title: The association between health utility and joint status among people with severe haemophilia A: findings from the KAPPA register.
abstract: BACKGROUND
  People with severe haemophilia A have reportedly impaired health related quality of life (utility) mainly due to recurrent bleeding, arthropathy and treatment burden.
  
  
  OBJECTIVE
  To estimate utilities and evaluate their potential correlates - most importantly the joint status - among people with severe haemophilia A.
  
  
  METHODS
  In this cross-sectional study, eligible participants had severe haemophilia A, were aged ≥15, negative for factor VIII inhibitor and included in the KAPPA register of Denmark, Norway and Sweden. Data on demographics, treatment history, haemophilia joint health score, and EQ-5D utility were obtained from the register. We used box plots to present utilities and joint status and ordinary least squares regression to evaluate correlates of utilities. Participants were consecutively enrolled in the KAPPA register between April 2013 and June 2016.
  
  
  RESULTS
  Overall, 173 participants with median age of 34 (interquartile range: 25-45) were included. Twelve (6.9%) participants were on episodic treatment while 161 (93.1%) were treated using prophylaxis. Concomitant diseases and positive inhibitor history were reported for 73 (43.2%) and 21 (12.1%) participants, respectively. The highest median utility (1.0) was observed among those aged <29 on prophylaxis and those aged 30-44 who had started prophylaxis by age 3. In the multi-variable regression, joint scores of 16-25 (Coef. -0.18, 95% CI: -0.30, -0.06), 26-35 (Coef. -0.21, 95% CI: -0.36, -0.06) and >35 (Coef. -0.37, 95% CI: -0.52, -0.23) were associated with lower utilities.
  
  
  CONCLUSIONS
  Moderate to severe joint manifestations are associated with reduced utilities among persons with severe haemophilia A.
authors.affiliation: Centre for Thrombosis and Haemostasis, Skåne University Hospital, Malmö, Sweden.|Department of Translational Sciences, Faculty of Medicine, Lund University, Malmo, Sweden.
authors.forename: M
authors.identifier: http://orcid.org/0000-0002-9862-3665
authors.initials: M
authors.lastname: Osooli
authors.affiliation: Department of Clinical Sciences, Faculty of Medicine, Lund University, Malmo, Sweden.|Swedish Institute for Health Economics, Lund, Sweden.
authors.forename: K
authors.initials: K
authors.lastname: Steen Carlsson
authors.affiliation: Section of Haematology and Coagulation, Department of Medicine, Coagulation Centre, Sahlgrenska University Hospital, Gothenburg, Sweden.
authors.forename: F
authors.initials: F
authors.lastname: Baghaei
authors.affiliation: Coagulation Unit, Haematology Centre, Karolinska University Hospital, Stockholm, Sweden.|Department of Internal Medicine, Karolinska Institute, Solna, Stockholm, Sweden.
authors.forename: M
authors.initials: M
authors.lastname: Holmström
authors.affiliation: Department of Global Medical Affairs Haematology, Bayer HealthCare Pharmaceuticals, Berlin, Germany.
authors.forename: S
authors.initials: S
authors.lastname: Rauchensteiner
authors.affiliation: Department of Haematology, Oslo University Hospital, Rikshospitalet, Oslo, Norway.|Institute of Clinical Medicine, University of Oslo, Oslo, Norway.
authors.forename: P A
authors.initials: PA
authors.lastname: Holme
authors.affiliation: Center for Haemophilia and Thrombosis, Aarhus University Hospital, Aarhus, Denmark.
authors.forename: L
authors.initials: L
authors.lastname: Hvitfeldt
authors.affiliation: Centre for Thrombosis and Haemostasis, Skåne University Hospital, Malmö, Sweden.|Department of Translational Sciences, Faculty of Medicine, Lund University, Malmo, Sweden.
authors.forename: J
authors.initials: J
authors.lastname: Astermark
authors.affiliation: Centre for Thrombosis and Haemostasis, Skåne University Hospital, Malmö, Sweden.|Department of Translational Sciences, Faculty of Medicine, Lund University, Malmo, Sweden.
authors.forename: E
authors.initials: E
authors.lastname: Berntorp
mesh_terms: D000293:Adolescent; D000328:Adult; D002675:Child, Preschool; D003430:Cross-Sectional Studies; D005260:Female; D006467:Hemophilia A / Q000150:complications* / Q000453:epidemiology / Q000628:therapy; D006801:Humans; D007592:Joint Diseases / Q000150:complications* / Q000517:prevention & control*; D008297:Male; D008875:Middle Aged; D010641:Phenotype; D011788:Quality of Life; D012042:Registries; D012537:Scandinavian and Nordic Countries / Q000453:epidemiology; D055815:Young Adult
keywords: EQ-5D; haemophilia A; haemophilia joint health score; health utility; treatment outcomes