Clinical trial opportunities in hemostasis and thrombosis: NHBLI State-of-the-Science symposium.

Clinical trials in hemostasis and thrombosis (HT) are needed to guide medical practice and future research. Providing public support for trials that could have the greatest impact on clinical care has been a major challenge. The National Heart, Lung and Blood Institute (NHLBI) convened a State-of-the-Science meeting in Bethesda on September 14th and 15th, 2009 to identify Phase II and III clinical trials in HT that could have critical impact on healthcare. An oversight committee composed of representatives of the NHLBI and three experienced extramural investigators chose chairs of subcommittees representing six broad areas of investigation in adult and pediatric HT. Chairs were charged with identifying important, feasible proposals. Nineteen trial concepts were presented at this public meeting, followed by open commentary from members of an independent external panel chosen to evaluate the trials and from symposium participants from the wider scientific community. Descriptions of two important clinical trial concepts from each of the six subcommittees are provided in the Supporting Information. Phase II-III clinical trials that could have high impact include studies for treatment of venous thromboembolism (TE) in children and in adults, the potential utility of statins in prophylaxis of TE, prophylaxis of adults with severe hemophilia, management of heparin-induced thrombocytopenia (HIT) and of primary immune thrombocytopenia (ITP). The external panel also provided recommendations concerning infrastructure and approach that could improve the conduct of studies including: development of core organizations with expertise in design of clinical trials, biostatistics, and contract development; funding based on output and milestones; and enhanced investment in coordinating centers.