Recombinant factor VIIa (
NovoSeven; also known as recombinant
activated factor VII or
eptacog alfa) is structurally similar to human plasma-derived
coagulation factor VIIa, but is manufactured using
DNA biotechnology.
Recombinant factor VIIa interacts with
thrombin-activated platelets to produce a
thrombin burst leading to accelerated
fibrin clot formation localized to the site of
vascular injury. It is approved in many countries for use as an intravenous
hemostatic agent in patients with congenital
hemophilia with inhibitors, and also for acquired
hemophilia,
factor VII deficiency, and
Glanzmann thrombasthenia in some countries. Studies have shown it to be effective and generally well tolerated when used intravenously to treat
bleeding episodes or provide
hemostatic cover during surgery in patients with congenital
hemophilia with inhibitors, acquired
hemophilia,
factor VII deficiency or
Glanzmann thrombasthenia. Based on available data, its efficacy in terms of patient-assessed response may be similar to that of activated
prothrombin complex concentrate (aPCC), but treatment with a single 270 microg/kg dose of
recombinant factor VIIa might reduce the need for rescue
therapy compared with aPCC.
Recombinant factor VIIa is not immunogenic in patients with
hemophilia, does not produce an anamnestic response in
hemophilia patients with inhibitors, and has very low thrombogenicity. It is recommended in guidelines as the treatment of choice for bleeds in patients with
hemophilia B with high-responding inhibitors and for patients with
factor VII deficiency, and is also a first-line therapeutic option for high-responder
hemophilia A patients with inhibitors and those with acquired
hemophilia. Cost data from pharmacoeconomic analyses support its use in
hemophilia patients with inhibitors. Thus,
recombinant factor VIIa is a valuable treatment option for patients with these rare, but potentially serious,
bleeding disorders.