Design of clinical trials to evaluate the efficacy of platelet transfusion: The euroSPRITE trial for components treated with helinx ™ technology

Abstract

Methods of collection, preparation, and transfusion of platelet components have evolved markedly since the introduction of modern platelet transfusion therapy three decades ago. Despite these improvements, few randomized, prospective, controlled studies have been conducted to evaluate the effects of these innovations on the outcome of platelet transfusion—prevention and treatment of bleeding due to thrombocytopenia. The majority of studies have used posttransfusion platelet count increments (CIs) as the primary outcome variable rather than bleeding assessments. In general, these studies have only examined average values for platelet CIs or adjusted ratio measures such as corrected count increment (CCI). Because platelet transfusions are given repeatedly over variable periods of time, this type of analysis has not provided information about the effects of multiple platelet transfusions or about specific product or patient-related covariates that may impact the outcome. Longitudinal regression analysis of platelet CIs offers the potential to provide more information than simple average values of ratio measures. The euroSPRITE trial, a European, multicenter, phase III study undertaken to assess the clinical efficacy and safety of platelets prepared with Helinx ™ technology (Cerus Corp, Concord, CA), used longitudinal regression analysis to characterize more fully the response to platelet transfusions with products prepared with this new pathogen inactivation technology. In contrast to previous studies, the euroSPRITE study examined peritransfusion hemostasis and global indices of hemostasis to correlate the effect of platelet CI with prevention and treatment of bleeding during a period of platelet transfusion support.