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Randomized clinical trials are the gold standard for evaluating interventions as randomized assignment equalizes known and unknown characteristics between intervention groups. However, when participants miss visits, the ability to conduct an intent-to-treat analysis and draw conclusions about a causal link is compromised. As guidance to those performing clinical trials, this review is a non-technical overview of the consequences of missing data and a prescription for its treatment beyond the typical analytic approaches to the entire research process. Examples of bias from incorrect analysis with missing data and discussion of the advantages/disadvantages of analytic methods are given. As no single analysis is definitive when missing data occurs, strategies for its prevention throughout the course of a trial are presented. We aim to convey an appreciation for how missing data influences results and an understanding of the need for careful consideration of missing data during the design, planning, conduct, and analytic stages.
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Int J Epidemiol
August 2025
Department of Biostatistics and Informatics, University of Colorado, Aurora, CO, United States.
Background: Existing longitudinal cohort study data and associated biospecimen libraries provide abundant opportunities to efficiently examine new hypotheses through retrospective specimen testing. Outcome-dependent sampling (ODS) methods offer a powerful alternative to random sampling when testing all available specimens is not feasible or biospecimen preservation is desired. For repeated binary outcomes, a common ODS approach is to extend the case-control framework to the longitudinal setting.
View Article and Find Full Text PDFJCO Precis Oncol
September 2025
Cell Biology and Biotherapy Unit, Istituto Nazionale Tumori IRCCS Fondazione G. Pascale, Napoli, Italy.
Purpose: Tumor comprehensive genomic profiling (CGP) may detect potential germline pathogenic/likely pathogenic (P/LP) alterations as secondary findings. We analyzed the frequency of potentially germline variants and large rearrangements (LRs) in the RATIONAL study, an Italian multicenter, observational clinical trial that collects next-generation sequencing-based tumor profiling data, and evaluated how these findings were managed by the enrolling centers.
Patients And Methods: Patients prospectively enrolled in the pathway-B of the RATIONAL study and undergoing CGP with the FoundationOne CDx assays were included in the analysis.
PLOS Glob Public Health
September 2025
International Training and Education Center for Health, Seattle, Washington, United States of America.
Retention on antiretroviral therapy (ART) is critical for achieving viral load suppression (VLS) among people living with HIV (PLHIV). Retention remains challenging in high-prevalence settings like Malawi. Short messaging service (SMS) interventions, particularly hybrid two-way texting (2wT), show promise in improving ART retention.
View Article and Find Full Text PDFPLoS One
September 2025
Endocrinology and Metabolism Research Center, Endocrinology and Metabolism Clinical Sciences Institute, Tehran University of Medical Sciences, Tehran, Iran.
Background: The prevalence of Metabolic Syndrome (MetS) increases with aging, significantly contributing to the rising burden of non-communicable diseases (NCDs). This study aimed to investigate over-time changes in the prevalence of MetS and its components among the elderly population of Iran.
Methods: We analyzed data from the 2016 and 2021 national STEPwise approach to non-communicable disease risk factor Surveillance (STEPS) for participants aged ≥65 who completed all three survey steps (questionnaire-based assessments, physical measurements, and laboratory tests) with no missing data on MetS components.
Health Expect
October 2025
JC School of Public Health and Primary Care, The Chinese University of Hong Kong, Hong Kong, China.
Objective: This study examined the content validity of EQ-5D for Chinese patients with DMD. Specifically, it investigated: (1) the content validity of EQ-5D-5L in adult DMD patients and (2) the content validity of EQ-5D-Y-3L (Y-3L) in DMD patients aged 8-15 years.
Method: This qualitative study used semi-structured interviews and conducted one-on-one and online, with two groups of individuals with DMD to examine the content validity of the EQ-5D-5L and Y-3L.