Recombinant activated factor VII (NovoSeven) treatment of platelet-related bleeding disorders. International Registry on Recombinant Factor VIIa and Congenital Platelet Disorders Group.

Recombinant activated factor VII (rFVIIa; NovoSeven, Novo Nordisk A/S, Bagsvaerd, Denmark), used extensively for the management of hemophilia patients with inhibitors, has also been shown to be effective in the treatment of severe bleeding episodes and for coverage of surgical procedures in patients with platelet disorders. Cases include seven patients with congenital platelet disorders [Glanzmann thrombasthenia (n = 5), Bernard-Soulier syndrome (n = 1), platelet type (pseudo-) von Willebrand disease (n = 1)] and two patients with acquired thrombocytopathy associated with myelodysplastic syndrome and uremia. The clinical efficacy of rFVIIa in functional platelet disorders has been reported as good or excellent, although some cases of ineffectiveness exist. The agent is well tolerated with a single published case of thromboembolism as a postoperative complication. In addition to these reported cases, there are others that remain unreported and unpublished. An International Registry on Recombinant Factor VIIa and Congenital Platelet Disorders (forms in Appendix 1) has been established to obtain more safety and efficacy data on patients with congenital platelet disorders treated with NovoSeven. Analysis of data from this larger population will allow better comprehension of the role of NovoSeven in these disorders, and assist in the design of formal studies to address issues associated with the treatment of these disorders.