再生障碍性贫血发病后不同时期抗人胸腺/淋巴细胞球蛋白治疗疗效观察

目的 以抗人胸腺/淋巴细胞球蛋白(ATG)和环孢素A(CsA)为主的免疫抑制治疗(IST)是治疗重型再生障碍性贫血(SAA)的标准治疗,临床应用日益广泛,但发病后应用ATG时机对临床疗效的影响尚不清楚.本研究旨在观察再生障碍性贫血(AA)发病后不同时期应用ATG治疗的临床疗效.方法 回顾分析应用ATG治疗的58例AA患者.根据发病后应用ATG的时间不同分为两组.观察组25例,发病至行ATG治疗的时间≤1年,中位时间为4.5个月.对照组33例,发病后至行ATG治疗的时间>1～5年,中位时间18.3个月.比较两组患者ATG治疗后6个月反应率、5年无事件生存率及5年总生存率.结果 观察组与对照组治疗6个月时反应率分别为76.0%(19/25)、45.4%(15/33),差异具有统计学意义(P＜0.05);两组5年内无事件生存率分别为68.0%(17/25)、39.4%(13/33),差异具有统计学意义(P＜0.05);两组5年总生存率分别为92.0%(23/25)、90.9%(30/33),差异无统计学意义(P>0.05).结论 AA发病后1年内行ATG治疗与1年后行相同治疗疗效相比,6个月反应率及5年无事件生存率较高.

Efficacy of antithymocyte globulin treatment in patients with aplastic anemia correlated with duration from diagnosis to antithymocyte globulin treatment

Objective Immunosuppressive therapy（IST） with antithymocyte globulin（ATG） plus cyclosporine（CSA） is standard therapy in patients with severe aplastic anemia（SAA）,but there is no practical and reliable method to predict response to this treatment.The aim of this study was to assess the predictive factors for response to IST in patients with aplastic anemia（AA） and to identify prognostic factors following IST.Methods We conducted a single institution retrospective analysis on 58 patients with aplastic anemia treated with ATG-based IST between January 2001 and January 2005.According ATG use after the disease onset,the patients were divided into two groups,observed group 25 cases,time from onset to ATG treatment≤1 year,median time 4.5 months,control group 33 cases,time from onset to ATG treatment 1-5 years,median time 18.3 months.Comparisons were made in 6-month reaction after ATG treatment,event-free survival at five years and overall survival at five years between two groups.Results The response rate（RR） of patients at six months treated in observed group and control group were 76.0%（19/25）,45.4%（15/33）,respectively.In two groups,event-free survival at five years was 68.0%（17/25）,39.4%（13/33）,respectively.The two groups had overall survival at five years 92.0% and 90.9%,respectively.Conclusion The shorter duration from diagnosis to ATG treatment correlated with the efficacy of ATG treatment.