title: Recombinant FVIIa concentrate-associated thrombotic events in congenital bleeding disorders other than hemophilias.
abstract: Recombinant FVIIa concentrate has been originally used in the treatment of hemophilia patients with inhibitors. Recently, its use has been expanded to a variety of off-label indications. Thrombosis is the most important side effect. This may occur especially in off-label use  but also in hemophiliacs with inhibitors. The present study investigated the occurrence of thrombosis in congenital bleeding disorders other than hemophilias as gathered from personal files and from the literature. Fifteen patients (seven FVII deficiency, one fibrinogen defect, four FXI deficiency,  one von Willebrand disease, and two Glanzmann's Thrombasthenia) have been evaluated. Thrombosis was arterial in eight instances, venous in six, whereas in one case the type of thrombosis was unspecified. In eight cases, associated risk factors were present. Two patients with FXI deficiency  had inhibitors. Dosage was variable. There was at least one fatality but in five cases evolution was not reported. The remaining patients recovered with variable sequels.
authors.affiliation: Department of Medicine, University of Padua, Medical School, Padua, Italy. antonio.girolami@unipd.it
authors.forename: Antonio
authors.initials: A
authors.lastname: Girolami
authors.forename: Giulia Berti
authors.initials: GB
authors.lastname: de Marinis
authors.forename: Emanuela
authors.initials: E
authors.lastname: Bonamigo
authors.forename: Anna Maria
authors.initials: AM
authors.lastname: Lombardi
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D025861:Blood Coagulation Disorders, Inherited / Q000150:complications* / Q000188:drug therapy; D002648:Child; D002675:Child, Preschool; D015942:Factor VIIa / Q000008:administration & dosage / Q000009:adverse effects* / Q000627:therapeutic use; D005260:Female; D006467:Hemophilia A; D006801:Humans; D008297:Male; D008875:Middle Aged; D011994:Recombinant Proteins / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use; D012307:Risk Factors; D013927:Thrombosis / Q000139:chemically induced*; D055815:Young Adult
chemical_list: D011994:Recombinant Proteins; D015942:Factor VIIa