title: Low-factor consumption for major surgery in haemophilia B with long-acting recombinant glycoPEGylated factor IX.
abstract: BACKGROUND
  Surgery in patients with haemophilia B carries a high risk of excessive bleeding and requires adequate haemostatic control until wound healing. Nonacog beta pegol, a long-acting recombinant glycoPEGylated factor IX (FIX), was used in the perioperative management of patients undergoing major surgery.
  
  
  OBJECTIVE
  To evaluate the efficacy and safety of nonacog beta pegol in patients with haemophilia B who undergo major surgery.
  
  
  METHODS
  This was an open-label, multicentre, non-controlled surgery trial aimed at assessing peri- and postoperative efficacy and safety of nonacog beta pegol in 13 previously treated patients with haemophilia B. All patients received a preoperative nonacog beta pegol bolus injection of 80 IU kg-1. Postoperatively, the patients received fixed nonacog beta pegol doses of 40 IU kg-1, repeated at the investigator's discretion. Safety assessments included monitoring of immunogenicity and adverse events.
  
  
  RESULTS
  Intraoperative haemostatic effect was rated 'excellent' or 'good' in all 13 cases. Apart from the preoperative injection, none of the patients needed additional doses of nonacog beta pegol on the day of surgery. The median number of postoperative doses of nonacog beta pegol was 2.0 from days 1 to 6 and 1.5 from days 7 to 13. No unexpected intra- or postoperative complications were observed including deaths or thromboembolic events. No patients developed inhibitors.
  
  
  CONCLUSIONS
  These results indicated that nonacog beta pegol was safe and effective in the perioperative setting, allowing major surgical interventions in patients with haemophilia B with minimal peri- and postoperative concentrate consumption and infrequent injections as reported with standard FIX products.
authors.affiliation: University of Texas Health Science Center and the Gulf States Hemophilia and Thrombophilia Center, Houston, TX, USA.
authors.forename: M A
authors.initials: MA
authors.lastname: Escobar
authors.affiliation: Medical and Science, Haemophilia R&D Portfolio, Novo Nordisk A/S, Søborg, Denmark.
authors.forename: R
authors.initials: R
authors.lastname: Tehranchi
authors.affiliation: National Blood Centre, Kuala Lumpur, Malaysia.
authors.forename: F A
authors.initials: FA
authors.lastname: Karim
authors.affiliation: Department of Pediatric Hematology, NEU Meram Faculty of Medicine, Konya, Turkey.
authors.forename: U
authors.initials: U
authors.lastname: Caliskan
authors.affiliation: Katharine Dormandy Haemophilia Centre and Thrombosis Unit, Royal Free Hospital, London, UK.
authors.forename: P
authors.initials: P
authors.lastname: Chowdary
authors.affiliation: Medical and Science, Haemophilia R&D Portfolio, Novo Nordisk A/S, Søborg, Denmark.
authors.forename: T
authors.initials: T
authors.lastname: Colberg
authors.affiliation: Oxford Haemophilia Centre and Thrombosis Unit, Churchill Hospital, Oxford, UK.
authors.forename: P
authors.initials: P
authors.lastname: Giangrande
authors.affiliation: Division of Hematology Oncology, University of California, San Francisco, CA, USA.
authors.forename: A
authors.initials: A
authors.lastname: Giermasz
authors.affiliation: Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico, Milan, Italy.
authors.forename: M E
authors.initials: ME
authors.lastname: Mancuso
authors.affiliation: Spitalul Clinic de Urgenta pentru Copii Louis Turcanu, IIIrd Paediatric Clinic, Timisoara, Romania.
authors.forename: M
authors.initials: M
authors.lastname: Serban
authors.affiliation: Department of Hematology, National Taiwan University Hospital, Taipei, Taiwan.
authors.forename: W
authors.initials: W
authors.lastname: Tsay
authors.affiliation: Department of Molecular Medicine and Haematology, Faculty of Health Sciences, University of the Witwatersrand, NHLS and Haemophilia Comprehensive Care Centre, Charlotte Maxeke Johannesburg Academic Hospital, Johannesburg, South Africa.
authors.forename: J N
authors.initials: JN
authors.lastname: Mahlangu
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D019468:Disease Management; D005164:Factor IX / Q000627:therapeutic use*; D002836:Hemophilia B / Q000188:drug therapy* / Q000601:surgery*; D006490:Hemostatics / Q000627:therapeutic use*; D006801:Humans; D008297:Male; D008875:Middle Aged; D011994:Recombinant Proteins / Q000627:therapeutic use*; D055815:Young Adult
chemical_list: D006490:Hemostatics; D011994:Recombinant Proteins; D005164:Factor IX
keywords: Phase III; factor IX; haemophilia B; long-acting recombinant factor IX; nonacog beta pegol; surgery