重组人血小板生成素联合小剂量利妥昔单抗治疗激素无效/复发成人ITP的多中心临床研究

Combination therapy with recombinant human thrombopoietin and low-dose rituximab for corticosteroid-resistant/relapsed adult ITP:a multicenter clinical trial

目的：探讨重组人血小板生成素（rhTPO）联合小剂量利妥昔单抗（LD-RIT）对激素无效／复发的成人原发免疫性血小板减少症（ITP）的疗效和安全性。方法：激素治疗无效／复发的ITP患者91例，应用rhTPO联合LD-RIT治疗。结果：治疗至第14天时患者完全有效率为40．7％，总有效率为71．4％；第28天治疗终止时，患者完全有效率为42．9％，总有效率为75．8％；ITP患者中位起效时间为9（4～35）d；治疗结束后随访至50天及90天时，患者复发率分别为27．1％、35．7％；治疗第14、28、90天时，患者出血评分均较治疗前明显降低（P〈0．001）；所有患者对rhTPO、LD-RIT治疗耐受良好。结论：rhTPO联合LD-RIT治疗激素无效／复发的ITP患者，可快速提升血小板水平，显著缩短起效时间，减少出血风险，是一项有潜力的ITP二线治疗方案。

Objective： To evaluate the efficacy and safety of combination therapy with recombinant human thrombopoietin （rhTPO） and low-dose rituximab （LD-RIT） for corticosteroid-resistant/relapsed adult ITP. Meth- od：A total of 91 corticosteroid-resistant/relapsed ITP patients were treated with combination therapy of rhTPO and LD-RIT. Result：The complete response rate （CRR） and overall response rate （ORR） at the end of the second week after the initial treatment were 40.7% and 71.4o//00 respectively. At the end of fourth week,CRR was 42.9%, and ORR was 75.8%. The median time to response was 9 （4 to 35） days. Relapse rate during follow-up at the 50th day and the 90th day was 27.1% and 35.7 % respectively. Bleeding scores at the 14th, 28th, 90th day were signifi- cantly lower than that before treatment （P〈0. 001）. All patients were well tolerated to rhTPO and LD-RIT thera- py. Conclusion：Combination therapy with rhTPO and LD-RIT is effective to corticosteroid-resistant/relapsed ITP patients, with a relatively short time to response and low bleeding risks, and it may be considered as a potential sec- ond-line treatment regimen for ITP.