title: Improvement in health-related quality of life in patients with haemophilia B treated with nonacog beta pegol, a new extended half-life recombinant FIX product.
abstract: BACKGROUND
  Health-related quality of life (HRQoL) of individuals with haemophilia has greatly improved with the use of factor replacement and routine prophylaxis.
  
  
  OBJECTIVE
  To explore the HRQoL of individuals with haemophilia B treated with nonacog beta pegol, an extended half-life recombinant factor IX, in a single-blind, randomized multinational phase III pivotal trial (paradigm(™) 2) and its open-label extension (paradigm(™) 4).
  
  
  METHODS
  In the pivotal trial, adolescents and adults with haemophilia B were allocated to 28-week on-demand treatment or randomized to 52 weeks of prophylaxis with 10 or 40 IU kg(-1) nonacog beta pegol administered every seven days. In the extension trial, patients could continue on the same treatment or switch to the alternate dosing regimen at any time. HRQoL was assessed with the HAEMO-QOL/HAEM-A-QOL age-specific questionnaires and the EQ-5D.
  
  
  RESULTS
  In the pivotal trial, adults receiving 40 IU kg(-1) prophylaxis reported significant improvements in the 'HAEM-A-QOL Total' score (-6.4 ± 8.5, P = 0.017) and in 'Sport' (-15.3 ± 8.5, P = 0.020), 'Feeling' (-15.2 ± 18.3, P = 0.010) and 'Partnership' (-9.6 ± 15.5, P = 0.046) domain scores; no significant improvements were seen in the other arms. At the pivotal trial end, fewer patients reported problems in the EQ-5D 'Mobility' and 'Pain/Discomfort' dimensions, in particular those receiving prophylaxis. In the extension trial, adult patients switching from 10 to 40 IU kg(-1) prophylaxis showed significant improvements in 'HAEM-A-QOL Total' score (-12.5 ± 8.7, P = 0.016) and 'Physical health' domain (-23.1 ± 14.4, P = 0.016).
  
  
  CONCLUSIONS
  Prophylactic treatment with nonacog beta pegol 40 IU kg(-1) once weekly leads to HRQoL benefits in individuals with haemophilia B; this might be related to fewer bleeding episodes and higher FIX activity levels.
authors.affiliation: Katharine Dormandy Haemophilia Centre and Thrombosis Unit, Royal Free London NHS Foundation Trust, London, UK.
authors.forename: P
authors.initials: P
authors.lastname: Chowdary
authors.affiliation: CHCMN Hemophilia and Thrombosis Center Children's Hospital and Clinics of Minnesota, Minneapolis, MN, USA.
authors.forename: S
authors.initials: S
authors.lastname: Kearney
authors.affiliation: Patient-Centered Outcomes, Mapi, Lyon, France.
authors.forename: A
authors.initials: A
authors.lastname: Regnault
authors.affiliation: Novo Nordisk A/S, Søborg, Denmark.
authors.forename: C S
authors.initials: CS
authors.lastname: Hoxer
authors.affiliation: Department of Pediatrics, Hematology-Oncology Section, Baylor College of Medicine, Texas Children's Hemophilia & Thrombosis Center, Houston, TX, USA.
authors.forename: D L
authors.initials: DL
authors.lastname: Yee
mesh_terms: D000293:Adolescent; D000328:Adult; D003029:Coagulants / Q000627:therapeutic use*; D004305:Dose-Response Relationship, Drug; D004334:Drug Administration Schedule; D004644:Emotions; D015444:Exercise; D005164:Factor IX / Q000627:therapeutic use*; D006207:Half-Life; D006304:Health Status; D002836:Hemophilia B / Q000188:drug therapy*; D006801:Humans; D008297:Male; D011092:Polyethylene Glycols / Q000627:therapeutic use*; D011788:Quality of Life; D011994:Recombinant Proteins / Q000627:therapeutic use; D016037:Single-Blind Method; D011795:Surveys and Questionnaires; D055815:Young Adult
chemical_list: D003029:Coagulants; D011994:Recombinant Proteins; C000592276:nonacog beta pegol; D011092:Polyethylene Glycols; D005164:Factor IX
keywords: factor IX; haemophilia B; nonacog beta pegol; quality of life; questionnaires