Glanzmann's
thrombasthenia (GT) is a rare
bleeding disorder characterized by a quantitative or qualitative defect of
glycoprotein IIb/IIIa on the platelet membrane. Managing
bleeding episodes is often difficult, and a variety of modalities have been used, including
platelet transfusions,
recombinant factor VIIa (
rFVIIa), and other supportive care. The aim of this review was to present the clinical experience with
rFVIIa bolus infusion (
rFVIIa BI) for treatment of
bleeding episodes and prevention of
bleeding during
surgical procedures in patients with GT. A literature search was performed to identify
rFVIIa-treated patients with GT. Overall, one international survey, one open-label study, and 40 case reports identified 172
bleeding episodes treated with
rFVIIa and 62 procedures covered with
rFVIIa. In the international survey,
rFVIIa BI was used for 96
bleeding episodes in 59 patients.
Recombinant FVIIa was effective in 76
bleeding episodes (79%). Of 34
surgical procedures, 25 procedures received
rFVIIa BI with 92%
bleeding-prevention efficacy. The open-label study reported 28 patients with 28
rFVIIa BI-treated bleeds, and 26 (93%)
bleeding episodes responded to
rFVIIa. Published case reports revealed that 25 (69%) of 36 bleeds and 27 (96%) of 28 surgeries responded to
rFVIIa BI treatment. Overall, 26 adverse events were reported in 19 patients, including five thromboembolic events in two patients where a possible relationship with
rFVIIa could not be excluded. Two large studies and 40 case reports provide a literature base to support the efficacy and safety of
rFVIIa BI in patients with GT.