皮质类固醇治疗非卧床杜氏肌营养不良症患儿肺功能与运动功能的纵向研究  

Longitudinal study of pulmonary and motor function in children with ambulatory Duchenne muscular dystrophy treated with corticosteroids

作　　者：吴越廷 周林雨涵 胡钦 许华燕 黄敏 陈晓勇[1] 张萌 李中会 茹凉[4] 王秋[1] 蔡晓唐[1] Wu Yueting;Zhou Linyuhan;Hu Qin;Xu Huayan;Huang Min;Chen Xiaoyong;Zhang Meng;Li Zhonghui;Ru Liang;Wang Qiu;Cai Xiaotang(Department of Rehabiitation Medicine Key Laboratory of Birth Defects and Reated Diseases of Women and Children(Sichuan University),Ministry of Education,West China Second University Hospital,Sichuan University,Chengdu 610041,Sichuan Province,China;Department of Radiology,Key Laboratory of Birth Defects and Related Diseases of Women and Children(Sichuan University),Ministry of Education,West China Second University Hospital,Sichuan University,Chengdu 610041,Sichuan Province,China;Department of Pediatric Genetics.Endocrinology and Metabolism,Chengdu Women's and Children's Central Hospital,Chengdu 610073,Sichuan Province,China;Department of Pediatric Medicine,the First Affiliated Hospital of Xinjiang Medical University,Urumqi 830054,Xinjiang Uyghur Autonomous Region,China)

机构地区：[1]四川大学华西第二医院康复医学科、出生缺陷与相关妇儿疾病教育部重点实验室,成都610041 [2]四川大学华西第二医院放射科、出生缺陷与相关妇儿疾病教育部重点实验室,成都610041 [3]成都市妇女儿童中心医院儿童遗传与内分泌代谢科,成都610073 [4]新疆医科大学第一附属医院儿内科,乌鲁木齐830054

出　　处：《中华妇幼临床医学杂志（电子版）》2024年第3期292-301,共10页Chinese Journal of Obstetrics & Gynecology and Pediatrics(Electronic Edition)

基　　金：四川省中央引导地方科技发展项目(2023ZYD0121、2023ZYD0100);四川省科技计划项目(重点研发项目)(2023YFG0284);四川省卫生健康委员会医学科技项目(21PJ048);西藏自治区科技计划项目(XZ202201ZY0049G)。

摘　　要：目的探讨对非卧床杜氏肌营养不良症(DMD)患儿采取皮质类固醇(CS)治疗≥1年的肺功能和运动功能指标变化。方法选择2020年4月至2021年5月四川大学华西第二医院、成都市妇女儿童中心医院和新疆医科大学第一附属医院儿内科门诊收治的采取CS治疗≥1年的32例DMD患儿为研究对象。根据患儿采取CS初治时年龄,将其分别纳入≤7岁组(n=13)与>7岁组(n=19)。对这32例患儿初治后进行为期2年的回顾性纵向研究,初治与初治后第1、2年(分别采用T0、T1、T2表示)时,使用肺功能测试仪评估2组患儿4项肺功能指标[用力肺活量占预计值百分比(FVC%)、第1秒用力呼气量占预计值百分比(FEV1%)、第1秒用力呼气量与用力肺活量的比值(FEV1/FVC)、呼气峰流速占预计值百分比(PEF%)],并采用《北极星移动评价量表》(NSAA)和3项关键运动功能指标计时测试(完成仰卧-站立、10 m走/跑和上4级楼梯各测试1次所需时间),评估患儿运动功能。所有DMD患儿与≤7岁组、>7岁组患儿不同时间点的4项肺功能指标、NSAA评分和3项关键运动功能指标比较,采用重复测量资料的方差分析或Friedman检验。采用多重线性回归分析方法,分析DMD患儿NSAA评分及3项关键运动功能指标对FVC%的影响。本研究遵循的程序符合四川大学华西第二医院伦理委员会规定,并获得该伦理委员会批准[审批文号:科研伦审2023(303)号]。结果①32例DMD患儿T0、T1、T2时,肺功能指标FVC%、FEV1%和FEV1/FVC分别总体比较,差异均无统计学意义(P>0.05),而PEF%呈增高趋势,总体比较,差异有统计学意义(F=4.40、P=0.016)。进一步两两比较结果显示,T1时,FVC%较T0时增加8.3%,差异有统计学意义(Z=-2.09、P=0.036);T2时,FEV1%、PEF%较T0时分别增加8.2%、9.2%,差异均有统计学意义(Z=-2.15、P=0.032,t=-2.69、P=0.011)。②T0、T1、T2时,32例DMD患儿NSAA评分呈下降趋势,而完成仰卧-站立、10 m走/跑测试所需�Objective To explore the changes of pulmonary and motor function indexes in children with ambulatory Duchenne muscular dystrophy(DMD)treated with corticosteroids(CS)for at least one year.Methods A total of 32 DMD children admitted to the outpatient department of West China Second University Hospital of Sichuan University,Chengdu Women′s and Children′s Central Hospital,and the First Affiliated Hospital of Xinjiang Medical University from April 2020 to May 2021 were selected into this study.According to the age of children when they initially received treatment with CS,they were divided into≤7 years group(n=13)and>7 years group(n=19).A 2-year retrospective longitudinal study of these 32 children was conducted after initial treatment.And the children′s pulmonary function[forced vital capacity percent predicted value(FVC%),forced expiratory volume in one second percent predicted value(FEV1%),ratio of forced expiratory volume in one second to forced vital capacity(FEV1/FVC),peak expiratory flow percent predicted value(PEF%)]was assessed by spirometry at initial treatment and at 1 and 2 years after initial treatment(recorded as T0,T1 and T2,respectively).The North Star Ambulatory Assessment(NSAA)score and three key monitor function indexes timing tests(the time required to complete supine-up,10 m walk/run,and climb 4 stairs test once each)were performed to evaluate the children′s motor function at T0,T1 and T2.Repeated measures analysis of variance or Friedman test was used to compare the 4 pulmonary function indexes and NSAA score and three key monitor function indexes of all DMD children,≤7 years group and>7 years group at different time points.Multiple linear regression analysis was used to analyze the effect of NSAA score and three key monitor function indexes on FVC%among 32 DMD children.This study was approved by the Medical Ethics Committee of West China Second University Hospital,Sichuan University(Ethics No.2023-303).Results①Among the 32 DMD children,there were no significant differences in FVC%,

关 键 词：肌营养不良 杜氏 呼吸功能试验 肺功能 肺活量 用力呼气量 峰值呼气流速 运动功能 儿童 

分 类 号：R725.6[医药卫生—儿科]