Tuesday, Aug 6: 9:35 AM - 9:55 AM
Invited Paper Session
Oregon Convention Center
In a six-year Phase III non-inferiority trial, we evaluated dietary interventions for neutropenic patients: a liberalized diet versus a standard one. Our primary goal was to compare the incidence of major infections. Two interim analyses (IAs) were prespecified to guide the continuation or termination of the study. The 1st IA in 2021 allowed the study to continue with some reservations due to infection rates closely approaching the prespecified margin of 10% difference. Despite the p-value from the IA, which suggested the continuation of patient enrollment based on the primary endpoint, we exercised caution. In a proactive move, our statistical team proposed an earlier second IA than the original plan. The results from the newly added IA in Spring 2023 indicated that the infection rate exceeded the threshold, leading to an immediate halt in patient enrollment and trial termination in June 2023. This presentation underscores the vital role of statistical leadership in multidisciplinary research involving multiple stakeholders, showcasing how statisticians prioritized patient care, made data-driven decisions, provided clear recommendations, and influenced clinical trial outcomes.