title: Human coagulation factor FVIIa (recombinant) in the management of limb-threatening bleeds unresponsive to alternative therapies: results from the NovoSeven emergency-use programme in patients with severe haemophilia or with acquired inhibitors.
abstract: This open-label, emergency-use study evaluated the efficacy and safety of activated human coagulation factor VIIa (recombinant) (rFVIIa) (NovoSeven; Novo Nordisk Pharmaceuticals, Inc., New Jersey, USA) in treating limb-threatening joint or muscle bleeds in 17 patients with haemophilia A or B and six patients with acquired inhibitors to factor VIII or factor IX. All patients had previously failed on one or more alternative therapies. rFVIIa administration was effective or partially effective in controlling joint or muscle bleeds in 34 out of 35 (97%) bleeding episodes; in 23 patients, 14 of 17 (82%) muscle bleeds and 16 of 18 (89%) joint bleeds were effectively controlled. These findings suggest that rFVIIa is an effective and well-tolerated therapeutic option in the management of joint or muscle haemorrhage in patients with haemophilia and in patients with acquired inhibitors.
authors.affiliation: Division of Pediatrics, Haemotology/Oncology, Mount Sinai School of Medicine, New York, New York 10029, USA. steven.arkin@mssm.edu
authors.forename: S
authors.initials: S
authors.lastname: Arkin
authors.forename: F
authors.initials: F
authors.lastname: Blei
authors.forename: J
authors.initials: J
authors.lastname: Fetten
authors.forename: R
authors.initials: R
authors.lastname: Foulke
authors.forename: G S
authors.initials: GS
authors.lastname: Gilchrist
authors.forename: M A
authors.initials: MA
authors.lastname: Heisel
authors.forename: N
authors.initials: N
authors.lastname: Key
authors.forename: C T
authors.initials: CT
authors.lastname: Kisker
authors.forename: C
authors.initials: C
authors.lastname: Kitchen
authors.forename: F E
authors.initials: FE
authors.lastname: Shafer
authors.forename: P C
authors.initials: PC
authors.lastname: Shah
authors.forename: D
authors.initials: D
authors.lastname: Strickland
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D000906:Antibodies / Q000097:blood; D002648:Child; D002675:Child, Preschool; D005121:Extremities / Q000503:physiopathology; D005164:Factor IX / Q000276:immunology; D005167:Factor VII / Q000008:administration & dosage*; D005169:Factor VIII / Q000276:immunology; D015942:Factor VIIa; D005260:Female; D006467:Hemophilia A / Q000097:blood / Q000188:drug therapy* / Q000276:immunology / Q000503:physiopathology; D002836:Hemophilia B / Q000097:blood / Q000188:drug therapy* / Q000276:immunology / Q000503:physiopathology; D006470:Hemorrhage / Q000188:drug therapy; D006801:Humans; D007223:Infant; D007275:Injections, Intravenous; D008297:Male; D008875:Middle Aged; D011994:Recombinant Proteins / Q000008:administration & dosage; D016879:Salvage Therapy
chemical_list: D000906:Antibodies; D011994:Recombinant Proteins; D005167:Factor VII; D005169:Factor VIII; D005164:Factor IX; C103587:recombinant FVIIa; D015942:Factor VIIa