Choosing outcome assessment tools in haemophilia care and research: a multidisciplinary perspective. Issue 1 (15th September 2016)

• Record Type: Journal Article
• Authors: Fischer, K.; Poonnoose, P.; Dunn, A. L.; Babyn, P.; Manco‐Johnson, M. J.; David, J. A.; van der Net, J.; Feldman, B.; Berger, K.; Carcao, M.; de Kleijn, P.; Silva, M.; Hilliard, P.; Doria, A.; Srivastava, A.; Blanchette, V.
• Other Names: Avila Laura investigator.; Boma‐Fischer Laurence investigator.; Brandao Leonardo investigator.; Busch Michael T. investigator.; Castro Denise investigator.; Chiu Aubrey investigator.; Funk Sharon investigator.; Luke Brian investigator.; Manco‐Johnson Michael investigator.; Mohanta Arun investigator.; Monahan Paul investigator.; St‐Louis Jean investigator.; Shupak Rachel investigator.; Stimec Jennifer investigator.; Teitel Jerry investigator.; Zourikian Nichan investigator.; Bonanad Santiago investigator.; Gouw Samantha investigator.; Querol Felipe investigator.; Santagostino Elena investigator.; Solimeno Pier Luigi investigator.; von Mackensen Sylvia investigator.; Wells Anna investigator.; Carneiro Jorges D.A. investigator.; Gibokte Sirdhar investigator.; Keshava Shyamkumar Nidugala investigator.; Matta Marcia investigator.; Natesirinilkul Rungrote investigator.; Ozelo Margareth investigator.; Poonoose Pradeep investigator.; Sakamoto Flavia investigator.; Sun Jing investigator.; Uchoa Marcia investigator.; Wu Runhui investigator.;
• Is Part Of: Haemophilia. Volume 23:Issue 1(2017)
• Page Start: 11
• Page End: 24
• Publication Date: 2016-09-15
• Subjects: activity -- arthropathy -- evaluation instruments -- haemophilia -- outcomes research -- participation; Hemophilia -- Periodicals; 616.1572005
• DOI: 10.1111/hae.13088
• Languages: English
• ISSNs: 1351-8216

Abstract

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.