title: Measuring patient-reported outcomes in haemophilia clinical research.
abstract: Patient-reported outcome (PRO) measures have been used to assess quality of life and health state preferences from the patient's perspective. However, they have not been fully utilized in haemophilia clinical practice and research. A series of meetings were convened to review and document the state of the art in PROs relevant to haemophilia. Experts developed a process for selection of measures and identified published measures of health-related quality of life (HRQoL) relevant to patients with haemophilia. These were synthesized and reviewed. Patient preference measures were also identified and reviewed. Although the majority of measures were developed for and validated in adults, several measures were identified for use in paediatric populations. This paper recommends an approach to the selection of PROs for application in haemophilia clinical research and practice and identifies several potential measures relevant for application in haemophilia clinical research and practice.
authors.affiliation: Amgen Inc, One Amgen Center Drive, Thousand Oaks, CA 91320, USA. dglobe@amgen.com
authors.forename: D
authors.initials: D
authors.lastname: Globe
authors.forename: N L
authors.initials: NL
authors.lastname: Young
authors.forename: S
authors.initials: S
authors.lastname: Von Mackensen
authors.forename: M
authors.initials: M
authors.lastname: Bullinger
authors.forename: J
authors.initials: J
authors.lastname: Wasserman
mesh_terms: D035843:Biomedical Research / Q000193:education; D006395:Hemarthrosis / Q000453:epidemiology* / Q000517:prevention & control / Q000523:psychology; D006467:Hemophilia A / Q000453:epidemiology* / Q000523:psychology / Q000628:therapy; D006801:Humans; D017063:Outcome Assessment, Health Care; D017060:Patient Satisfaction / Q000706:statistics & numerical data*; D011788:Quality of Life / Q000523:psychology