Recombinant factor VIIa (
rFVIIa) was developed for the treatment of
bleeding in
haemophilia patients with inhibitors and has also been used successfully in non-
haemophilia patients with acquired
antibodies against FVIII (acquired
haemophilia). Based on dose-finding trials and a compassionate-use programme,
rFVIIa was approved for use in
haemophilia patients with inhibitors in 1996. At pharmacological doses,
rFVIIa has been found to enhance
thrombin generation on already activated platelets. Therefore, it is likely that
rFVIIa will also be beneficial in providing haemostasis in other situations characterised by profuse bleedings and an impaired
thrombin generation. Patients with
thrombocytopenia have a decreased number of platelets and thus an impaired
thrombin generation. A reduction in bleeding time was reported in approximately 50% of patients with
thrombocytopenia and a prolonged bleeding time who participated in a trial of
rFVIIa. Moreover, in 8 patients with 9 overt bleeds who were involved in the study,
bleeding stopped in 7 episodes after
rFVIIa administration. Case reports on the haemostatic effect of
rFVIIa in
thrombocytopenia have also been published. Reports have also been published on the successful use of
rFVIIa in patients with platelet function deficiencies such as Glanzmann's
thrombasthenia and
Bernard-Soulier syndrome. A number of haemostatic changes occur after extensive trauma, surgery and
bleeding, all of which potentially contribute to an impaired
thrombin generation. The effect of
rFVIIa has been demonstrated in a number of patients after
trauma and bleeds and upper gastrointestinal
bleeding episodes. Reports on the beneficial use of
rFVIIa in
liver transplantation have also been published. Several randomised blinded studies are now underway in e.g.
hepatectomy, upper gastrointestinal bleedings,
transplantations and intra-cerebral bleeds. In summary,
rFVIIa may be an effective and safe method to induce haemostasis in patients within areas of
coagulation factor deficiency or platelet disorders and the ongoing and planned randomised studies may lead the way to the use of
rFVIIa in general haemostasis.