The international prospective Glanzmann Thrombasthenia Registry: treatment and outcomes in surgical intervention

Independent adjudicator assessments of platelet refractoriness and rFVIIa efficacy in bleeding episodes and surgeries from the multinational Glanzmann's thrombasthenia registry

“…A detailed description of the GTR data collection process has been presented elsewhere . Briefly, the registry was a prospective, observational, multinational, Web‐based platform that collected safety and efficacy data regarding the use of systemic hemostatic treatments in patients with GT (see Supporting Information appendix for a list of data fields).…”

“…Briefly, the registry was a prospective, observational, multinational, Web‐based platform that collected safety and efficacy data regarding the use of systemic hemostatic treatments in patients with GT (see Supporting Information appendix for a list of data fields). Patients were eligible for participation in the GTR if they had a diagnosis of congenital GT and exhibited a normal platelet count and morphology . Congenital GT was defined by having a lifelong bleeding tendency characterized by impaired or absent platelet aggregation, impaired clot retraction and prolonged bleeding time, or prolonged platelet function analyzer closure time; optional diagnostic criteria were quantitative or qualitative evaluation of GPIIb/IIIa receptors, including flow cytometry and identification of gene defects .…”

“…The registry was initiated as a postmarketing surveillance commitment to the European Union's Committee for Medicinal Products for Human Use, following approval by the European Medicines Agency of rFVIIa for the treatment of GT . An external expert group monitored the registry and provided ongoing support of recruitment and analysis, including publication of primary data on the efficacy and safety of rFVIIa associated with specific patient events (bleeding episodes and surgeries) . Investigators found rFVIIa used alone to be effective in a majority of bleeding episodes (111 of 122; 91.0%) and surgical procedures (59 of 62; 95.2%).…”

Management of Abdominoperineal Excision of the Rectum in a Patient with Glanzmann Thrombasthenia

“…Oral tranexamic acid at 15-25 mg/kg TDS should be given during the post-operative period. Recombinant FVIIa has been used in the context of major elective surgery with some success [16], but its efficacy had not been fully evaluated at the time of guideline publication and so it is not formally recommended.…”

“…The effectiveness of rVIIa in the prevention and treatment of surgical bleeding in patients with GT was evaluated via the Glanzmann Thrombasthenia Registry [16]. This international, multi-centre, post-marketing database was compiled between 2007 and 2011 and comprises 96 patients with variable platelet antibody and refractoriness status who were categorized post hoc as having had a major or minor procedure.…”