鼠神经生长因子联合康复治疗全面性发育迟缓前瞻性多中心临床随机对照研究  

Mouse nerve growth factor combined with rehabilitation in the treatment of global developmental delay:a prospective multicenter clinical randomized controlled trial

作　　者：唐久来[1] 李晓捷[2] 吴德[1] 庞伟[2] 宋虎杰 黄艳[4] 郭津[2] 李玲 杨李[1] 华冉 宋福祥[2] 刘玉堂 张明达[4] 吕洋 Tang Jiulai;Li Xiaojie;Wu De;Pang Wei;Song Hujie;Huang Yan;Guo Jin;Li Ling;Yang Li;Hua Ran;Song Fuxiang;Liu Yutang;Zhang Mingda;Lyu Yang(Pediatric Neurology Rehabilitation Center,the First Affiliated Hospital of Anhui Medical University,Hefei 230022,China;Department of Pediatric Cerebral Palsy Rehabilitation,the Third Affiliated Hospital of Jiamusi University,Jiamusi 154002,China;Department of Pediatric Rehabilitation,Xi′an Traditional Chinese Medicine Hospital of Encephalopathy,Xi′an 710032,China;Department of Rehabilitation,Children′s Hospital Affiliated to Shandong University,Jinan 250022,China;Department of Pediatric Rehabilitation,Hainan Women and Children′s Medical Center,Haikou 570206,China)

机构地区：[1]安徽医科大学第一附属医院小儿神经康复中心,合肥230022 [2]佳木斯大学附属第三医院脑瘫康复科,佳木斯154002 [3]西安中医脑病医院儿童康复科,西安710032 [4]山东大学附属儿童医院康复科,济南250022 [5]海南省妇女儿童医学中心儿童康复科,海口570206

出　　处：《中华实用儿科临床杂志》2023年第11期857-862,共6页Chinese Journal of Applied Clinical Pediatrics

摘　　要：目的探索鼠神经生长因子(mNGF)联合康复治疗全面性发育迟缓(GDD)儿童的临床疗效。方法前瞻性多中心临床随机对照研究。选取2020年5月至2022年1月国内5家医院收治的120例GDD患儿,采用区组随机化方法将患儿随机分入mNGF组和常规康复组。所有受试者在研究开始后均接受规范化康复治疗,mNGF组在康复治疗的基础上加用mNGF;所有受试者于基线期、治疗90 d和120 d访视时各接受1次Gesell发育诊断量表评价,并记录受试者发育商(DQ);采用成组t检验、秩和检验和χ2检验进行疗效分析评价。结果治疗90 d并继续进行120 d访视时,mNGF组受试者的粗动作、细动作、应物能和应人能的DQ较基线期提高值高于常规康复组(粗动作:7.520±13.900比0.450±11.459;细动作:7.800±15.346比1.250±11.581;应物能:7.730±13.428比2.100±12.022;应人能:6.780±11.651比1.780±10.120),差异均有统计学意义(均P<0.05);本研究中未发生严重不良事件,未发现与研究药物相关的重要医学事件。结论mNGF联合康复治疗GDD患儿可更有效地提高患儿粗动作、细动作、应物能和应人能的发育水平,持续改善发育迟缓状况,且安全性高。Objective To observe the clinical efficacy of mouse nerve growth factor(mNGF)combined with rehabilitation on children with global developmental delay(GDD).Methods It was a prospective multicenter clinical randomized controlled trial(RCT)involving 120 children with GDD admitted to 5 hospitals in China from May 2020 to January 2022.They were randomly divided into mNGF group and conventional rehabilitation group using block randomization method.All children were managed by standardized rehabilitation after recruitment,and those in the mNGF group were additionally given mNGF injections.All subjects were surveyed using the Gesell Development Diagnosis Schedules(GDDS)at baseline,90 days and 120 days after treatment,and their developmental quotient(DQ)was recorded.Clinical efficacy was analyzed by the paired t-test,rank sum test and Chi-squared test.Results After 90 days of treatment and the continuous follow-up to 120 days,the increases in the DQ of gross motor(7.520±13.900 vs.0.450±11.459),fine motor(7.800±15.346 vs.1.250±11.581),adaptive behavior(7.730±13.428 vs.2.100±12.022)and personal-social behavior(6.780±11.651 vs.1.780±10.120)than baseline were significantly higher in mNGF group than those of conventional rehabilitation group(all P<0.05).Serious adverse events and important drug-related medical events were not reported.Conclusions mNGF combined with rehabilitation effectively enhances the development levels of gross motor,fine motor,adaptive behavior and personal-social behavior,and continuously improves the condition of GDD in children with a high safety.

关 键 词：全面性发育迟缓 鼠神经生长因子 多中心 随机对照 

分 类 号：R748[医药卫生—神经病学与精神病学]