Abstract:
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It is very important to align a trial’s scientific objective with its estimand, including appropriate testing procedures and estimation of effect size. BELINDA is a randomized phase 3 study of tisagenlecleucel (Kymriah) in adult patients with aggressive B-cell Non-Hodgkin's Lymphoma (NHL) who are relapsed or refractory after frontline therapy and eligible to stem cell transplant (SCT). Patients receiving tisagenlecleucel, a chimeric antigen receptor T-cell (CAR-T) therapy, will undergo a unique cell manufacturing process (~4-6 weeks) before the tisagenlecleucel infusion. Similarly in the control arm, patients will receive platinum-based immunochemotherapy followed in responding patients by SCT. This brings attention to the definition of appropriate estimand, hypothesis testing as well as estimation of the treatment effects based on time-to-event endpoints due to non-proportional hazards. In this poster, we will present this case study and focus on the considerations to connect the scientific objective, testing procedure, and estimation under the framework of estimand.
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