中文摘要：

目的初步探讨重组人EPO（rhEPO）±rhG．CSF治疗较低危（国际预后积分系统的低危和中危一1）骨髓增生异常综合征（MDS）患者的疗效及其影响因素。方法52例较低危原发MDS患者接受至少8周以上的rhEPO单独或联合rhG—CSF每日或隔日单次皮下注射治疗，待红系达到最佳疗效且维持4周后rhEPO±rhG．CSF逐渐减量至停用或维持最佳疗效的最小剂量。回顾性分析患者的临床特征、疗效、生存情况及其影响因素。结果52例患者接受rhEPO±rhG—CSF治疗，中位治疗时间为8（2～45）个月。其中27例（51．9％）治疗有效（9例达完全缓解，18例出现红系反应），中位疗效持续时间为37（6-94）个月。10例患者复发，其中4例为疾病进展。中位随访时间为37（6～114）个月，中位生存时间为47（6～114）个月。多因素分析结果显示初诊时血清EPO（sEPO）水平〈500u／L（P=0．030）、红细胞爆式集落形成单位（BFU．E）计数≥25／10^5骨髓单个核细胞（BMMNc）（P=0．040）及接受中高剂量rhEPO±rhG．CSF治疗（P=-0．020）的患者治疗8周后MDS相关贫血容易获得改善；HGB水平（P=0．040）及染色体核型（P〈0．01）对疗效持续时间有显著影响；年龄〈60岁（P=-0．050）、染色体核型为预后良好或中等（P=0．03）和治疗有效（P=0．020）对患者生存时间有显著影响。结论中高剂量rhEPO±rhG—CSF能够有效改善较低危原发性MDS患者相关贫血症状，延长患者生存期。

英文摘要：

Objective To investigate the efficacy and impact factors in lower-risk [International prognostic scoring system （IPSS） low or intermediate-1 risk] myelodysplastic syndrome （MDS）patients treated with recombinant human erythropoictin （rhEPO） alone or in combination with recombinant human granulocyte colony-stimulating factor （rhG-CSF）. Methods A total of 52 consecutive lower-risk MDS patients received subcutaneous injection of rhEPO alone or in combination with rhG-CSF at least 8 weeks, the rhEPO dose would be reduced slowly to stop or kept at minimum to maintain the response when the best efficacy achieved and maintained for 4 weeks. Their clinical features, efficacy, survival and the predictors of efficacy were analyzed retrospectively. Results The overall response rate was 51.9% （27/ 52） with 33.3% （9/27） achieving complete remission （CR） and 66.7% （18/27） achieving erythroid response （HI-E）. In multivariate analysis, sEPO level （less than 500 U/L）, BFU-E count （more than 25/10^5 BMMNC）, intermediate and high doses rhEPO±rhG-CSF therapy were independent predictors of better response. The median therapy period was 8 （2-45） months and the median efficacy duration was 37 （6- 94） months （38 months for CR, 36 months for HI-E）. Ten of the 27 responsive patients relapsed and 40% of them had disease progressions. Hemoglobin levels and karyotype affect response duration. Median overall survival was 47 （6-114） months on a 37 （6-114） months median follow-up. In multivariate analysis, ages （less than 60 years old）, karyotype （good or intermediate） and response to rhEPO＋rhG-CSF therapy may have a favorable survival impact on MDS. Conclusion rhEPO, alone or in combination with rhG-CSF, is a useful drug for the treatment of anemia in lower-risk MDS patients and has favorable impact on life expectancy.