Title | Inference on treatment effects from a randomized clinical trial in the presence of premature treatment discontinuation: the SYNERGY trial. |
Publication Type | Journal Article |
Year of Publication | 2011 |
Authors | Zhang, Min, Anastasios A. Tsiatis, Marie Davidian, Karen S. Pieper, and Kenneth W. Mahaffey |
Journal | Biostatistics |
Volume | 12 |
Issue | 2 |
Pagination | 258-69 |
Date Published | 2011 Apr |
ISSN | 1468-4357 |
Keywords | Acute Coronary Syndrome, Algorithms, Computer Simulation, Endpoint Determination, Enoxaparin, Hemorrhage, Heparin, Humans, Intention to Treat Analysis, Models, Statistical, Monte Carlo Method, Mortality, Multicenter Studies as Topic, Probability, Proportional Hazards Models, Randomized Controlled Trials as Topic, Survival Analysis, Treatment Outcome, Withholding Treatment |
Abstract | The Superior Yield of the New Strategy of Enoxaparin, Revascularization, and GlYcoprotein IIb/IIIa inhibitors (SYNERGY) was a randomized, open-label, multicenter clinical trial comparing 2 anticoagulant drugs on the basis of time-to-event endpoints. In contrast to other studies of these agents, the primary, intent-to-treat analysis did not find evidence of a difference, leading to speculation that premature discontinuation of the study agents by some subjects may have attenuated the apparent treatment effect and thus to interest in inference on the difference in survival distributions were all subjects in the population to follow the assigned regimens, with no discontinuation. Such inference is often attempted via ad hoc analyses that are not based on a formal definition of this treatment effect. We use SYNERGY as a context in which to describe how this effect may be conceptualized and to present a statistical framework in which it may be precisely identified, which leads naturally to inferential methods based on inverse probability weighting. |
DOI | 10.1093/biostatistics/kxq054 |
Alternate Journal | Biostatistics |
Original Publication | Inference on treatment effects from a randomized clinical trial in the presence of premature treatment discontinuation: the SYNERGY trial. |
PubMed ID | 20797983 |
PubMed Central ID | PMC3062147 |
Grant List | P01 CA142538-01 / CA / NCI NIH HHS / United States R01CA051962 / CA / NCI NIH HHS / United States R37AI031789 / AI / NIAID NIH HHS / United States R01CA085848 / CA / NCI NIH HHS / United States P01CA142538 / CA / NCI NIH HHS / United States P01 CA142538 / CA / NCI NIH HHS / United States R01 CA085848-11 / CA / NCI NIH HHS / United States |
Inference on treatment effects from a randomized clinical trial in the presence of premature treatment discontinuation: the SYNERGY trial.
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