本文選題：鼠神經生長因子　切入點：兒童脊髓性肌萎縮癥　出處：《中國人民解放軍醫(yī)學院》2012年碩士論文

【摘要】：目的：觀察鼠神經生長因子（mouse nerve growth factor，mNGF）治療兒童脊髓性肌萎縮癥（spinal muscular atrophy，SMA）的臨床療效及安全性。 方法：本課題采用多中心、治療前后自身隨機交叉對照臨床研究（ChiCTR-TRC-10001093）。將解放軍總醫(yī)院兒科、北京兒童醫(yī)院神經內科、首都兒科研究所三個中心自2010年12月至2011年12月門診就診的48例兒童脊髓性肌萎縮癥患兒按照序列編號的、密封的、不透光的信封進行隨機分組，分為A組和B組，A組先給予注射mNGF18μg/日，療程24周，觀察12周；B組先觀察12周，再給予注射mNGF18μg/日，療程24周。在治療前、治療后12周，24周，36周分別進行一般體格檢查、神經系統查體、能力低下兒童評定量表（Pediatric Evaluation of Disability Inventory，PEDI）評估，測定血常規(guī)、尿常規(guī)及肝腎功能。在給藥前、給藥后24周分別進行肌電圖（Electromyogram，EMG）檢測。A組23例，其中9例全部完成隨訪，14例正在隨訪中，0例脫落，B組25例，其中6例全部完成隨訪，16例正在隨訪中，3例脫落。收集15例已經全部完成隨訪的患兒數據進行統計學的分析。 結果：15例患兒均完成鼠神經生長因子治療兒童脊髓性肌萎縮癥隨機交叉對照臨床研究，初步結果表明mNGF對兒童脊髓性肌萎縮癥有一定療效，特別是將肌力提高0.5～1級，，治療效果與病情輕重呈反比，Ⅲ型好于Ⅱ型；PEDI各項評分均有好轉趨勢，其中Ⅲ型患兒治療24周PEDI功能性日�；顒釉u分較Ⅱ型患兒治療24周PEDI功能性日常活動評分有明顯提高（P＜0.05）；15例患兒治療24周PEDI照顧者協調日�；顒釉u分較治療12周時有明顯提高（P＜0.05）；部分患兒的肌電圖治療后腓總神經及脛神經復合肌肉動作電位波幅較治療前增高，自發(fā)電位消失。所有患兒血、尿常規(guī)及肝腎功能檢查均未見異常。 結論：鼠神經生長因子治療兒童脊髓性肌萎縮癥隨機交叉對照臨床研究的初步結果證明mNGF對兒童脊髓性肌萎縮癥有一定療效，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

本文編號：1713424