重型再生障碍性贫血患儿的临床特征及预后分析  

Clinical Characteristics and Prognosis Analysis of Pediatric Severe Aplastic Anemia

作　　者：蔡玉丽 兰洋 易美慧 冯静 任媛媛 章婧嫽 王书春 郭晔 陈玉梅 竺晓凡 CAI Yu-Li;LAN Yang;YI Mei-Hui;FENG Jing;REN Yuan-Yuan;ZHANG Jing-Liao;WANG Shu-Chun;GUO Ye;CHEN Yu-Mei;ZHU Xiao-Fan(Department of Pediatric Hematology,State Key Laboratory of Experimental Hematology,National Clinical Research Center for Blood Diseases,Institute of Hematology&Blood Diseases Hospital,Chinese Academy of Medical Sciences&Peking Union Medical College,Tianjin 300020,China)

机构地区：[1]中国医学科学院北京协和医学院血液病医院(中国医学科学院血液学研究所),实验血液学国家重点实验室,国家血液系统疾病临床医学研究中心儿童血液病诊疗中心,天津300020

出　　处：《中国实验血液学杂志》2021年第6期1896-1902,共7页Journal of Experimental Hematology

摘　　要：目的:分析重型再生障碍性贫血(SAA)患儿的基本临床特征及预后影响因素。方法:选择2008年1至2018年4月收治的SAA患儿205例,对其基本临床特征及预后影响因素进行回顾性分析。结果:205例SAA患儿中,免疫抑制治疗(IST)后3、6和12个月的有效率(CR+PR)分别为50.9%、59.0%和73.9%,患儿5年总生存(OS)率为93.1%±2.0%。单因素生存分析显示,顺产出生的SAA患儿较剖宫产出生者5年OS率高(P=0.039),多因素Cox回归模型分析显示,出生方式对SAA患儿5年OS率无显著影响(P>0.05)。发病近期有装修材料接触史的SAA患儿IST后3个月反应率高于无装修材料接触史的SAA患儿(P<0.05)。结论:SAA患儿经IST治疗后可获得较高的反应率和OS率,发病近期有家庭/学校装修材料接触史可能为SAA患儿IST后3个月反应率的影响因素,但对患儿长期生存未见显著影响。Objective: To analyze the clinical characteristics and factors affecting prognosis in children with severe aplastic anemia(SAA). Methods: Two hundred and five children with SAA treated in our department from January 2008 to April 2018 were selected, and the clinical characteristics and factors affecting prognosis were retrospectively analyzed. Results: Among 205 SAA children, the effective rate(CR+PR) at 3, 6 and 12 months after immunosuppressive therapy(IST) treatment was 50.9%, 59.0% and 73.9%, respectively, and 5-year overall survival rate was 93.1%±2.0%. Univariate analysis showed that 5-year overall survival rate of SAA children of spontaneous delivery was higher than that of cesarean section(P=0.039), while multivariate analysis showed that birth way had no significant influence on 5-year overall survival rate(P>0.05). The response rate at 3 months after IST of children with a recent history of decoration before SAA onset was higher than those without history of decoration(P<0.05).Conclusion: Most of the SAA children can achieve high response rate and overall survival rate. Patients with recent history of home/school decoration may be the factor affecting hematological response after 3 months of IST, but have no influence on long-term overall survival.

关 键 词：再生障碍性贫血 免疫抑制治疗 生存 儿童 

分 类 号：R556.5[医药卫生—血液循环系统疾病]