Abstract
Background Standard treatment for Glanzmann thrombasthenia (GT), a severe inherited bleeding
disorder, is platelet transfusion. Recombinant activated factor VII (rFVIIa) is reported
to be effective in GT with platelet antibodies and/or refractoriness to platelet transfusions.
Methods We evaluated rFVIIa effectiveness and safety for the treatment and prevention of
surgical and nonsurgical bleeding in children <18 years old, with or without platelet
antibodies and/or refractoriness, as reported in the GT Registry (GTR). Data were
used from the GTR, an international, multicenter, observational, postmarketing study
of rFVIIa that prospectively collected data on the treatment and outcomes of bleeds
in patients with GT. Only patients with a diagnosis of congenital GT were included
in the registry.
Results Between 2007 and 2011, 27 children were treated for 44 surgical procedures (minor:
36; major: 8); nonsurgical bleeds occurred in 104 patients (599 episodes: severe,
145; moderate, 454; spontaneous, 423; posttraumatic, 176). The effectiveness of treatment
for minor procedures, major procedures, nonsurgical bleeds was 6/6, 1/1, and 75/84
for rFVIIa, 6/6, 2/2, and 64/76 for rFVIIa + antifibrinolytics (AF), 11/12, 1/1, and
162/214 for platelets ± AF, and 5/6, 0/3, and 33/45 for rFVIIa + platelets ± AF. In
all, 25 adverse events were reported in children; no thromboembolic events were reported.
Conclusion For all patients, regardless of platelet antibody or refractoriness status, rFVIIa,
administered with or without platelets (± AF), provided effective hemostasis with
a low frequency of adverse events in surgical, as well as nonsurgical, bleeding in
patients with GT.
clinicaltrials.gov identifier: NCT01476423.
Keywords
recombinant activated factor VII - Glanzmann thrombasthenia - autosomal dominant -
pediatric - registry - observational study
