Revisiting study design and methodology for pathogen reduced platelet transfusions: a round table discussion.

Pathogen inactivation/reduction technologies for platelet components have been developed to enhance microbial safety, and many studies have been carried out to determine whether this technique adversely affects the platelet's ability to stop or prevent bleeding. These clinical trials require inclusion of several hundred patients, are costly, and take many years to complete. To address these challenges, a meeting was organized consisting of two expert presentations followed by a roundtable discussion focused on possible new approaches to evaluate the clinical efficacy of pathogen-reduced platelets. The value of laboratory measures to provide information on platelet count after transfusion or to serve as a surrogate for bleeding risk was discussed. Also, other types of trial designs (cluster trials, stepped wedge designs, and Phase 4 postmarketing surveillance studies) as well as a clinically meaningful standardized safety endpoint to evaluate pathogen- reduced platelets were also discussed.