重组人血小板生成素治疗继发性免疫性血小板减少症的临床观察

Recombinant human thrombopoietin in the treatment of secondary immune thrombocytopenia

目的探讨重组人血小板生成素（recombinant humanized thrombopoietin,rh TPO）治疗继发性免疫性血小板减少症（ITP）的临床疗效和安全性。方法回顾性分析浙江省中医药大学附属第一医院2009年1月—2014年10月收治的21例继发性ITP患者,应用皮下注射rh TPO 300 U/（kg·d）,4周内获得完全反应和有效患者,改为皮下注射rh TPO 300 U/（kg·d）,每周1-3次间断维持治疗3个月。结果在21例继发性ITP患者中,应用rh TPO治疗4周时,完全反应10例（47.6%）,有效4例（19.0%）,总有效率为66.7%。与治疗前血小板计数比较,治疗后血小板计数最高平均值显著升高,差异有统计学意义（P〈0.01）;14例患者接受rh TPO间断维持治疗3个月,血小板计数显著高于治疗前,差异有统计学意义（P〈0.05）。但治疗前、后辅助性T细胞、杀伤性T细胞和调节性T细胞（Treg）差异均无统计学意义（P〉0.05）。治疗过程中共3例（14.3%）出现不良反应,主要表现为肌肉关节酸痛、乏力和头晕,对症治疗后好转。结论 rh TPO治疗继发性ITP疗效显著且耐受性好,虽不能提高Treg细胞数量,但已获得完全反应和有效的患者予以rh TPO间断维持治疗可能使其更长时间获益。

Objective To evaluate the efficacy and safety of recombinant human thrombopoietin（ rh TPO） in the treatment of secondary immune thrombocytopenia（ ITP）. Methods The clinical data of 21 patients with secondary ITP in our hospital from January 2009 to October 2014 were reviewed retrospectively. All patients were treated with subcutaneous rh TPO300 U /（ kg·d）. For the patients who achieved complete response or effective response in 4 four weeks,the intermittent maintenance therapy with rh TPO were conducted for 1- 3 times a week last 3 months. Results Among the 21 secondary ITP patients,the complete response rates（ CR）,effective response rates（ R） and overall response（ OS） after 4 weeks of rh TPO treatment were 47. 6%,19. 0% and 66. 7%,respectively. Compared with the value before the treatment,the highest average of the platelet count after the treatment was significantly higher（ P〈0. 01）; 14 patients received rh TPO intermittent maintenance therapy,whose the count of platelet after the 3 months treatment was significantly higher than that before the treatment（ P〈0. 05）. But the percentage of Th,Tc and Tregs of secondary ITP patients had no remarkably changed as compared with those before the treatment（ P〉0. 05）. The adverse reactions were found in 3 patients（ 14. 3%）,included pains in joints and muscles,fatigue and dizziness,and were relieved after the symptomatic treatment.Conclusion The rh TPO has significant clinical effect and good tolerance for secondary ITP patients,although it cannot improve the numbers of Treg cells. The intermittent maintenance therapy can get a longer benefit for the patients who achieved complete response or effective response after rh TOP continuous treatment.