比较不同重组人TPO方案联合免疫抑制治疗对重型再生障碍性贫血近期疗效的影响

Comparison of efficacy and safety of two different dose of recombinant human thrombopoietin regimens in severe aplastic anemia patients with immunosuppressive therapy

目的比较不同重组人TPO （rhTPO）方案联合免疫抑制剂治疗重型再生障碍性贫血（SAA）的近期疗效。方法回顾性分析接受一线免疫抑制治疗（IST）的61例成人SAA初诊患者资料，对比分析18例IST联合rhTPO每日1次（连续组）与43例IST联合rhTPO隔日1次（间日组）患者的疗效差异。结果两组患者在IST前基础临床特征差异无统计学意义。IST后3个月和6个月进行疗效评估，连续组与间日组患者总体血液学反应率比较差异无统计学意义（3个月：50.0%对51.2%，P= 0.934；6个月：77.8%对69.8%，P=0.525）。连续组IST后3个月良好血液学反应率明显高于间日组（38.9%对9.3%，P=0.011）。rhTPO应用后4周和8周两组脱离红细胞输注率差异无统计学意义（4周： 22.2%对18.6%，P=0.736; 8周：55.6%对46.5%，P=0.519），而治疗后8周脱离血小板输注率连续组明显高于间日组（88.9%对48.8%，P=0.003）。每日连续应用rhTPO治疗并不增加不良反应事件的发生。结论每日1次较隔日1次应用rhTPO促进SAA造血恢复和减少血小板输注依赖更为有效。

ObjectiveTo compare the short-term therapeutic effect of different regimens combining recombinant human thrombopoietin （rhTPO） with immunosuppressive therapy （IST） applied in severe aplastic anemia （SAA）.MethodsThe clinical data of newly diagnosed adult SAA patients treated with first-line IST, including 18 patients with rhTPO daily and 43 patients with rhTPO every other day, was analyzed retrospectively.ResultsThere was no significant difference between the basic clinical characteristics of the patients classified in different groups. The therapeutic effect was assessed 3 months and 6 months after IST. The statistical data revealed that the overall responses （OR） were not significantly different in daily group and every other day group （3 months： 50.0% vs 51.2%, P=0.934; 6 months： 77.8% vs 69.8%, P=0.525）, while the good hematological response （CR＋GPR） in SAA treated with rhTPO daily was significantly higher than that of patients treated with rhTPO every other day at 3 month after IST （38.9% vs 9.3%,P=0.011）. RBC transfusion independence were not significantly different between the two groups after 4 weeks as well as 8 weeks treatment（4 weeks： 22.2% vs 18.6%,P=0.736; 8 weeks： 55.6% vs 46.5%,P=0.519）, while platelet transfusion independence in rhTPO daily treated group was significantly higher than that in every other day group （88.9% vs 48.8%,P=0.003）. In addition, there were no more adverse events observed in rhTPO daily group.ConclusionsIt＇s more effective to promote hematopoietic recovery and reduce platelet transfusion dependence when rhTPO was daily used other than used every other day.