Use of recombinant activated factor VII in patients with Glanzmann's thrombasthenia: a review of the literature

Summary 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 ( rFVII a), and other supportive care. The aim of this review was to present the clinical experience with rFVII a bolus infusion ( rFVII a BI) for treatment of bleeding episodes and prevention of bleeding during surgical procedures in patients with GT. A literature search was performed to identify rFVII a‐treated patients with GT. Overall, one international survey, one open‐label study, and 40 case reports identified 172 bleeding episodes treated with rFVII a and 62 procedures covered with rFVII a. In the international survey, rFVII a 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 rFVII a BI with 92% bleeding‐prevention efficacy. The open‐label study reported 28 patients with 28 rFVII a BI‐treated bleeds, and 26 (93%) bleeding episodes responded to rFVII a. Published case reports revealed that 25 (69%) of 36 bleeds and 27 (96%) of 28 surgeries responded to rFVII a BI treatment. Overall, 26 adverse events were reported in 19 patients, including five thromboembolic events in two patients where a possible relationship with rFVII a could not be excluded. Two large studies and 40 case reports provide a literature base to support the efficacy and safety of rFVII a BI in patients with GT.