title: Treatment of acute bleeding in acquired haemophilia A with recombinant activated factor VII: analysis of 10-year Japanese postmarketing surveillance data.
abstract: BACKGROUND
  Patients with acquired haemophilia A (AHA) have autoantibodies against factor VIII (FVIII), and may develop spontaneous bleeding that requires treatment with FVIII inhibitor bypassing agents such as recombinant activated FVII (rFVIIa, NovoSeven®). However, data regarding the use of rFVIIa are limited.
  
  
  OBJECTIVE
  To investigate the use, efficacy and safety of rFVIIa for the treatment of AHA by analysis of 10-year multicentre Japanese postmarketing surveillance data.
  
  
  METHODS
  Treatment regimens, haemostatic efficacy and adverse events were recorded for rFVIIa therapy of AHA patients with bleeding episodes. Treatment was evaluated as markedly effective, effective, moderate or ineffective.
  
  
  RESULTS
  Data were collected for 371 bleeding episodes in 132 patients. Bleeding improved after rFVIIa therapy in 92% of episodes (markedly effective in 41%, effective in 10%, moderate in 41%). The response rate was significantly better in patients who received an initial dose of ≥90 μg kg-1than in those who received an initial dose of <90 μg kg-1. The response rate was also significantly better when rFVIIa was administered earlier after the onset of bleeding. Twelve serious adverse events were recorded in six patients, including five serious thromboembolic events in three patients who were all elderly with significant comorbidities.
  
  
  CONCLUSIONS
  This is the largest, single-country study of rFVIIa therapy in AHA patients reported to date. The Japanese surveillance data show comparable efficacy and safety to prior multinational studies. Doses of 90-120 μg kg-1and prompt initiation of treatment may be important to achieve good bleeding control.
authors.affiliation: Tokyo Medical University, Tokyo, Japan.
authors.forename: K
authors.initials: K
authors.lastname: Amano
authors.affiliation: Tokyo Medical University, Tokyo, Japan.
authors.forename: I
authors.initials: I
authors.lastname: Seita
authors.affiliation: Hyogo College of Medicine, Nishinomiya, Japan.
authors.forename: S
authors.initials: S
authors.lastname: Higasa
authors.affiliation: Hyogo College of Medicine, Nishinomiya, Japan.
authors.forename: A
authors.initials: A
authors.lastname: Sawada
authors.affiliation: Novo Nordisk Pharma Ltd., Tokyo, Japan.
authors.forename: M
authors.initials: M
authors.lastname: Kuwahara
authors.affiliation: Nara Medical University, Kashihara, Japan.
authors.forename: M
authors.initials: M
authors.lastname: Shima
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D002648:Child; D002675:Child, Preschool; D015331:Cohort Studies; D062665:Epidemiological Monitoring; D015942:Factor VIIa / Q000009:adverse effects / Q000627:therapeutic use*; D005260:Female; D006467:Hemophilia A / Q000150:complications* / Q000188:drug therapy; D006470:Hemorrhage / Q000188:drug therapy*; D006801:Humans; D007564:Japan; D008297:Male; D008875:Middle Aged; D011994:Recombinant Proteins / Q000009:adverse effects / Q000627:therapeutic use; D055815:Young Adult
chemical_list: D011994:Recombinant Proteins; C103587:recombinant FVIIa; D015942:Factor VIIa
keywords: acquired; haemophilia A; haemorrhage; recombinant activated factor VII; safety; treatment efficacy