title: Choosing outcome assessment tools in haemophilia care and research: a multidisciplinary perspective.
abstract: BACKGROUND
  The implementation of early long-term, regular clotting factor concentrate (CFC) replacement therapy ('prophylaxis') has made it possible to offer boys with haemophilia a near normal life. Many different regimens have reported favourable results, but the optimum treatment regimens have not been established and the cost of prophylaxis is very high. Both for optimizing treatment and reimbursement issues, there is a need to provide objective evidence of both short- and long-term results and benefits of prophylactic regimens.
  
  
  OBJECTIVE
  This report presents a critical review of outcome measures for use in the assessment of musculoskeletal health in persons with haemophilia according to the International Classification of Functioning, Disability and Health (ICF). This framework considers structural and functional changes, activities and participation in a context of both personal and environmental factors.
  
  
  METHODS
  Results were generated by a combination of a critical review of available literature plus expert opinion derived from a two day consensus conference between 48 health care experts from different disciplines involved in haemophilia assessment and care. Outcome tools used in haemophilia were reviewed for reliability and validity in different patient groups and for resources required.
  
  
  CONCLUSIONS
  Recommendations for choice of outcome tools were made according to the ICF domains, economic setting, and reason for use (clinical or research). The next step will be to identify a 'core' set of outcome measures for use in clinical care or studies evaluating treatment.
authors.affiliation: Van Creveldkliniek, University Medical Center Utrecht, Utrecht, The Netherlands.
authors.forename: K
authors.initials: K
authors.lastname: Fischer
authors.affiliation: Department of Orthopaedics, Christian Medical College, Vellore, Tamil Nadu, India.
authors.forename: P
authors.initials: P
authors.lastname: Poonnoose
authors.affiliation: Division of Hematology and Oncology, Nationwide Children's Hospital and The Ohio State University, Columbus, OH, USA.
authors.forename: A L
authors.initials: AL
authors.lastname: Dunn
authors.affiliation: Department of Medical Imaging, University of Saskatchewan and Saskatoon Health Region Royal University Hospital, Saskatoon, SK, Canada.
authors.forename: P
authors.initials: P
authors.lastname: Babyn
authors.affiliation: Section of Hematology/Oncology/Bone Marrow Transplantation, Department of Pediatrics, University of Colorado Anschutz Medical Campus and Children's Hospital, Aurora, CO, USA.
authors.forename: M J
authors.initials: MJ
authors.lastname: Manco-Johnson
authors.affiliation: Department of PMR, Christian Medical College, Vellore, Tamil Nadu, India.
authors.forename: J A
authors.initials: JA
authors.lastname: David
authors.affiliation: Child Health Services, Child Development and Exercise Center, University Medical Center and Children's Hospital, Utrecht, The Netherlands.
authors.forename: J
authors.initials: J
authors.lastname: van der Net
authors.affiliation: Division of Rheumatology, Department of Paediatrics and Child Health Evaluative Sciences, Research Institute, Hospital for Sick Children, University of Toronto, Toronto, ON, Canada.
authors.forename: B
authors.initials: B
authors.lastname: Feldman
authors.affiliation: Division of Haematology/Oncology, University Hospital of Munich, Munich, Germany.
authors.forename: K
authors.initials: K
authors.lastname: Berger
authors.affiliation: Division of Haematology/Oncology, Department of Paediatrics and Child Health Evaluative Sciences, Research Institute, Hospital for Sick Children, University of Toronto, Toronto, ON, Canada.
authors.forename: M
authors.initials: M
authors.lastname: Carcao
authors.affiliation: Department of Rehabilitation, Nursing Science and Sports, and Van Creveldkliniek, University Medical Center Utrecht, Utrecht, The Netherlands.
authors.forename: P
authors.initials: P
authors.lastname: de Kleijn
authors.affiliation: Department of Orthopaedic Surgery, Orthopaedic Institute for Children, David Geffen School of Medicine at UCLA, Los Angeles, CA, USA.
authors.forename: M
authors.initials: M
authors.lastname: Silva
authors.affiliation: Department of Rehabilitation, Hospital for Sick Children, University of Toronto, Toronto, ON, Canada.
authors.forename: P
authors.initials: P
authors.lastname: Hilliard
authors.affiliation: Department of Diagnostic Imaging, Research Institute, Hospital for Sick Children, University of Toronto, Toronto, ON, Canada.
authors.forename: A
authors.initials: A
authors.lastname: Doria
authors.affiliation: Department of Haematology, Christian Medical College, Vellore, Tamil Nadu, India.
authors.forename: A
authors.initials: A
authors.lastname: Srivastava
authors.affiliation: Division of Haematology/Oncology, Department of Paediatrics and Child Health Evaluative Sciences, Research Institute, Hospital for Sick Children, University of Toronto, Toronto, ON, Canada.
authors.forename: V
authors.initials: V
authors.lastname: Blanchette
mesh_terms: D006467:Hemophilia A / Q000628:therapy*; D006801:Humans; D017063:Outcome Assessment, Health Care / Q000379:methods*
keywords: activity; arthropathy; evaluation instruments; haemophilia; outcomes research; participation