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Meta-Analysis of Placebo-Treated Patients: Dropout Rates From Treatment in MASH Randomised Controlled Trials.
Matheus Souza1, Marcio J M Amaral1, Luan C V Lima1
1Department of Internal Medicine, Federal University of Rio de Janeiro, Rio de Janeiro, Brazil.
Alimentary Pharmacology & Therapeutics
|January 14, 2025
Summary
Dropout in metabolic dysfunction-associated steatohepatitis (MASH) randomized controlled trials (RCTs) is significant, primarily driven by patient choice. Longer trial durations and later phases are associated with higher dropout rates, impacting study power.
Area of Science:
- Clinical Trials
- Hepatology
- Pharmacology
Background:
- Dropout is a common issue in randomized controlled trials (RCTs), potentially compromising statistical power and randomization balance.
- Metabolic dysfunction-associated steatohepatitis (MASH) is a growing health concern, necessitating effective treatments and rigorous clinical trial evaluations.
Purpose of the Study:
- To quantify the dropout rate in MASH RCTs.
- To identify factors influencing dropout, specifically in placebo-treated participants.
Main Methods:
- A systematic search of PubMed and Cochrane databases was conducted for phase 2-4 MASH RCTs with placebo arms.
- Dropout was defined as patient attrition from the intention-to-treat analysis before treatment completion.
- A generalized linear mixed model was employed to estimate pooled dropout rates.
Main Results:
- Analysis of 60 MASH RCTs (3230 placebo participants) revealed an overall dropout rate of 11.06%.
- Dropout rates were higher in phase 3-4 trials compared to phase 2 trials, and increased with longer treatment durations.
- Patient choice (4.06%) was the leading cause of dropout, followed by adverse events (2.41%) and loss to follow-up (1.79%).
Conclusions:
- Placebo dropout in MASH RCTs is substantial and significantly influenced by patient choice.
- Trial phase and treatment duration are critical factors to consider for accurate sample size calculations in future MASH clinical trials.
Keywords:
MASLDNAFLDdropoutmetabolic dysfunction‐associated steatotic liver diseasenon‐alcoholic fatty liver diseaseMore Related Videos
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