title: The international, prospective Glanzmann Thrombasthenia Registry: treatment modalities and outcomes of non-surgical bleeding episodes in patients with Glanzmann thrombasthenia.
abstract: 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.
authors.affiliation: Department of Clinical Medicine and Surgery, Regional Reference Center for Coagulation Disorders, Federico II University, Naples, Italy diminno@unina.it.
authors.forename: Giovanni
authors.initials: G
authors.lastname: Di Minno
authors.affiliation: Center for Blood Coagulation and Transfusion Medicine (CBT), Dusseldorf, Germany.
authors.forename: Rainer B
authors.initials: RB
authors.lastname: Zotz
authors.affiliation: Regional Reference Centre for Hemophilia and Constitutional Bleeding Disorders, Hôpital Bicêtre AP-HP, University Paris XI, Le Kremlin-Bicêtre, France.
authors.forename: Roseline
authors.initials: R
authors.lastname: d'Oiron
authors.affiliation: Biostatistics, Novo Nordisk A/S, Vandtårnsvej 108-110, DK-2860 Søborg, Denmark.
authors.forename: Niels
authors.initials: N
authors.lastname: Bindslev
authors.affiliation: Department of Clinical Medicine and Surgery, Regional Reference Center for Coagulation Disorders, Federico II University, Naples, Italy Unit of Cell and Molecular Biology in Cardiovascular Diseases, Centro Cardiologico Monzino, IRCCS, Milan, Italy.
authors.forename: Matteo Nicola Dario
authors.initials: MN
authors.lastname: Di Minno
authors.affiliation: Departments of Medicine, Pediatrics and Oncology, University of Calgary, Southern Alberta Rare Blood and Bleeding Disorders Comprehensive Care Program, Foothills Hospital, Calgary, Canada.
authors.forename: Man-Chiu
authors.initials: MC
authors.lastname: Poon
mesh_terms: D000933:Antifibrinolytic Agents / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use; D004334:Drug Administration Schedule; D015942:Factor VIIa / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use; D006470:Hemorrhage / Q000175:diagnosis / Q000209:etiology* / Q000628:therapy*; D006801:Humans; D017713:Platelet Transfusion / Q000009:adverse effects; D011994:Recombinant Proteins / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use; D012042:Registries; D012720:Severity of Illness Index; D013915:Thrombasthenia / Q000150:complications*; D017211:Treatment Failure; D016896:Treatment Outcome
chemical_list: D000933:Antifibrinolytic Agents; D011994:Recombinant Proteins; C103587:recombinant FVIIa; D015942:Factor VIIa