Browsing by Subject "Study design"

Now showing 1 - 3 of 3
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    Career Development in Pragmatic Clinical Trials to Improve Care for People Living with Dementia
    (Wiley, 2023) Gabbard, Jennifer; Sadarangani, Tina R.; Datta, Rupak; Fabius, Chanee D.; Gettel, Cameron J.; Douglas, Natalie F.; Juckett, Lisa A.; Kiselica, Andrew M.; Murali, Komal Patel; McCarthy, Ellen P.; Torke, Alexia M.; Callahan, Christopher M.; Medicine, School of Medicine
    The growing number of people living with dementia (PLWD) requires a coordinated clinical response to deliver pragmatic, evidence-based interventions in frontline care settings. However, infrastructure to support such a response is lacking. Moreover, there are too few researchers conducting rigorous embedded pragmatic clinical trials (ePCTs) to make the vision of high quality, widely accessible dementia care a reality. National Institute on Aging (NIA) Imbedded Pragmatic Alzheimer's disease and Related Dementias Clinical Trials (IMPACT) Collaboratory seeks to improve the pipeline of early career researchers qualified to lead ePCTs by funding career development awards. Even with support from the Collaboratory, awardees face practical and methodological challenges to success, recently exacerbated by the COVID-19 pandemic. We first describe the training opportunities and support network for the IMPACT CDA recipients. This report then describes the unique career development challenges faced by early-career researchers involved in ePCTs for dementia care. Topics addressed include challenges in establishing a laboratory, academic promotion, mentoring and professional development, and work-life balance. Concrete suggestions to address these challenges are offered for early-career investigators, their mentors, and their supporting institutions. While some of these challenges are faced by researchers in other fields, this report seeks to provide a roadmap for expanding the work of the IMPACT Collaboratory and initiating future efforts to recruit, train, and retain talented early-career researchers involved in ePCTs for dementia care.
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    Rates of adverse and serious adverse events in children with cystic fibrosis
    (Elsevier, 2021) Pittman, Jessica E.; Khan, Umer; Laguna, Theresa A.; Heltshe, Sonya; Goss, Christopher H.; Sanders, Don B.; Pediatrics, School of Medicine
    Background: Cystic fibrosis (CF) is an autosomal recessive disease characterized by chronic sinopulmonary symptoms and chronic gastrointestinal symptoms that begins in infancy. Children with CF are increasingly being included in clinical trials. In order to fully evaluate the impact of new therapies in future clinical trials, an understanding of baseline adverse event (AE) rates in children with CF is needed. To address this, we determined the rates of common AEs in pediatric patients with CF who participated in two clinical trials. Methods: We reviewed AEs for placebo recipients in the AZ0004 study and inhaled tobramycin recipients in the Early Pseudomonas Infection Control (EPIC) clinical trial. AEs were categorized based on Medical Dictionary for Regulatory Activities (MedDRA) coding classifications and pooled into common, batched AE descriptors. AE rates were estimated from negative binomial models according to age groups, severity of lung disease, and season. Results: A total of 433 children had 8,266 total AEs reported, or 18.1 (95% CI 17.0, 19.2) AEs per person per year. Respiratory AEs were the most commonly reported AEs, with a rate of 7.6 events per person-year. The total SAE rate was 0.33 per person per-year. Cough was the most commonly reported respiratory AE, with 61% of subjects reporting at least one episode of cough within 4 months. The rate ratio of any AE was higher in Spring, Fall, and Winter, compared with Summer. Conclusions: AEs occur commonly in pediatric CF clinical trial participants. Season of enrollment could affect AE rates.
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    Research methods in genetic counseling: Statistical approaches and resources
    (Wiley, 2025) Helm, Benjamin M.; Wetherill, Leah; Medical and Molecular Genetics, School of Medicine
    The continuing evolution of the genetic counseling profession necessitates an ongoing reflection on the perceived validity and role of our research in the larger systems we operate in. Despite our need for an analytically inclined professional culture and decision-making process, many genetic counselors may not have the training or support needed to ensure such rigor. In this special issue of the Journal of Genetic Counseling, authors were tasked with providing methodological foundations for genetic counselors navigating various phases of research, to improve the quality of our research output and to incorporate our findings into decision-making in healthcare and non-healthcare settings. In this manuscript, we describe various statistical approaches in lay terms and provide resources for genetic counselors new and seasoned alike. We hope to ease some of the trepidation in applying statistical approaches to genetic counseling research and provide resources to increase the analytical confidence of our workforce. This can increase the validity of the analyses and findings disseminated within and beyond our profession. First, we review some history and foundations of statistical practices that inform study design, sampling, data collection, analysis, and interpretation. Next, we highlight how different study designs inform the choice of data analysis and provide resources for statistical strategy choice. Finally, we provide resources on how to interpret statistical test results, recommend best practices, and highlight common but avoidable misconceptions in statistical interpretation. We hope this review provides a framework for novices in quantitative methodology and provides the language needed to collaborate with analytical/statistical colleagues.