Methodological challenges in pragmatic trials in Alzheimer's disease and related dementias: Opportunities for improvement.
Methodological challenges in pragmatic trials in Alzheimer's disease and related dementias: Opportunities for improvement.
复制标题
DOI：
10.1177/17407745211046672
复制
发表时间：
2022-03
期刊：
影响因子：
--
通讯作者：
Mitchell SL
中科院分区：
文献类型：
--
作者：
Taljaard M;Li F;Qin B;Cui C;Zhang L;Nicholls SG;Carroll K;Mitchell SL
我们需要更多务实的干预试验，以改善阿尔茨海默病和相关痴呆症患者的护理和结果。然而，这些试验在其设计、分析和报告中提出了独特的方法挑战——通常是由于一个或多个聚类源的存在。未能在设计和分析中考虑聚类可能导致I型和II型错误的风险增加。我们进行了一项综述，描述了痴呆症研究中实用试验的关键方法学特征，并获得了方法学质量的“基线评估”，以期开发新的方法和实用指导，以支持研究者和方法学家在该领域进行实用试验。我们使用MEDLINE上发布的搜索过滤器来确定更可能是务实的试验，并确定了一个亚组，重点关注阿尔茨海默病或其他痴呆症患者，或将其纳入定义的亚组。成对的审稿人从每个试验中提取描述性信息和关键的方法学质量指标。我们确定了发表在36种不同期刊上的N=62份符合条件的主要试验报告。有15个（24%）单独随机，38个（61%）集群随机和9个（15%）单独随机组治疗设计；54项（87%）试验对同一个体和/或群体进行重复测量，17项（27%）试验采用多变量主要结果（例如，由于同时测量患者及其护理者的结果）。在38个聚类随机试验中，16个（42%）没有报告考虑聚类内相关性的样本量计算，13个（34%）没有考虑分析中的聚类内相关性。在9个单独随机分组治疗试验中，6个（67%）没有报告考虑簇内相关性的样本量计算，8个（89%）没有在分析中考虑到它。在54个重复测量的试验中，45个（83%）没有报告用于重复测量的样本量计算，19个（35%）没有在分析中至少使用一些重复测量。没有试验在样本量计算中考虑其主要结局的多变量性质；在分析中，只有一家公司这样做了。我们有必要也有机会改进痴呆研究中实用试验的设计、分析和报告。调查人员应注意可能存在的一个或多个群集源。虽然纵向和聚类随机试验的方法已经发展得很好，但需要可访问的资源和处理多聚类来源的新方法。建议在纵向和集群设计的专业知识统计学家的参与。
We need more pragmatic trials of interventions to improve care and outcomes for people living with Alzheimer’s disease and related dementias. However, these trials present unique methodological challenges in their design, analysis, and reporting — often, due to the presence of one or more sources of clustering. Failure to account for clustering in the design and analysis can lead to increased risks of type I and type II errors. We conducted a review to describe key methodological characteristics and obtain a “baseline assessment” of methodological quality of pragmatic trials in dementia research, with a view to developing new methods and practical guidance to support investigators and methodologists conducting pragmatic trials in this field. We used a published search filter in MEDLINE to identify trials more likely to be pragmatic and identified a subset that focused on people living with Alzheimer’s disease or other dementias or included them as a defined subgroup. Pairs of reviewers extracted descriptive information and key methodological quality indicators from each trial. We identified N=62 eligible primary trial reports published across 36 different journals. There were 15 (24%) individually randomized, 38 (61%) cluster randomized, and 9 (15%) individually randomized group treatment designs; 54 (87%) trials used repeated measures on the same individual and/or cluster over time and 17 (27%) had a multivariate primary outcome (e.g., due to measuring an outcome on both the patient and their caregiver). Of the 38 cluster randomized trials, 16 (42%) did not report sample size calculations accounting for the intracluster correlation and 13 (34%) did not account for intracluster correlation in the analysis. Of the 9 individually randomized group treatment trials, 6 (67%) did not report sample size calculations accounting for intracluster correlation and 8 (89%) did not account for it in the analysis. Of the 54 trials with repeated measurements, 45 (83%) did not report sample size calculations accounting for repeated measurements and 19 (35%) did not utilize at least some of the repeated measures in the analysis. No trials accounted for the multivariate nature of their primary outcomes in sample size calculation; only one did so in the analysis. There is a need and opportunity to improve the design, analysis, and reporting of pragmatic trials in dementia research. Investigators should pay attention to the potential presence of one or more sources of clustering. While methods for longitudinal and cluster randomized trials are well-developed, accessible resources and new methods for dealing with multiple sources of clustering are required. Involvement of a statistician with expertise in longitudinal and clustered designs is recommended.
登录
查看更多内容
影响因子：
2
作者：
Andridge, Rebecca R.;Shoben, Abigail B.;Muller, Keith E.;Murray, David M.
通讯作者：
Murray, David M.
对群体随机试验的设计和分析的约束随机化评估。
影响因子：
2
作者：
Li F;Lokhnygina Y;Murray DM;Heagerty PJ;DeLong ER
通讯作者：
DeLong ER
影响因子：
2.3
作者：
Hossain A;Diaz-Ordaz K;Bartlett JW
通讯作者：
Bartlett JW
影响因子：
7.7
作者：
Hemming, Karla;Kasza, Jessica;Taljaard, Monica
通讯作者：
Taljaard, Monica
影响因子：
7.2
作者：
Nicholls SG;Carroll K;Hey SP;Zwarenstein M;Zhang JZ;Nix HP;Brehaut JC;McKenzie JE;McDonald S;Weijer C;Fergusson DA;Taljaard M
通讯作者：
Taljaard M