دورية أكاديمية
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.
| العنوان: | 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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| المؤلفون: | Jackson CL; Department of Biostatistics and Informatics, Colorado School of Public Health, Aurora, CO, USA.; Department of Immunology and Microbiology, University of Colorado School of Medicine, Aurora, CO, USA., Colborn K; Department of Biostatistics and Informatics, Colorado School of Public Health, Aurora, CO, USA.; Surgical Outcomes and Applied Research Program, Department of Surgery, University of Colorado School of Medicine, University of Colorado Anschutz Medical Campus, Aurora, CO, USA., Gao D; Department of Pediatrics, University of Colorado School of Medicine, Aurora, CO, USA., Rao S; Department of Clinical Sciences, Colorado State University, Fort Collins, CO, USA., Slater HC; Malaria and NTDs, PATH, Seattle, WA, USA.; MRC Centre for Global Infectious Disease Analysis, Imperial College London, London, UK., Parikh S; Department of Epidemiology of Microbial Diseases, Yale School of Public Health, New Haven, CT, USA., Foy BD; Department of Microbiology, Immunology, and Pathology, Colorado State University, Fort Collins, CO, USA., Kittelson J; Department of Biostatistics and Informatics, Colorado School of Public Health, Aurora, CO, USA. |
| المصدر: | Clinical trials (London, England) [Clin Trials] 2021 Oct; Vol. 18 (5), pp. 582-593. Date of Electronic Publication: 2021 Jul 03. |
| نوع المنشور: | Journal Article; Research Support, N.I.H., Extramural |
| اللغة: | English |
| بيانات الدورية: | Publisher: SAGE Publications Country of Publication: England NLM ID: 101197451 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1740-7753 (Electronic) Linking ISSN: 17407745 NLM ISO Abbreviation: Clin Trials Subsets: MEDLINE |
| أسماء مطبوعة: | Publication: London : SAGE Publications Original Publication: London : Arnold, c2004- |
| مواضيع طبية MeSH: | Ivermectin* , Malaria*/drug therapy , Malaria*/prevention & control, Cluster Analysis ; Follow-Up Studies ; Humans ; Mass Drug Administration ; Randomized Controlled Trials as Topic ; Research Design ; Sample Size |
| مستخلص: | Background: 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. Methods: 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. Results: 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. Conclusion: 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 the 2-year parallel follow-up of repeated ivermectin mass drug administrations for control of malaria is already underway as a 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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| معلومات مُعتمدة: | U01 AI138910 United States AI NIAID NIH HHS; UL1 TR001863 United States TR NCATS NIH HHS |
| فهرسة مساهمة: | Keywords: Cluster-randomized trials; count data; malaria; small sample size; vector-borne diseases |
| المشرفين على المادة: | 70288-86-7 (Ivermectin) |
| تواريخ الأحداث: | Date Created: 20210705 Date Completed: 20211125 Latest Revision: 20220327 |
| رمز التحديث: | 20221213 |
| مُعرف محوري في PubMed: | PMC8478782 |
| DOI: | 10.1177/17407745211028581 |
| PMID: | 34218684 |
| قاعدة البيانات: | MEDLINE |
Full Text Finder | تدمد: | 1740-7753 |
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| DOI: | 10.1177/17407745211028581 |