A multidisciplinary "think tank": the top 10 clinical trial opportunities in transfusion medicine from the National Heart, Lung, and Blood Institute-sponsored 2009 state-of-the-science symposium.

Background: In September 2009, the National Heart, Lung, and Blood Institute convened the State-of-the-Science Symposium in Transfusion Medicine to identify Phase II and/or III clinical trials that would provide important information to advance transfusion medicine.

Study Design And Methods: Seven multidisciplinary subcommittees developed proposals in the following areas: 1) platelet (PLT) product use, 2) neonatal and/or pediatric transfusion practice, 3) surgical transfusion practice, 4) intensive care unit and/or in trauma transfusion practice, 5) plasma and/or cryoprecipitate product use and therapeutic apheresis practice, 6) red blood cell (RBC) product use and/or blood conservation management, and 7) medical transfusion practice or blood donor studies. The committees consisted of transfusion medicine specialists, hematologists, cardiovascular surgeons, anesthesiologists, neonatologists, critical care physicians, and clinical trial methodologists. Proposals were presented and an external panel evaluated and prioritized each concept for scientific merit, clinical importance, and feasibility.

Results: Twenty-four concepts were presented by the subcommittees. Ten concepts addressed four areas deemed most important: 1) PLT transfusion strategies to prevent and/or mitigate bleeding in neonates and patients with hematologic malignancies, 2) RBC transfusion trigger strategies to improve overall outcomes in different patient populations, 3) evaluation of optimal plasma:PLT:RBC ratios in trauma resuscitation, and 4) pathogen inactivation of PLTs to improve PLT transfusion safety.

Conclusions: The proposal themes not only represent inquiries about the indications for transfusion, but also epitomize the lack of consensus when clinical practice lacks a strong evidence base. Ultimately, the purpose of this publication is to provide a "blueprint" of ideas for further development rather than endorse any one specific clinical trial design.