Bayesian Statistical Methods in Stem Cell Transplantation and Cellular Therapy

A brief overview of Bayesian statistics is given, followed by illustrative applications of Bayesian methods in allogeneic hematopoietic cell transplantation (allo-HCT) and cellular therapy. Three clinical trial designs are described, including a study to evaluate safety and efficacy of cytotoxic T-lymphocytes for posttransplant viral infections, a trial to optimize chimeric antigen receptor T-cell dose for hematologic malignancies based on efficacy-toxicity trade-offs, and a randomized study of cord blood derived regulatory T-cells for COVID-19 induced acute respiratory distress syndrome. Three data analyses are described, including a sensitivity analysis of preparative regimen effects for allo-HCT that are confounded with institutional effects, regression-based estimation of the effects on survival of antigens in convalescent plasma therapy for COVID-19, and precision pharmacokinetic-guided dosing of intravenous busulfan in allo-HCT.