Comparative efficacy of surgical approaches to disease modification in Parkinson disease.
Comparative efficacy of surgical approaches to disease modification in Parkinson disease.
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DOI：
10.1038/s41531-022-00296-w
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发表时间：
2022-03-25
期刊：
影响因子：
--
通讯作者：
Turner DA
中科院分区：
文献类型：
--
作者：
Rahimpour S;Zhang SC;Vitek JL;Mitchell KT;Turner DA
帕金森病（PD）可以最佳地用疾病修饰疗法来治疗以减缓进展。我们比较了用于改善PD疾病的手术方法的基础数据：（1）用干细胞衍生的多巴胺能神经元替代受损细胞的细胞移植治疗;（2）生长因子促进现有多巴胺能神经元存活的临床试验;（3）PD病程早期的丘脑底核脑深部电刺激;（4）腹部迷走神经切断术以降低潜在的疾病从肠道传播到大脑的风险。尽管这些手术方法的目标是参与PD的潜在机制，但仍处于实验阶段，这表明将治疗概念转化为临床实践存在困难。选择与症状获益分开的结局指标来评估疾病缓解，对于评价疾病缓解干预对长期疾病负担的影响至关重要，包括影像学研究和临床评定量表，即，统一帕金森病评定量表。治疗干预将需要较长的随访时间（即，5-10年），以分析与对症治疗相比的疾病改善。通过机械方法实现疾病改变的侵入性手术治疗的前景受到将这些概念转化为有效临床试验的现实的限制。
Parkinson’s disease (PD) may optimally be treated with a disease-modifying therapy to slow progression. We compare data underlying surgical approaches proposed to impart disease modification in PD: (1) cell transplantation therapy with stem cell-derived dopaminergic neurons to replace damaged cells; (2) clinical trials of growth factors to promote survival of existing dopaminergic neurons; (3) subthalamic nucleus deep brain stimulation early in the course of PD; and (4) abdominal vagotomy to lower risk of potential disease spread from gut to brain. Though targeted to engage potential mechanisms of PD these surgical approaches remain experimental, indicating the difficulty in translating therapeutic concepts into clinical practice. The choice of outcome measures to assess disease modification separate from the symptomatic benefit will be critical to evaluate the effect of the disease-modifying intervention on long-term disease burden, including imaging studies and clinical rating scales, i.e., Unified Parkinson Disease Rating Scale. Therapeutic interventions will require long follow-up times (i.e., 5–10 years) to analyze disease modification compared to symptomatic treatments. The promise of invasive, surgical treatments to achieve disease modification through mechanistic approaches has been constrained by the reality of translating these concepts into effective clinical trials.
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DOI：
10.3233/jpd-179001
发表时间：
2017
期刊：
Journal of Parkinson's disease
影响因子：
--
作者：
Braak H;Del Tredici K
通讯作者：
Del Tredici K
影响因子：
17.1
作者：
Brocker DT;Swan BD;So RQ;Turner DA;Gross RE;Grill WM
通讯作者：
Grill WM
影响因子：
8.6
作者：
Fischer, D. Luke;Sortwell, Caryl E.
通讯作者：
Sortwell, Caryl E.
影响因子：
5.3
作者：
Johnson, Matthew D.;Vitek, Jerrold L.;McIntyre, Cameron C.
通讯作者：
McIntyre, Cameron C.
影响因子：
8.6
作者：
Breen, David P.;Halliday, Glenda M.;Lang, Anthony E.
通讯作者：
Lang, Anthony E.