帕金森病中脑内注射胶质细胞源性神经营养因子：一项为期两年的结果研究。

Intraputamenal infusion of glial cell line-derived neurotrophic factor in PD: a two-year outcome study.

作者信息

Patel Nikunj K, Bunnage Martin, Plaha Puneet, Svendsen Clive N, Heywood Peter, Gill Steven S

机构信息

Frenchay Hospital, Institute of Neurosciences, Bristol, United Kingdom.

出版信息

Ann Neurol. 2005 Feb;57(2):298-302. doi: 10.1002/ana.20374.

DOI:10.1002/ana.20374

PMID:15668979

Abstract

We have shown previously that intraparenchymal infusion of glial cell line-derived neurotrophic factor (GDNF) continuously into the posterior putamen in five Parkinson's disease patients is safe and may represent a new treatment option. Here, we report a continuation of this phase I study. After 2 years of continual GDNF infusion, there were no serious clinical side effects and no significant detrimental effects on cognition. Patients showed a 57% and 63% improvement in their off-medication motor and activities of daily living subscores of the Unified Parkinson's Disease Rating Scale, respectively, and health-related quality-of-life measures (Parkinson's Disease Questionnaire-39 and Short Form-36) showed general improvement over time.

摘要

我们之前已经表明，在5名帕金森病患者的壳核后部持续脑实质内输注胶质细胞系源性神经营养因子（GDNF）是安全的，这可能代表一种新的治疗选择。在此，我们报告这项I期研究的延续情况。在持续输注GDNF 2年后，未出现严重临床副作用，对认知也无显著有害影响。患者在非服药状态下的统一帕金森病评定量表运动和日常生活亚评分分别改善了57%和63%，与健康相关的生活质量指标（帕金森病问卷-39和简明健康状况调查量表-36）随时间推移总体有所改善。