纹状体 GDNF 输注在停止 GDNF 后仍能维持帕金森病的益处。
Benefits of putaminal GDNF infusion in Parkinson disease are maintained after GDNF cessation.
机构信息
From the Institute of Clinical Neurosciences (N.K.P., S.J., S.S.G.), Frenchay Hospital, Bristol; and Imperial College London (N.P., G.R.H., D.J.B.), Division of Brain Sciences, Hammersmith Hospital, London, UK.
出版信息
Neurology. 2013 Sep 24;81(13):1176-8. doi: 10.1212/WNL.0b013e3182a55ea5. Epub 2013 Aug 14.
Abstract
We previously reported clinical improvement, increase in putamen [(18)F]-dopa uptake on PET imaging, and neuropathologic evidence of sprouting of dopaminergic fibers following chronic intraputaminal delivery of glial cell line-derived neurotrophic factor (GDNF) in idiopathic Parkinson disease (PD).(1-3) We now provide clinical and PET evidence of persistent efficacy lasting for at least 3 years following cessation of GDNF infusion in a patient with PD. This is a single-case observational study, providing Class IV evidence.
摘要
我们之前曾报道过一例临床改善的病例，该患者患有特发性帕金森病（PD），在接受胶质细胞源性神经营养因子（GDNF）慢性脑内给药后，纹状体[(18)F]-多巴摄取增加，且 PET 成像显示多巴胺能纤维有发芽现象。(1-3)我们现在提供了一名 PD 患者停止 GDNF 输注后至少 3 年持续有效的临床和 PET 证据。这是一项单病例观察研究，提供了 IV 级证据。
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本文引用的文献
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2
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3
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