鼠神经生长因子治疗儿童脊髓性肌萎缩症随机交叉对照初步临床研究

发布时间：2018-04-05 05:28

  本文选题：鼠神经生长因子　切入点：儿童脊髓性肌萎缩症　出处：《中国人民解放军医学院》2012年硕士论文

【摘要】：目的：观察鼠神经生长因子（mouse nerve growth factor，mNGF）治疗儿童脊髓性肌萎缩症（spinal muscular atrophy，SMA）的临床疗效及安全性。 方法：本课题采用多中心、治疗前后自身随机交叉对照临床研究（ChiCTR-TRC-10001093）。将解放军总医院儿科、北京儿童医院神经内科、首都儿科研究所三个中心自2010年12月至2011年12月门诊就诊的48例儿童脊髓性肌萎缩症患儿按照序列编号的、密封的、不透光的信封进行随机分组，分为A组和B组，A组先给予注射mNGF18μg/日，疗程24周，观察12周；B组先观察12周，再给予注射mNGF18μg/日，疗程24周。在治疗前、治疗后12周，24周，36周分别进行一般体格检查、神经系统查体、能力低下儿童评定量表（Pediatric Evaluation of Disability Inventory，PEDI）评估，测定血常规、尿常规及肝肾功能。在给药前、给药后24周分别进行肌电图（Electromyogram，EMG）检测。A组23例，其中9例全部完成随访，14例正在随访中，0例脱落，B组25例，其中6例全部完成随访，16例正在随访中，3例脱落。收集15例已经全部完成随访的患儿数据进行统计学的分析。 结果：15例患儿均完成鼠神经生长因子治疗儿童脊髓性肌萎缩症随机交叉对照临床研究，初步结果表明mNGF对儿童脊髓性肌萎缩症有一定疗效，特别是将肌力提高0.5～1级，，治疗效果与病情轻重呈反比，Ⅲ型好于Ⅱ型；PEDI各项评分均有好转趋势，其中Ⅲ型患儿治疗24周PEDI功能性日常活动评分较Ⅱ型患儿治疗24周PEDI功能性日常活动评分有明显提高（P＜0.05）；15例患儿治疗24周PEDI照顾者协调日常活动评分较治疗12周时有明显提高（P＜0.05）；部分患儿的肌电图治疗后腓总神经及胫神经复合肌肉动作电位波幅较治疗前增高，自发电位消失。所有患儿血、尿常规及肝肾功能检查均未见异常。 结论：鼠神经生长因子治疗儿童脊髓性肌萎缩症随机交叉对照临床研究的初步结果证明mNGF对儿童脊髓性肌萎缩症有一定疗效，且安全性好。
[Abstract]:Objective: to observe the clinical efficacy and safety of nerve growth factor nerve growth factor (NGF) in the treatment of spinal muscular atrophy in children with spinal muscular atrophy.Methods: a multi-center, self-randomized cross-control clinical study of ChiCTR-TRC-1000 1093 was conducted before and after treatment.From December 2010 to December 2011, 48 children with spinal muscular atrophy who were treated in three centers of Beijing Children's Hospital, Beijing Children's Hospital, Beijing Children's Hospital and Beijing Children's Hospital were sequentially numbered and sealed.The impermeable envelopes were randomly divided into two groups: group A and group B were given mNGF18 渭 g / d for 24 weeks. Group B was given mNGF18 渭 g / day for 24 weeks.Before treatment, 12 weeks, 24 weeks and 36 weeks after treatment, general physical examination, physical examination of nervous system, evaluation of the Pediatric Evaluation of Disability Inventory DIY, blood routine examination, urine routine examination and liver and kidney function were performed respectively.Before and 24 weeks after administration, electromyogram electromyogramme EMG (EMG) was detected in 23 cases of group A, of which 9 cases were all followed up 14 cases were in group B, 25 cases were in group B, 6 cases were followed-up in 16 cases and 3 cases were abscission.Data of 15 children with complete follow-up were collected and analyzed statistically.Results the randomized cross-control clinical study of 15 children with spinal muscular atrophy treated by nerve growth factor was completed. The preliminary results showed that mNGF had certain curative effect on spinal muscular atrophy in children, especially the increase of muscle strength by 0.5 ~ (-1) grade.The therapeutic effect was inversely proportional to the severity of the disease, and the scores of type 鈪

本文编号：1713424