Platelet transfusion is the standard treatment to control or prevent
bleeding in patients with Glanzmann's
thrombasthenia (GT), but platelets are often unavailable. Recombinant
activated factor VII (
rFVIIa) is an effective alternative to platelets in patients with GT with past/present refractoriness to
platelet transfusions and
antibodies to platelets. However, there is an unmet need for an alternative to platelets in patients without
antibodies. This report summarizes evidence of efficacy and safety of
rFVIIa in patients with GT without refractoriness or
antibodies to platelets from three different sources: the Glanzmann's
Thrombasthenia Registry (GTR), published literature (January 01, 1999 to December 01, 2017), and the Novo Nordisk safety surveillance database. In the GTR, 133 patients received
rFVIIa for the treatment of 333
bleeding episodes and prevention of
bleeding in 157
surgical procedures. Overall efficacy rates were 79 and 88%, respectively, in patients treated for
bleeding episodes or for the prevention of
bleeding during surgery; effectiveness was generally similar across refractoriness/antibody status categories. Median dose per infusion of
rFVIIa was close to that recommended for patients with GT (90 µg/kg). Data from 14 published case reports also demonstrated that
rFVIIa is effective with an acceptable safety profile in patients with GT without
antibodies to platelets. Analysis of adverse events reported in GTR and in Novo Nordisk safety surveillance database did not raise any new safety concerns. These data supported the label extension of
rFVIIa to include cases where platelets are not readily available, which was approved by the European Medicines Agency in December 2018.