Standard treatment for
Glanzmann thrombasthenia is
platelet transfusion. Recombinant
activated factor VII has been shown to be successful in patients with
Glanzmann thrombasthenia with platelet
antibodies or who are refractory to
platelet transfusions. The
Glanzmann Thrombasthenia Registry prospectively collected worldwide information on the effectiveness and safety of
platelet transfusion, recombinant
activated factor VII and/or
antifibrinolytics for the treatment of bleeds in patients with
Glanzmann thrombasthenia. Data relating to 829 non-surgical
bleeding episodes were entered into the
Glanzmann Thrombasthenia Registry (severe/moderate: 216/613; spontaneous/post-traumatic: 630/199). Recombinant
activated factor VII alone was used in 124/829 bleeds, recombinant activated factor VII+antifibrinolytics in 107/829, platelets±antifibrinolytics in 312/829,
antifibrinolytics alone in 219/829, and recombinant activated factor VII+platelets±antifibrinolytics in 67/829. The proportion of successful treatments to stop
bleeding was 91.0% in cases treated with recombinant
activated factor VII only, 82.7% for recombinant activated factor VII+antifibrinolytics, 72.7% for treatment with recombinant activated factor VII+platelets±antifibrinolytics, 78.8% for platelets±antifibrinolytics and 84.7% for
antifibrinolytics alone. Treatment failure was documented in 18
bleeding events (2% of the total treatments), the majority of which were in patients receiving treatment with
antifibrinolytics;
bleeding re-started in 6% of bleeds after initial effective treatment. Thirty-five adverse events were reported, none of which was a thromboembolic event. Among treatments that included recombinant
activated factor VII, only one patient reported three possibly
drug-related non-serious adverse events (
nausea,
dyspnea and
headache). To conclude, non-surgical bleeds were common and often severe in
Glanzmann thrombasthenia; both platelets and recombinant
activated factor VII appeared to be effective, and with good safety profiles, for the treatment of non-surgical bleeds. This trial was registered at clinicaltrials.gov identifier: NCT01476423.