n-of-1 trial

N - of - 1 试验
  • 文章类型: Journal Article
    研究人员和从业者经常使用单例设计(SCD),或n-of-1试验,开发和验证新的治疗方法。已经发布了标准和指南,以提供有关如何实施SCD的指导,但他们的许多建议并非来自研究文献。例如,其中一项建议表明,研究人员和从业人员应在引入自变量之前等待基线稳定性.然而,这一建议没有得到经验证据的有力支持.为了解决这个问题,我们使用蒙特卡罗模拟来生成具有固定,响应引导,和随机基线长度,同时操纵趋势和变异性。然后,我们的分析比较了两种分析方法产生的I型错误率和功效:保守双标准方法(结构化视觉辅助)和支持向量分类器(源自机器学习的模型).保守的对偶标准方法在使用响应引导决策时产生的错误较少(即,等待稳定性)和随机基线长度。相比之下,等待稳定性并没有减少支持向量分类器的决策错误。我们的发现质疑在使用SCD和机器学习时等待基线稳定性的必要性,但是这项研究必须与其他设计和图形参数重复,这些参数会随着时间的推移而变化,以支持我们的结果。
    Researchers and practitioners often use single-case designs (SCDs), or n-of-1 trials, to develop and validate novel treatments. Standards and guidelines have been published to provide guidance as to how to implement SCDs, but many of their recommendations are not derived from the research literature. For example, one of these recommendations suggests that researchers and practitioners should wait for baseline stability prior to introducing an independent variable. However, this recommendation is not strongly supported by empirical evidence. To address this issue, we used Monte Carlo simulations to generate graphs with fixed, response-guided, and random baseline lengths while manipulating trend and variability. Then, our analyses compared the type I error rate and power produced by two methods of analysis: the conservative dual-criteria method (a structured visual aid) and a support vector classifier (a model derived from machine learning). The conservative dual-criteria method produced fewer errors when using response-guided decision-making (i.e., waiting for stability) and random baseline lengths. In contrast, waiting for stability did not reduce decision-making errors with the support vector classifier. Our findings question the necessity of waiting for baseline stability when using SCDs with machine learning, but the study must be replicated with other designs and graph parameters that change over time to support our results.
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  • 文章类型: Journal Article
    单病例设计(SCD)用于评估干预措施对个体参与者的影响。通过在不同条件下重复测量参与者,SCD研究侧重于个体影响,而不是群体总结。SCD的主要局限性仍然是其对更广泛人群的普遍性,降低他们的发现与实践和政策制定的相关性。考虑到这个限制,在过去的几十年中,从调查同一研究问题的不同研究中合成SCD数据的方法学发展(例如,多层次建模)得到了加强。然而,这些技术仅限于一次比较两种干预措施,并且只能纳入直接比较两种感兴趣治疗方法的研究证据。这些限制可以通过使用结合直接和间接证据的网络荟萃分析来解决,以同时比较多种干预措施。尽管有潜力,网络元分析技术尚未应用于SCD数据。因此,在本文中,我们认为,网络荟萃分析可能是一个有价值的工具,以综合SCD数据。我们使用真实的数据集演示了网络荟萃分析在SCD数据中的使用,我们通过反思SCD研究人员在将网络荟萃分析方法应用于其数据时可能面临的挑战来得出结论。
    Single-case designs (SCDs) are used to evaluate the effects of interventions on individual participants. By repeatedly measuring participants under different conditions, SCD studies focus on individual effects rather than on group summaries. The main limitation of SCDs remains its generalisability to wider populations, reducing the relevance of their findings for practice and policy making. With this limitation in mind, methodological developments for synthesising SCD data from different studies that investigate the same research question have intensified in the past decades (e.g. multilevel modelling). However, these techniques are restricted to comparing two interventions at a time and can only incorporate evidence from studies that directly compare the two treatments of interest. These limitations could be addressed by using network meta-analysis that incorporates both direct and indirect evidence to simultaneously compare multiple interventions. Despite its potential, network meta-analytical techniques have yet to be applied to SCD data. Thus, in this paper, we argue that network meta-analysis can be a valuable tool to synthesise SCD data. We demonstrate the use of network meta-analysis in SCD data using a real dataset, and we conclude by reflecting on the challenges that SCD researchers might face when applying network meta-analysis methods to their data.
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  • 文章类型: Journal Article
    行为分析师通常使用视觉检查来分析单例图,但是对其可靠性的研究产生了不同的结果。为了研究这个问题,我们将I型错误率和视觉检查的能力与一种新颖的方法-机器学习进行了比较。五位专家视觉评估员分析了1,024个模拟AB图,每个阶段的点数不同,自相关,趋势,可变性,和效果大小。将评级与通过保守的双重标准方法和从机器学习得出的两个模型获得的评级进行比较。平均而言,视觉评估者仅在75%的图表上达成一致。相比之下,从机器学习中得出的两个模型都显示出I型错误率和功率之间的最佳平衡,同时在不同的图形特征中产生更一致的结果。结果表明,机器学习可以支持研究人员和从业人员在分析单例图时减少错误。但复制仍然是必要的。
    Behavior analysts commonly use visual inspection to analyze single-case graphs, but studies on its reliability have produced mixed results. To examine this issue, we compared the Type I error rate and power of visual inspection with a novel approach-machine learning. Five expert visual raters analyzed 1,024 simulated AB graphs, which differed on number of points per phase, autocorrelation, trend, variability, and effect size. The ratings were compared to those obtained by the conservative dual-criteria method and two models derived from machine learning. On average, visual raters agreed with each other on only 75% of graphs. In contrast, both models derived from machine learning showed the best balance between Type I error rate and power while producing more consistent results across different graph characteristics. The results suggest that machine learning may support researchers and practitioners in making fewer errors when analyzing single-case graphs, but replications remain necessary.
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  • 文章类型: Journal Article
    简介:患有多动运动障碍的人,包括肌张力障碍,经历往往是痛苦的,影响功能的非自愿运动。座位舒适度是家庭确定的关键未满足的需求。本文报告了一项方案,以评估动态座椅改善肌张力障碍脑瘫(DCP)幼儿功能结局的可行性和初步证据。设计:一系列单例实验设计N-of-1试验,随着参与者的重复,使用随机基线间隔,和一个治疗期(n=6)。方法:纳入标准:DCP;21.5cm Introduction: People with hyperkinetic movement disorders, including dystonia, experience often painful, involuntary movements affecting functioning. Seating comfort is a key unmet need identified by families. This paper reports a protocol to assess the feasibility and preliminary evidence for the efficacy of dynamic seating to improve functional outcomes for young children with dystonic cerebral palsy (DCP). Design: A series of single-case experimental design N-of-1 trials, with replications across participants, with a random baseline interval, and one treatment period (n = 6). Methods: Inclusion criteria: DCP; 21.5 cm < popliteal fossa to posterior sacrum < 35 cm; Gross Motor Function Classification System level IV-V; mini-Manual Ability Classification System level IV-V; difficulties with seating. Intervention: Trial of the seat (8 weeks), with multiple baseline before, during and after intervention and 2 month follow up. The baseline duration will be randomised per child (2-7 weeks). Primary outcomes: Performance Quality Rating Scale; Canadian Occupational Performance Measure; seating tolerance. The statistician will create the randomization, with allocation concealment by registration of participants prior to sending the allocation arm to the principal investigator. Primary outcomes will be assessed from video by an assessor blind to allocation. Analysis: Participant outcome data will be plotted over time, with parametric and non-parametric analysis including estimated size effect for N-of-1 trials.
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  • 文章类型: Journal Article
    背景:鞭打相关障碍(WAD),一种常见的和致残的情况,给澳大利亚带来了巨大的负担和成本。然而,目前鞭打治疗效果不是很好;迫切需要改善结局.临床指南推荐简单的镇痛(扑热息痛和非甾体抗炎药),但没有指南推荐药物的试验。本研究将调查循证建议(EBA)的有效性,扑热息痛,萘普生,扑热息痛和萘普生,减少日常颈部疼痛和预防鞭打损伤后的慢性颈部疼痛。
    方法:本研究是一系列多周期试验,双盲,随机N-of-1试验,嵌套在多基线设计中。设计将包括三个基线,持续时间为5、8或11天。登记后,参与者将被随机分配到其中一个基线。15名急性(<2周)II级WAD的参与者,经历至少中度疼痛(NRS:≥5/10),并且有康复不良的风险将从昆士兰州的医院招募,澳大利亚,通过当地的物理治疗师。患者将接受EBA加三个周期的随机序列,为期十天的治疗三联(扑热息痛指定为C期,萘普生,指定为D阶段,扑热息痛和萘普生,指定为E相)。
    结论:我们将测试不同治疗方法对每日和伤后4和7个月收集的平均颈部疼痛强度的主要结果的影响。次要结果,包括残疾,抑郁症,创伤后应激症状,痛苦的灾难,和研究程序的可行性,也将进行评估。这项研究的结果将为一项更大的试验提供信息,旨在加强EBA和WAD简单镇痛药的证据。
    背景:临床试验主要注册:澳大利亚和新西兰临床试验注册。
    背景:ACTRN12618001291279。
    31/07/2018。
    昆士兰大学,布里斯班QLD4072澳大利亚。
    背景:昆士兰大学.
    BACKGROUND: Whiplash associated disorder (WAD), a common and disabling condition, incurs huge burden and costs to Australia. Yet, current treatments for whiplash are not very effective; improved outcomes are urgently needed. Clinical guidelines recommend simple analgesia (paracetamol and non-steroidal anti-inflammatory drugs) but there have been no trials of guideline-recommended drugs. This study will investigate the effectiveness of evidence-based advice (EBA), paracetamol, naproxen, and both paracetamol and naproxen, in reducing daily neck pain and preventing chronic neck pain after whiplash injury.
    METHODS: This study is a pilot series of multi-cycle, double-blinded, randomised N-of-1 trials, nested in a multiple baseline design. The design will comprise three baselines of 5, 8 or 11 days duration. Post enrolment, participants will be randomly assigned to one of the baselines. Fifteen participants with acute (<2 weeks) Grade II WAD, experiencing at least moderate pain (NRS: ≥ 5/10), and at risk of poor recovery will be recruited from hospitals in Queensland, Australia, and through local physiotherapists. Patients will receive EBA plus a randomised sequence of three cycles of ten day treatment triplets (paracetamol designated as a C phase, naproxen, designated as a D phase, and both paracetamol and naproxen, designated as an E phase).
    CONCLUSIONS: We will test the effects of different treatments on the primary outcome of average neck pain intensity collected daily and at 4 and 7 months post-injury. Secondary outcomes, including disability, depression, post-traumatic stress symptoms, pain catastrophizing, and feasibility of study procedures, will also be evaluated. The results of this study will inform a larger trial aiming to strengthen the evidence on EBA and simple analgesics for WAD.
    BACKGROUND: Clinical Trials Primary Registry: Australian and New Zealand Clinical Trials Registry.
    BACKGROUND: ACTRN12618001291279.
    UNASSIGNED: 31/07/2018.
    UNASSIGNED: The University of Queensland, Brisbane QLD 4072 Australia.
    BACKGROUND: The University of Queensland.
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  • 文章类型: Journal Article
    发表的报告描述了以单个参与者为特征的干预措施在神经康复中很常见。然而,并非所有此类报告都使用严格的单案例方法,并且有越来越多的证据表明该设计,在行为科学(包括神经康复)中进行和报告单病例研究需要改进。本文的第一部分描述了将指导改进设计的资源,对单一案例研究进行批判性评估,包括最近发布的特殊教育领域的标准和N-of-1试验中的偏差风险量表(RoBiNT),用于使用单个参与者评估设计的内部和外部有效性。本文的第二部分报告了目前在CONSORT传统中制定报告指南的工作,专门针对行为科学中的单案例实验设计,题为“行为干预中的单病例报告指南”(SCRIBE)。预计作者对这些资源的采用和使用,审稿人和期刊编辑将改进报告,潜在的,单案例文献的质量。
    Published reports describing interventions featuring a single participant are common in neurorehabilitation. Yet, not all such reports use rigorous single-case methodology and there is mounting evidence to suggest that the design, conduct and report of single-case research in the behavioural sciences (including neurorehabilitation) needs improvement. The first part of this article describes resources that will guide the improved design, conduct and critical appraisal of single-case research, including recently published standards in the field of special education and the Risk of Bias in N-of-1 Trials (RoBiNT) Scale for evaluating internal and external validity of designs using a single participant. The second part of the article reports on work currently in progress in developing a reporting guideline in the CONSORT tradition specifically for single-case experimental designs in the behavioural sciences, entitled the Single-Case Reporting guideline In BEhavioural interventions (SCRIBE). It is anticipated that adoption and use of these resources by authors, reviewers and journal editors will improve the reporting and, potentially, the quality of the single-case literature.
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