title: Use of recombinant activated factor VII in patients with Glanzmann's thrombasthenia: a review of the literature.
abstract: 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.
authors.affiliation: Division of Hematology/Oncology, Carman and Ann Adams Department of Pediatrics, Children's Hospital of Michigan, Detroit, MI, USA; Wayne State University, Detroit, MI, USA.
authors.forename: M
authors.initials: M
authors.lastname: Rajpurkar
authors.forename: M
authors.initials: M
authors.lastname: Chitlur
authors.forename: M
authors.initials: M
authors.lastname: Recht
authors.forename: D L
authors.initials: DL
authors.lastname: Cooper
mesh_terms: D000906:Antibodies / Q000276:immunology; D015942:Factor VIIa / Q000009:adverse effects / Q000276:immunology / Q000627:therapeutic use*; D006801:Humans; D011994:Recombinant Proteins / Q000009:adverse effects / Q000276:immunology / Q000627:therapeutic use; D012449:Safety; D013915:Thrombasthenia / Q000188:drug therapy* / Q000276:immunology; D017211:Treatment Failure
chemical_list: D000906:Antibodies; D011994:Recombinant Proteins; C103587:recombinant FVIIa; D015942:Factor VIIa
keywords: Glanzmann's thrombasthenia; bleeding; rFVIIa