RESUMEN
Importance: It is unknown whether adenotonsillectomy causes undesirable weight gain in children with mild obstructive sleep-disordered breathing (oSDB). Objective: To compare changes in anthropometric measures in children with mild oSDB treated with adenotonsillectomy vs watchful waiting. Design, Setting, and Participants: This was an exploratory analysis of the Pediatric Adenotonsillectomy Trial for Snoring (PATS) randomized clinical trial of adenotonsillectomy vs watchful waiting for mild oSDB (snoring with obstructive apnea-hypopnea index of <3 events/hour) that took place at 7 pediatric tertiary care centers across the US and included 458 children aged 3.0 to 12.9 years with mild oSDB. Participants were recruited from June 29, 2016, to February 1, 2021. Anthropomorphic measures taken at baseline and 12 months after randomization were standardized for age and sex, including each participant's percentage of the 95th body mass index percentile (%BMIp95). Data analyses were performed from March 15, 2023, to April 1, 2024. Intervention: Early adenotonsillectomy (eAT) vs watchful waiting with supportive care (WWSC). Main Outcomes and Measures: Twelve-month change in %BMIp95 from baseline and undesirable weight gain (defined as any weight gain in a child who already had overweight or obesity or an increase from baseline normal weight/underweight to overweight/obesity) at follow-up assessment. Results: The study analysis included 375 children (mean [SD] age, 6.1 [2.3] years; 188 [50.2%] females), of whom 143 (38%) had overweight or obesity at baseline. At 12 months, children in the eAT group experienced a 1.25-point increase in %BMIp95 compared with a 0.59-point increase in the WWSC group (mean difference, 0.93; 95% CI, -0.39 to 2.25). Undesirable weight gain was also similar between the eAT (n = 120; 32%) and WWSC (n = 101; 27%) groups (mean difference, 4%; 95% CI, 5% to 14%). Conclusions and Relevance: The findings of this exploratory analysis of the PATS trial indicate that adenotonsillectomy was not independently associated with an increased risk of undesirable weight gain in children with mild oSDB. However, one-third of the children gained undesirable weight during the study, which suggests that there is an opportunity to address healthy weight management during the evaluation and treatment of children with mild oSDB. Trial Registration: ClinicalTrials.gov Identifier: NCT02562040.
RESUMEN
Both individually and cluster randomized study designs have been used for vaccine trials to assess the effects of vaccine on reducing the risk of disease or infection. The choice between individually and cluster randomized designs is often driven by the target estimand of interest (eg, direct versus total), statistical power, and, importantly, logistic feasibility. To combat emerging infectious disease threats, especially when the number of events from one single trial may not be adequate to obtain vaccine effect estimates with a desired level of precision, it may be necessary to combine information across multiple trials. In this article, we propose a model formulation to estimate the direct, indirect, total, and overall vaccine effects combining data from trials with two types of study designs: individual-randomization and cluster-randomization, based on a Cox proportional hazards model, where the hazard of infection depends on both vaccine status of the individual as well as the vaccine status of the other individuals in the same cluster. We illustrate the use of the proposed model and assess the potential efficiency gain from combining data from multiple trials, compared to using data from each individual trial alone, through two simulation studies, one of which is designed based on a cholera vaccine trial previously carried out in Matlab, Bangladesh.