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 In addition to these reported cases, there are others that remain unreported and unpublished.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.