神经病学中的 N-of-1 试验：系统评价。
N-of-1 Trials in Neurology: A Systematic Review.
机构信息
From the Department of Neurology (B.C.S., J.B., B.G.M.v.E.), Donders Institute for Brain, Cognition and Behaviour, Donders Center for Medical Neuroscience, and Department of Health Evidence (H.G., G.J.v.d.W.), Radboud University Medical Center, Nijmegen, the Netherlands; Department of Neurology (R.C.G.), University of Rochester Medical Center, NY; Department of Neurology (J.S.), University of Kansas Medical Center, Kansas City; University of Groningen (G.D.), Department of Neurology and Neurosurgery, University Medical Center Groningen, Groningen, the Netherlands; University of Queensland Centre for Clinical Research (J.N.), University of Queensland, Herston, Brisbane, Australia; and Amsterdam UMC (J.R.), University of Amsterdam, Department of Neurology, Amsterdam Neuroscience, Netherlands.
出版信息
Neurology. 2022 Jan 11;98(2):e174-e185. doi: 10.1212/WNL.0000000000012998. Epub 2021 Oct 21.
BACKGROUND AND OBJECTIVES
To perform a systematic review of published N-of-1 trials (e.g., single patient crossover trials) in neurologic disorders, including an assessment of methodologic quality and reporting.
METHODS
We searched PubMed, MEDLINE, and Embase from inception date to the December 1, 2019, for reports on N-of-1 trials in neurologic disorders. Basic trial information on design, disease, intervention, analysis, and treatment success was extracted. Strengths and weaknesses of the N-of-1 trials were assessed with the Consolidated Standards of Reporting Trials extension for N-of-1 trials (CENT) 2015 criteria checklist and the Jadad score as measures of quality and reporting.
RESULTS
We retrieved 40 reports of N-of-1 trials in neurologic disorders (19 individual N-of-1 trials, 21 series of N-of-1 trials). Most N-of-1 trials were performed in neuromuscular and neurodegenerative/movement disorders. Unlike the majority of trials that studied the main symptom(s) of a chronic stable condition, 9 N-of-1 trials studied a stable chronic symptom of a progressive or acute neurologic disorder. Besides pharmacologic interventions, electric stimulation protocols and nutritional products were studied. A mean total CENT score of 20.88 (SD 9.10, range 0-43) and mean total Jadad score of 2.90 (SD 2.15, range 0-5) were found as methodologic measures of quality and reporting across all N-of-1 trials.
DISCUSSION
N-of-1 trials have been reported in numerous neurologic disorders, not only in chronic stable disorders, but also in progressive or acute disorders with a stable symptom. This indicates the emerging therapeutic area of N-of-1 trials in neurology. Methodologic quality and reporting of N-of-1 trials were found to be suboptimal and can easily be improved in future trials by appropriately describing the methods of blinding and randomization and following CENT guidelines. Because most N-of-1 trials remain unreported in medical literature, this systematic review probably represents only the tip of the iceberg of conducted N-of-1 trials in neurologic disorders. In addition to conventional trial designs, N-of-1 trials can help to bridge the gap between research and clinical care by providing an alternative, personalized level 1 evidence base for suitable treatments.
背景与目的
对已发表的神经病学领域 N-of-1 试验（如单病例交叉试验）进行系统评价，包括评估方法学质量和报告。
方法
我们检索了 PubMed、MEDLINE 和 Embase 从成立日期到 2019 年 12 月 1 日的神经病学领域 N-of-1 试验报告。提取了设计、疾病、干预、分析和治疗成功的基本试验信息。使用 CONSORT 扩展的 N-of-1 试验（CENT）2015 核对清单和 Jadad 评分评估 N-of-1 试验的优缺点，以评估质量和报告。
结果
我们检索到 40 篇神经病学领域的 N-of-1 试验报告（19 项单病例 N-of-1 试验，21 项 N-of-1 试验系列）。大多数 N-of-1 试验都在神经肌肉和神经退行性/运动障碍中进行。与大多数研究慢性稳定状态主要症状的试验不同，9 项 N-of-1 试验研究了进行性或急性神经病学疾病的稳定慢性症状。除了药物干预外，还研究了电刺激方案和营养产品。所有 N-of-1 试验的平均总 CENT 得分为 20.88（SD 9.10，范围 0-43），平均总 Jadad 得分为 2.90（SD 2.15，范围 0-5），作为质量和报告的方法学措施。
讨论
N-of-1 试验已在许多神经病学疾病中报告，不仅在慢性稳定疾病中，而且在具有稳定症状的进行性或急性疾病中。这表明 N-of-1 试验在神经病学中的治疗领域正在出现。N-of-1 试验的方法学质量和报告被发现并不理想，通过适当描述盲法和随机化方法并遵循 CENT 指南，未来试验可以很容易地改进。由于大多数 N-of-1 试验在医学文献中仍未报告，因此本系统评价可能仅代表神经病学中进行的 N-of-1 试验的冰山一角。除了传统的试验设计外，N-of-1 试验还可以通过提供适合治疗的替代个性化 1 级证据基础，帮助缩小研究和临床护理之间的差距。
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