title: Assessments of outcome in haemophilia - what is the added value of QoL tools?
abstract: BACKGROUND
  Access to treatment and especially to long-term regular replacement treatment with clotting factor concentrates (prophylaxis) have caused dramatic contrasts in the clinical picture between haemophilia populations. An individual patient with severe haemophilia age 20 years can have normal joints or can be severely crippled and unable to work. Assessment of outcome in a standardized way has therefore become essential.
  
  
  OBJECTIVE
  Discuss the relevance and utility of the different outcome assessment tools in patient groups with different access to treatment.
  
  
  METHODS
  In the last decade new outcome assessment tools specific for haemophilia have been developed that measure all aspects of health according to the International Classification of Functioning, Disability and Health (ICF) model. These tools are directed at assessing the clinical and radiological status of joints as well as overall functioning, such as participation and psychosocial aspects, evaluating overall health-related quality of life (HRQOL). For deciding which tools to use in clinical practice or research, one needs to consider the specific context with regard to disease burden, healthcare environment and socioeconomic background of the patients being evaluated.
  
  
  CONCLUSIONS
  Prospective systematic assessment of outcome in haemophilia and related bleeding disorders is important. Based upon recent literature a critical appraisal of outcome tools is described.
authors.affiliation: Julius Center for Health Sciences and Primary Care, University Medical Center Utrecht, Utrecht, The Netherlands.
authors.forename: H M
authors.initials: HM
authors.lastname: van den Berg
authors.affiliation: Division of Rheumatology, The Hospital for Sick Children, Toronto, ON, Canada.|Departments of Paediatrics, Medicine, and the Institute of Health Policy, Management and Evaluation, University of Toronto, Toronto, ON, Canada.
authors.forename: B M
authors.initials: BM
authors.lastname: Feldman
authors.affiliation: Julius Center for Health Sciences and Primary Care, University Medical Center Utrecht, Utrecht, The Netherlands.|Van Creveldkliniek, University Medical Center Utrecht, Utrecht, The Netherlands.
authors.forename: K
authors.initials: K
authors.lastname: Fischer
authors.affiliation: Division of Haematology/Oncology, The Hospital for Sick Children, Toronto, ON, Canada.|Department of Paediatrics, University of Toronto, Toronto, ON, Canada.
authors.forename: V
authors.initials: V
authors.lastname: Blanchette
authors.affiliation: Department of Orthopaedics, Christian Medical College, Vellore, India.
authors.forename: P
authors.initials: P
authors.lastname: Poonnoose
authors.affiliation: Department of Haematology, Christian Medical College, Vellore, India.
authors.forename: A
authors.initials: A
authors.lastname: Srivastava
mesh_terms: D005164:Factor IX / Q000627:therapeutic use; D005169:Factor VIII / Q000627:therapeutic use; D006467:Hemophilia A / Q000188:drug therapy / Q000473:pathology*; D006801:Humans; D007596:Joints / Q000503:physiopathology; D009140:Musculoskeletal Diseases / Q000000981:diagnostic imaging / Q000473:pathology / Q000628:therapy; D017063:Outcome Assessment, Health Care; D011788:Quality of Life; D011859:Radiography
chemical_list: D005169:Factor VIII; D005164:Factor IX
keywords: haemophilia; haemophilia health joint score; health-related quality of life; outcome assessment