title: Facilitating the implementation of pharmacokinetic-guided dosing of prophylaxis in haemophilia care by discrete choice experiment.
abstract: BACKGROUND
  Patients', parents' and providers' preferences with regard to medical innovations may have a major impact on their implementation.
  
  
  OBJECTIVE
  To evaluate barriers and facilitators for individualized pharmacokinetic (PK)-guided dosing of prophylaxis in haemophilia patients, parents of young patients, and treating professionals by discrete choice experiment (DCE) questionnaire.
  
  
  METHODS
  The study population consisted of patients with haemophilia currently or previously on prophylactic treatment with factor concentrate (n = 114), parents of patients aged 12-18 years (n = 19) and haemophilia professionals (n = 91). DCE data analysis was performed, taking preference heterogeneity into account.
  
  
  RESULTS
  Overall, patients and parents, and especially professionals were inclined to opt for PK-guided dosing of prophylaxis. In addition, if bleeding was consequently reduced, more frequent infusions were acceptable. However, daily dosing remained an important barrier for all involved. 'Reduction of costs for society' was a facilitator for implementation in all groups.
  
  
  CONCLUSIONS
  To achieve implementation of individualized PK-guided dosing of prophylaxis in haemophilia, reduction of bleeding risk and reduction of costs for society should be actively discussed as they are motivating for implementation; daily dosing is still reported to be a barrier for all groups. The knowledge of these preferences will enlarge support for this innovation, and aid in the drafting of implementable guidelines and information brochures for patients, parents and professionals.
authors.affiliation: Department of Paediatric Haematology, Erasmus University Medical Centre - Sophia Children's Hospital, Rotterdam, The Netherlands.
authors.forename: J
authors.initials: J
authors.lastname: Lock
authors.affiliation: Department of Public Health, Erasmus University Medical Centre, Rotterdam, The Netherlands.
authors.forename: E W
authors.initials: EW
authors.lastname: de Bekker-Grob
authors.affiliation: Department of Paediatric Haematology, Erasmus University Medical Centre - Sophia Children's Hospital, Rotterdam, The Netherlands.
authors.forename: G
authors.initials: G
authors.lastname: Urhan
authors.affiliation: Department of Paediatric Haematology, Academic Medical Centre, Amsterdam, The Netherlands.
authors.forename: M
authors.initials: M
authors.lastname: Peters
authors.affiliation: Department of Haematology, University Medical Centre Groningen, University of Groningen, Groningen, The Netherlands.
authors.forename: K
authors.initials: K
authors.lastname: Meijer
authors.affiliation: Department of Paediatric Haematology, Radboud university medical centre, Nijmegen, The Netherlands.
authors.forename: P
authors.initials: P
authors.lastname: Brons
authors.affiliation: Department of Thrombosis and Haemostasis, Leiden University Medical Centre, Leiden, The Netherlands.
authors.forename: F J M
authors.initials: FJ
authors.lastname: van der Meer
authors.affiliation: Netherlands Haemophilia Patient Society (NVHP), Nijkerk, The Netherlands.
authors.forename: M H E
authors.initials: MH
authors.lastname: Driessens
authors.affiliation: Arthur Bloom Haemophilia Centre, School of Medicine, Cardiff University, Cardiff, UK.
authors.forename: P W
authors.initials: PW
authors.lastname: Collins
authors.affiliation: Department of Paediatric Haematology, Academic Medical Centre, Amsterdam, The Netherlands.
authors.forename: K
authors.initials: K
authors.lastname: Fijnvandraat
authors.affiliation: Department of Haematology, Erasmus University Medical Centre, Rotterdam, The Netherlands.
authors.forename: F W G
authors.initials: FW
authors.lastname: Leebeek
authors.affiliation: Department of Paediatric Haematology, Erasmus University Medical Centre - Sophia Children's Hospital, Rotterdam, The Netherlands.
authors.forename: M H
authors.initials: MH
authors.lastname: Cnossen
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D002755:Choice Behavior; D054796:Drug Dosage Calculations; D005260:Female; D006467:Hemophilia A / Q000517:prevention & control*; D006801:Humans; D008297:Male; D008875:Middle Aged; D015233:Models, Statistical; D011795:Surveys and Questionnaires; D014018:Tissue Distribution; D055815:Young Adult
keywords: discrete choice experiment; haemophilia; implementation; pharmacokinetic-guided dosing; preferences; prophylaxis