慢性肉芽肿病治疗进展  

Therapeutic advances in chronic granulomatous disease

作　　者：王佳[1] 韩晓华[1] 尚云晓[1] 蔡栩栩[1] 陈宁[1] Wang Jia;Han Xiaohua;Shang Yunxiao;Cai Xuxu;Chen Ning(Department of Pediatric Respiratory,Shengjing Hospital of China Medical University,Shenyang 110004,China)

机构地区：[1]中国医科大学附属盛京医院小儿呼吸科,沈阳110004

出　　处：《中国小儿急救医学》2020年第12期929-932,共4页Chinese Pediatric Emergency Medicine

基　　金：国家留学基金(201908865002)。

摘　　要：慢性肉芽肿病(chronic granulomatous disease,CGD)是一种罕见的遗传性免疫缺陷,由于吞噬细胞烟酰胺腺嘌呤二核苷酸磷酸(NADPH)氧化酶复合物的功能障碍,导致儿童早期即出现严重且反复的感染。近年来随着治疗的进步,CGD患者的存活率和生活质量明显提高。本文就近年来造血干细胞移植、基因治疗和常规治疗在CGD的研究进展进行综述,以期进一步提高临床医生对CGD治疗的认识。Chronic granulomatous disease(CGD)is a rare inherited immunodeficiency due to dysfunction of the phagocytic nicotinamide adenine dinucleotide phosphate(NADPH)oxidase complex leading to severe and recurrent infections in early childhood.With the progress of treatment in recent years,the survival rate and quality of life of CGD patients have been significantly improved.This study reviewed the research progress of hematopoietic stem cell transplantation,gene therapy and conventional therapy in CGD in recent years,in order to improve clinicians′understanding of CGD therapy.

关 键 词：慢性肉芽肿病 造血干细胞移植 基因治疗 常规治疗 感染 炎症 

分 类 号：R725.9[医药卫生—儿科]