ArticleClinical trials (London, England)2024
Design of a clinical trial using generalized pairwise comparisons to test a less intensive treatment regimen.
Article in Clinical trials (London, England), 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 7 papers.
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Who cites it
7 citing papers in PubMed.
- Comparing Missing Data Strategies for Generalized Pairwise Comparisons in Randomized Clinical Trials: A Simulation Study.Statistics in medicine · 2026Article
- Generalized Pairwise Comparisons in Dose Optimization Oncology Trials: Beyond Safety to Multi-outcome Dose Selection.Clinical cancer research : an official journal of the American Association for Cancer Research · 2026Article
- Benefit-risk balance of S-1 versus UFT as adjuvant chemotherapy for stage II/III rectal cancer (JFMC35-C1: ACTS-RC).The oncologist · 2026Article
- Making Sense of Hierarchical Composite End Points in Randomized Clinical Trials-A Primer for Infectious Diseases Clinicians and Researchers.Clinical infectious diseases : an official publication of the Infectious Diseases Society of America · 2025Review
- The Rethinking Clinical Trials (REaCT) Program: A Pragmatic Research Strategy to Improve Cancer Care for Patients, Caregivers, and Healthcare Systems.Current oncology (Toronto, Ont.) · 2025Review
- Justification, margin values, and analysis populations for oncologic noninferiority and equivalence trials: a meta-epidemiological study.Journal of the National Cancer Institute · 2025Article
- Efficacy and safety of short-course radiotherapy versus total neoadjuvant therapy in older rectal cancer patients: a randomised pragmatic trial (SHAPERS).ESMO gastrointestinal oncology · 2024Article
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8 authors.
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No grant is acknowledged in the PubMed record.
Abstract
BACKGROUND/
aimsShowing "similar efficacy" of a less intensive treatment typically requires a non-inferiority trial. Yet such trials may be challenging to design and conduct. In acute promyelocytic leukemia, great progress has been achieved with the introduction of targeted therapies, but toxicity remains a major clinical issue. There is a pressing need to show the favorable benefit/risk of less intensive treatment regimens.
methodsWe designed a clinical trial that uses generalized pairwise comparisons of five prioritized outcomes (alive and event-free at 2 years, grade 3/4 documented infections, differentiation syndrome, hepatotoxicity, and neuropathy) to confirm a favorable benefit/risk of a less intensive treatment regimen. We conducted simulations based on historical data and assumptions about the differences expected between the standard of care and the less intensive treatment regimen to calculate the sample size required to have high power to show a positive Net Treatment Benefit in favor of the less intensive treatment regimen.
resultsAcross 10,000 simulations, average sample sizes of 260 to 300 patients are required for a trial using generalized pairwise comparisons to detect typical Net Treatment Benefits of 0.19 (interquartile range 0.14-0.23 for a sample size of 280). The Net Treatment Benefit is interpreted as a difference between the probability of doing better on the less intensive treatment regimen than on the standard of care, minus the probability of the opposite situation. A Net Treatment Benefit of 0.19 translates to a number needed to treat of about 5.3 patients (1/0.19 ≃ 5.3).
conclusionGeneralized pairwise comparisons allow for simultaneous assessment of efficacy and safety, with priority given to the former. The sample size required would be of the order of 300 patients, as compared with more than 700 patients for a non-inferiority trial using a margin of 4% against the less intensive treatment regimen for the absolute difference in event-free survival at 2 years, as considered here.
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