Recombinant factor VIIa (rVIIa) has proved effective for the treatment and prevention of
hemorrhage in patients with inherited
hemophilia A and B who develop inhibitors to
factor VIII or IX, and patients with acquired
hemophilia A. More recently, there is evidence that rVIIa may also be effective in the control of abnormal
bleeding in a variety of other conditions, such as inherited
factor VII deficiency,
thrombocytopenia, Glanzmann's
thrombasthenia, and
liver disease. In some of the reports, rVIIa appeared to be effective in controlling massive
hemorrhage in which there was no response to conventional measures. It is now considered by some to be potentially the first universal
hemostatic agent. However, further prospective, controlled, and adequately powered clinical studies are clearly required. It will be of particular interest to determine the efficacy of rVIIa in conditions such as severe
thrombocytopenia, severe
von Willebrand disease, severe defects in platelet activation, and severe deficiencies of factors V, X, II, and
fibrinogen in which effectiveness would seem to be unlikely based on our current understanding of mechanisms of action of rVIIa.