cases）.The study group was scheduled to receive high-dose dexamethasone （40 mg intravenously dl-4） combined with rhTPO （300 U · kg-1 · d-1 subcutaneously dl-14）.Once absolute platelet count reached 〉 50 × 109/L,rhTPO stopped.Patients in control group were just administrated with high-dose dexamethasone （40 mg intravenously dl-4）.Efficacy and adverse reactions were evaluated.Results The short-term （15 days） and mid-term （3 months） response rates in the study group were 83.3% （25/ 30） and 76.7% （23/30） respectively,which were both significantly better than those in the control group [51.7% （15/29） and 20.7% （6/29） respectively] （P 〈0.01）.In the study group and control group,the median time platelet count reached 100 × 10^9/L was 6.0 and 6.8 days respectively.In the study group,the time of TPO usage was （6.1 ± 1.7） days.The incidence of adverse reactions in both groups was comparable and slight.The most common TPO related adverse events included knee ache and fatigue,which accounted for 6.7% （2/30） in the study group.Conclusions Recombinant human TPO combined with dexamethasone as front line treatment for primary ITP shows significant advantages in both short-term and mid-term responses with less and manageable adverse reactions.This may provide a new method to treat patients with primary ITP.