环孢素A联合雄激素治疗输血依赖非重型再生障碍性贫血预后因素分析

Prognostic factors of cyclosporine A combined with androgen in the treatment of transfusion dependent non-severe aplastic anemia

目的调查影响环孢素A(CsA)联合雄激素方案治疗输血依赖非重型再生障碍性贫血(TD-NSAA)获得血液学反应的因素。方法回顾性分析2010-2013年连续收治的77例TD-NSAA患者临床资料,单因素和多因素分析影响CsA联合雄激素治疗方案获得血液学反应患者的基线临床和血液学特征。结果77例TD-NSAA患者治疗后6个月和12个月获得血液学反应分别为43例(55.8%)和53例(68.8%),单因素分析基线血小板计数[19(6~61)×10^9/L对13.5(5~45)×10^9/L,P=0.001]是影响6个月获得血液学反应的唯一因素;基线血小板计数[18(6~61)×10^9/L对10.5(5~45)×10^9/L,P<0.001]、网织红细胞绝对值[0.03(0.01~0.06)×10^12/L对0.03(0.02~0.06)×10^12/L,P=0.043]、血小板输注依赖(P=0.007)和红细胞及血小板输注依赖(P=0.012)为治疗后12个月能否获得血液学反应相关因素。多因素分析显示基线血小板水平为获得血液学反应独立影响因素(P值分别为0.010和0.009)。受试者工作特征曲线(ROC曲线)方法显示基线PLT界值为15.5×10^9/L。结论TD-NSAA患者初诊时较高的血小板基线水平、网织红细胞基线水平和不伴血小板输注依赖均提示预后较好,血小板水平≥15.5×10^9/L时可以考虑采用CsA联合雄激素治疗。

Objective To analyze the prognostic factors of transfusion-dependent non-severe aplastic anemia(TD-NSAA)patients treated with cyclosporine A(CsA)and androgen.Methods Clinical data of 77 consecutive TD-NSAA patients treated with CsA and androgen were retrospectively analyzed between 2010 and 2013.We obtained clinical manifestations and baseline parameters of routine blood test from responders,and compared those with non-responders.All data were analyzed by univariate analysis and multivariate analysis.Results In 77 patients,there were 43(55.8%)patients achieved hematological response after 6 months’treatment,and 53(68.8%)patients got response after 12 months.Univariate analysis showed that platelets baseline was the only factor related to hematological response[19(6-61)×10^9/L vs 13.5(5-45)×10^9/L,P=0.001]after 6 months therapy.After 12 months,the statistical differences were maintained,which were platelets baseline[18(6-61)×10^9/L vs 10.5(5-45)×10^9/L,P<0.001],absolute reticulocytes[0.03(0.01-0.06)×10^12/L vs 0.029(0.02-0.06)×10^12/L,P=0.043],transfusion-dependent of platelet(P=0.007),transfusion-dependent of platelet and erythrocyte(P=0.012).Multivariate analysis showed that platelets baseline could be an independent prognostic factor of hematological response(P=0.010 or 0.009).Cutoff value of platelets by receiver operating characteristic curve was 15.5×10^9/L.Conclusion Baseline of higher platelets,higher reticulocyte,and no transfusion dependence of platelet are favorable prognostic factors.When platelets baseline is higher than 15.5×10^9/L,CsA and androgen regimen is rational.