K Fischer1, P Poonnoose2, A L Dunn3, P Babyn4, M J Manco-Johnson5, J A David6, J van der Net7, B Feldman8, K Berger9, M Carcao10, P de Kleijn11, M Silva12, P Hilliard13, A Doria14, A Srivastava15, V Blanchette10. 1. Van Creveldkliniek, University Medical Center Utrecht, Utrecht, The Netherlands. 2. Department of Orthopaedics, Christian Medical College, Vellore, Tamil Nadu, India. 3. Division of Hematology and Oncology, Nationwide Children's Hospital and The Ohio State University, Columbus, OH, USA. 4. Department of Medical Imaging, University of Saskatchewan and Saskatoon Health Region Royal University Hospital, Saskatoon, SK, Canada. 5. Section of Hematology/Oncology/Bone Marrow Transplantation, Department of Pediatrics, University of Colorado Anschutz Medical Campus and Children's Hospital, Aurora, CO, USA. 6. Department of PMR, Christian Medical College, Vellore, Tamil Nadu, India. 7. Child Health Services, Child Development and Exercise Center, University Medical Center and Children's Hospital, Utrecht, The Netherlands. 8. Division of Rheumatology, Department of Paediatrics and Child Health Evaluative Sciences, Research Institute, Hospital for Sick Children, University of Toronto, Toronto, ON, Canada. 9. Division of Haematology/Oncology, University Hospital of Munich, Munich, Germany. 10. Division of Haematology/Oncology, Department of Paediatrics and Child Health Evaluative Sciences, Research Institute, Hospital for Sick Children, University of Toronto, Toronto, ON, Canada. 11. Department of Rehabilitation, Nursing Science and Sports, and Van Creveldkliniek, University Medical Center Utrecht, Utrecht, The Netherlands. 12. Department of Orthopaedic Surgery, Orthopaedic Institute for Children, David Geffen School of Medicine at UCLA, Los Angeles, CA, USA. 13. Department of Rehabilitation, Hospital for Sick Children, University of Toronto, Toronto, ON, Canada. 14. Department of Diagnostic Imaging, Research Institute, Hospital for Sick Children, University of Toronto, Toronto, ON, Canada. 15. Department of Haematology, Christian Medical College, Vellore, Tamil Nadu, India.
Abstract
INTRODUCTION: 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. AIMS: 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. RESULTS AND CONCLUSION: 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.
INTRODUCTION: 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. AIMS: 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. RESULTS AND CONCLUSION: 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: Erna C van Balen; Brian O'Mahony; Marjon H Cnossen; Gerard Dolan; Victor S Blanchette; Kathelijn Fischer; Deborah Gue; Jamie O'Hara; Alfonso Iorio; Shannon Jackson; Barbara A Konkle; Diane J Nugent; Donna Coffin; Mark W Skinner; Cees Smit; Alok Srivastava; Fred van Eenennaam; Johanna G van der Bom; Samantha C Gouw Journal: Res Pract Thromb Haemost Date: 2021-03-06
Authors: Saunya Dover; Victor S Blanchette; Alok Srivastava; Kathelijn Fischer; Audrey Abad; Brian M Feldman Journal: Res Pract Thromb Haemost Date: 2020-04-09
Authors: Isolde A R Kuijlaars; Janjaap van der Net; Brian M Feldman; Magnus Aspdahl; Melanie Bladen; Wypke de Boer; Rubén Cuesta-Barriuso; Ruth E D Matlary; Sharon M Funk; Pamela Hilliard; Judy A John; Christine L Kempton; Piet de Kleijn; Marilyn Manco-Johnson; Pia Petrini; Pradeep Poonnoose; Jean St-Louis; Sylvia Thomas; Merel A Timmer; Sonata Saulyte Trakymiene; Leo van Vlimmeren; Kathelijn Fischer Journal: Haemophilia Date: 2020-10-15 Impact factor: 4.287
Authors: Nihal Bakeer; Saunya Dover; Paul Babyn; Brian M Feldman; Annette von Drygalski; Andrea S Doria; Danial M Ignas; Audrey Abad; Cindy Bailey; Ian Beggs; Eric Y Chang; Amy Dunn; Sharon Funk; Sridhar Gibikote; Nicholas Goddard; Pamela Hilliard; Shyamkumar N Keshava; Rebecca Kruse-Jarres; Yingjia Li; Sébastien Lobet; Marilyn Manco-Johnson; Carlo Martinoli; James S O'Donnell; Olympia Papakonstantinou; Helen Pergantou; Pradeep Poonnoose; Felipe Querol; Alok Srivastava; Bruno Steiner; Karen Strike; Merel Timmer; Pascal N Tyrrell; Logi Vidarsson; Victor S Blanchette Journal: Res Pract Thromb Haemost Date: 2021-07-10