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中文摘要：
目的 分析极重型/重型再生障碍性贫血（V/SAA）患者一线免疫抑制治疗（IST）迟发血液学反应特征,探讨难治性V/SAA尽早二次治疗的合理性.方法 回顾性分析一线接受IST的533例V/SAA患者临床资料,定义IST后6个月内获得血液学反应为应时反应,定义6～12个月获得血液学反应为迟发反应,观察迟发反应的发生率、血液学反应质量及其影响因素.结果 533例患者中,45例（8.44％）获得迟发反应,占未获得应时反应且继续接受环孢素A治疗患者的29.03％（45/155）.至IST后12个月及随访结束时迟发反应组血液学反应质量均劣于应时反应组（χ^2=62.616,P＜0.001和χ^2=6.299,P=0.043）.迟发反应组VSAA患者比例高于应时反应组（57.8％对38.3％,P=0.013）,外周血网织红细胞（ARC）比例、ARC计数以及ANC更低,多因素分析显示治疗前ARC＜10× 10^9/L的患者获得应时反应的机会明显减少[OR=3.641 （95％ CI 1.1718～7.719）,P=0.001];未发现独立预测IST后6个月无效患者获得迟发血液学反应的因素.6个月未获血液学反应患者5年总生存率为76.50％（95％ CI 71.6％～81.4％）、无事件生存率为29.10％（95％ CI 25.2％～33.0％）,均显著低于应时反应组患者的97.6％（95％CI96.6％～98.6％）、84.0％（95％ CI81.1％～86.9％）（P值均＜0.001）.结论 V/SAA患者IST获得迟发血液学反应难以预测,比例较小,疗效质量相对较差.难治性V/SAA患者尽早进行挽救治疗是合理的.
英文摘要：
Objective To explore the characteristics of delayed hematologic response in very/ severe aplastic anemia （V/SAA） patients who were treated with immunosuppressive treatment （IST） as first-line approach,and investigate the rationality of early salvage treatment in refractory patients.Methods The data of V/SAA patients front-line treated with IST were retrospectively analyzed.Delayed response was defined as acquiring hematologic response between 6 and 12 months after 1 course of IST.The clinical as well as hematologic characteristics of the delayed responded patients were investigated.Results Of the 533 patients,45 （8.44%,45/533） were delayed hematologic responders,which accounted for 29.03% （45/155） of the whole non-responders at 6 months.The quality of response in delayed responders analyzed at 12 months （χ^2=62.616,P 〈 0.001） and at the end of follow-up （χ^2=6.299,P=0.043） was significantly worse than that of robust response group.There were more VSAA patients in delayed response group compared with robust response group （57.8% vs 38.3%,P=0.013）,and all the baseline absolute reticulocyte （ARC） count,ARC proportion and absolute neutrophil count （ANC） were much lower than that in delayed response group.Multivariate analysis about the above 2 groups showed that the baseline ARC count 〈10 × 10^9/L significanty reduced the chance of hematologic response within 6 months [OR=3.641 （95% CI 1.718-7.719）,P=0.001],and not any factor was found to predict delayed hematologic response in non-responders at 6 months.The 5-year overall survival of 76.50% （95% CI 71.6%-81.4%）and event free survival of 29.10% （95% CI 25.2%-33.0%） in non-responders at 6 months,both were worse than 97.6% （95% CI 96.6%-98.6%） and 84.0% （95% CI 81.1%-86.9%） （P 〈 0.001） of robust response group.Conclusion The incidence of delayed hematologic response in V/SAA patients by IST is low.The quality of delayed response is not satisfactory and there is no effective means to predict the delaye
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