Design and analysis of a 2-year parallel follow-up of repeated ivermectin mass drug administrations for control of malaria: Small sample considerations for cluster-randomized trials with count data.
Design and analysis of a 2-year parallel follow-up of repeated ivermectin mass drug administrations for control of malaria: Small sample considerations for cluster-randomized trials with count data.
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DOI：
10.1177/17407745211028581
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发表时间：
2021-10
期刊：
影响因子：
--
通讯作者：
Kittelson J
中科院分区：
文献类型：
--
作者：
Jackson CL;Colborn K;Gao D;Rao S;Slater HC;Parikh S;Foy BD;Kittelson J
集群随机试验允许评估社区水平或组/集群水平的干预措施。对于需要集群随机试验设计来评估旨在控制媒介传播疾病的集群水平干预措施的研究，在进行监测参与者、媒介和与疾病相关的环境因素所需的额外工作时，可能难以评估大量集群。“反复使用伊维菌素大规模药物控制疟疾的疗效和危害风险”试验就是这样一个集群随机试验的例子。虽然以前的工作为分析诸如反复给药伊维菌素以控制疟疾等试验提供了建议，但对于具有计数结果的研究设计，需要对多种分析方法进行进一步评价。通过一项模拟研究，我们将三种分析框架应用于三个集群随机试验设计（单年、2年平行和2年交叉），在重复伊维菌素大规模用药以控制疟疾的2年平行随访背景下。评估了混合效应模型、广义估计方程和聚类水平分析。另外还探讨了不同簇数和不同簇相关性的2年并行设计。混合效应模型具有小样本校正和未加权的簇级汇总，产生了高功率和控制I型错误率。使用小样本修正的广义估计方程方法控制了I型错误率，但与使用小样本修正的混合模型方法相比，并没有赋予更大的权力。交叉设计通常产生更高的功率相对于并联等效。随着聚类数量的增加，分析方法之间的能力差异变得不那么明显。聚类内相关的强度影响了功率的相对差异。无论研究设计如何，聚类水平分析以及混合效应模型或小样本量修正的广义估计方程等个人水平分析都可以在小聚类设置中提供可靠的结果。对于为控制疟疾而反复给药伊维菌素的2年平行随访，我们建议采用伪似然近似法和Kenward-Roger校正的混合效应模型。设计类似的小样本量和计数结果的研究，在使用混合效应模型或广义估计方程进行分析时，应考虑对小样本量进行调整。虽然为控制疟疾而重复给予伊维菌素大量药物的2年平行随访已经作为2年平行试验进行，但将模拟参数应用于交叉设计产生了更高的功效，这表明交叉设计在可用群集数量有限的情况下可能是有价值的。最后，在选择集群随机试验的主要分析时，应仔细考虑分析方法对集群内相关强度的敏感性。
Cluster-randomized trials allow for the evaluation of a community-level or group-/cluster-level intervention. For studies that require a cluster-randomized trial design to evaluate cluster-level interventions aimed at controlling vector-borne diseases, it may be difficult to assess a large number of clusters while performing the additional work needed to monitor participants, vectors, and environmental factors associated with the disease. One such example of a cluster-randomized trial with few clusters was the “efficacy and risk of harms of repeated ivermectin mass drug administrations for control of malaria” trial. Although previous work has provided recommendations for analyzing trials like repeated ivermectin mass drug administrations for control of malaria, additional evaluation of the multiple approaches for analysis is needed for study designs with count outcomes. Using a simulation study, we applied three analysis frameworks to three cluster-randomized trial designs (single-year, 2-year parallel, and 2-year crossover) in the context of a 2-year parallel follow-up of repeated ivermectin mass drug administrations for control of malaria. Mixed-effects models, generalized estimating equations, and cluster-level analyses were evaluated. Additional 2-year parallel designs with different numbers of clusters and different cluster correlations were also explored. Mixed-effects models with a small sample correction and unweighted cluster-level summaries yielded both high power and control of the Type I error rate. Generalized estimating equation approaches that utilized small sample corrections controlled the Type I error rate but did not confer greater power when compared to a mixed model approach with small sample correction. The crossover design generally yielded higher power relative to the parallel equivalent. Differences in power between analysis methods became less pronounced as the number of clusters increased. The strength of within-cluster correlation impacted the relative differences in power. Regardless of study design, cluster-level analyses as well as individual-level analyses like mixed-effects models or generalized estimating equations with small sample size corrections can both provide reliable results in small cluster settings. For 2-year parallel follow-up of repeated ivermectin mass drug administrations for control of malaria, we recommend a mixed-effects model with a pseudo-likelihood approximation method and Kenward–Roger correction. Similarly designed studies with small sample sizes and count outcomes should consider adjustments for small sample sizes when using a mixed-effects model or generalized estimating equation for analysis. Although 2-year parallel follow-up of repeated ivermectin mass drug administrations for control of malaria is already underway as a 2-year parallel trial, applying the simulation parameters to a crossover design yielded improved power, suggesting that crossover designs may be valuable in settings where the number of available clusters is limited. Finally, the sensitivity of the analysis approach to the strength of within-cluster correlation should be carefully considered when selecting the primary analysis for a cluster-randomized trial.
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影响因子：
2.3
作者：
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通讯作者：
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作者：
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DOI：
10.1080/10705511.2016.1186549
发表时间：
2016-09-01
影响因子：
6
作者：
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通讯作者：
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影响因子：
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作者：
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