输血依赖型非重型再生障碍性贫血临床特点及转归研究  

Stucly on clinical characteristics and consequences of transfusion dependent-non severe aplastic anemia

作　　者：王素丽 杨慧[2] 李瑞鑫[2] 陈曦 龙启强 杨岩[4] 贾晋松[5] 何广胜[2] 李建勇[2] WANG Su-li;YANG Hui;LI Rui-xin;CHEN Xi;LONG Qi-qiang;YANG Yan;JIA Jin-song;HE Guang-sheng;LI Jianyong(Department of Hematology,the Sixth People’s Hospital of Shanghai,Shanghai 201400,China;不详)

机构地区：[1]上海市第六人民医院南院奉贤区中心医院血液科,上海201400 [2]南京医科大学第一附属医院,江苏省人民医院血液科,江苏南京210029 [3]南京市第二医院,南京中医药大学附属南京医院血液科,江苏南京210029 [4]吉林大学第一医院血液肿瘤中心,吉林长春130021 [5]北京大学人民医院血液科,北京100044

出　　处：《中国实用内科杂志》2021年第10期877-881,共5页Chinese Journal of Practical Internal Medicine

基　　金：卫生公益性行业科研专项经费项目(201202017)。

摘　　要：目的总结输血依赖型非重型再生障碍性贫血(TD-NSAA)患者的临床特点及转归。方法收集2013年12月至2017年1月就诊于中国贫血东部协作组各医院的124例TD-NSAA患者临床资料,分析临床表现、输血频率、实验室检查结果、并发症、转归及相关因素。结果共纳入124例TD-NSAA,中位病程38(3~363)个月,中位年龄32(3~80)岁。常见并发症有肝肾功能损害(42例,33.9.%)、糖尿病或糖耐量异常(24例,19.3%)、严重感染(29例,23.4%)、铁超负荷(53/101,52.5%)。环孢菌素(CsA)治疗119例,23例有效(19.3%);抗人胸腺细胞免疫球蛋白(ATG)联合CsA治疗30例,17例有效(56.7%)(P<0.001)。57例进展为SAA(46.0%),中位进展时间24(3~216)个月。中性粒细胞绝对值<0.5×10^(9)/L、严重感染和铁超负荷是SAA进展的危险因素(P=0.022,P=0.025,P=0.001)。转变为阵发性睡眠性血红蛋白尿症11例、骨髓增生异常综合征2例、急性髓系白血病1例,10例死亡(8.1%)。结论TD-NSAA患者病程迁延,易并发重要脏器功能损伤和疾病进展;CsA治疗效果欠佳,ATG联合CsA强化免疫抑制治疗可能改善预后,值得探索。Objective To review and summarize the clinical and laboratory data,risk factors of progression and surviving state of the patients with transfusion dependent-non-severe aplastic anemia(TD-NSAA).Methods This study included124 patients with TD-NSAA who were diagnosed in China Anemia East Cooperation Group(5 hospitals)from December2013 to January 2017.The clinical and laboratory data,as well as factors related with prognosis and survival rate were summarized and evaluated.Results The median age of the patients was 32 years(range from 3 to 80 years),the median duration of disease was 38 months(range from 3 to 363 months).Common complications included iron overload(53 cases/101 cases,52.5%),damage of liver and renal function(42 cases,33.9%),diabetes/impaired glucose tolerance(24 cases,19.3%),and severe infection(29 cases,23.4%).In 30 patients received antithymocyte globulin(ATG)combined with CsA,17 patients(56.7%)got response,which was higher than that of 23(19.3%)of 119 case who were initially treated with cyclosporine(CsA)alone(P<0.001).57 patients(46.0%)progressed to severe aplastic anemia(SAA).The median progression time was 24 months(range from 3 to 216 months).ANC<0.5×10^(9)/L,severe infection,and iron overload were markedly related with the progression to SAA(P=0.022,P=0.025,P=0.001).Fourteen patients developed secondary colonal hematologic disease:11 patoents with paroxysmal nocturnal hemoglobinuria(PNH),two patients wiht myelodysplastic syndromes(MDS),one patient with acute myeloid leukemia(AML),respectively.10 patients(8.1%)had died duringthefollow-up.Conclusions TD-NSAApatientshave a persistent course and are prone to diseaseprogression and important organ function damage.The therapeutic effect of CSA is poor.Intensiveimmunosuppressivetherapyof ATGcombinedwith CSAmight improve the prognosis,which is worth exploring.

关 键 词：输血依赖 再生障碍性贫血 预后 环孢菌素 抗人胸腺细胞免疫球蛋白 

分 类 号：R556.5[医药卫生—血液循环系统疾病]