NHLBI Working Group: Hemophilia and Thrombosis Disorders
Executive Summary On November 13, 2009 the National Heart, Lung
and Blood Institute convened a Working Group of senior
scientists from both academia and government to define
mechanisms whereby important clinical research can be developed
and implemented for hemophilia and other rare hemostatic and
thrombotic disorders. The objective was to identify and evaluate
existing data resources, clinical trial capacities, candidate
grant mechanisms and collaborative opportunities. Dr. Keith
Hoots, Director of the Division of Blood Diseases and Resources,
welcomed the participants and requested their advice and
recommendations to advance clinical research. Hemophilia is a
rare bleeding disorder that is caused by a deficiency in either
blood coagulation factor VIII or IX. Current treatment with
infusion of plasma derived or recombinant factor to replace the
missing protein, has greatly reduced morbidity and mortality
associated with the disease. A recent study demo