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.

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Introduction:Monitoring clinical outcome in persons with haemophilia (PWH) is essential in order to provide optimal treatment for individual patients and compare effectiveness of treatment strategies. Experience with measurement of activities and participation in haemophilia is limited and consensus on preferred tools is lacking. Aim:The aim of this study was to give a comprehensive overview of the measurement properties of a selection of commonly used tools developed to assess activities and participation in PWH.Methods: Electronic databases were searched for articles that reported on reliability, validity or responsiveness of predetermined measurement tools (5 self-reported and 4 performance based measurement tools). Methodological quality of the studies was assessed according to the COSMIN checklist. Best evidence synthesis was used to summarize evidence on the measurement properties. Results:The search resulted in 3453 unique hits. Forty-two articles were included.The self-reported Haemophilia Acitivity List (HAL), Pediatric HAL (PedHAL) and the performance based Functional Independence Score in Haemophilia (FISH) were studied most extensively. Methodological quality of the studies was limited. Measurement error, cross-cultural validity and responsiveness have been insufficiently evaluated. Conclusion:Albeit based on limited evidence, the measurement properties of the PedHAL, HAL and FISH are currently considered most satisfactory. Further research needs to focus on measurement error, responsiveness, interpretability and crosscultural validity of the self-reported tools and validity of performance based tools which are able to assess limitations in sports and leisure activities. K E Y W O R D Sactivities, haemophilia, measurement properties, outcome measure, participationThis is an open access article under the terms of the Creative Commons Attribution-NonCommercial-NoDerivs License, which permits use and distribution in any medium, provided the original work is properly cited, the use is non-commercial and no modifications or adaptations are made. 4 According to the ICF "activity" is defined as "the execution of a task or action by an individual" and participation is defined as "involvement in a life situation". Consistent monitoring outcome at activity and participation level still needs to find its way into haemophilia care and research.Various haemophilia specific and generic tools for assessing activity and participation are available, but consensus on a preferred set of measurement tools is not yet reached. Differences in access to expensive clotting factor concentrates cause significant differences in joint status between PWH in different parts of the world. Differences between age groups, severity and cultural differences also contribute to heterogeneity in the haemophilia population. Moreover, measurement tools might be used for different purposes, eg. monitoring clinical outcome vs comparing treatment groups and evaluation of chronic complaints vs acute bleeds. A core set of measurement tools shou...

Trusted evidence. Informed decisions. Better health. Cochrane Database of Systematic Reviews Analysis 6.3. Comparison 6 Local steroid injection versus extracorporeal shock wave therapy (ESWT), Outcome 3 Pain and function: Mayo CSS

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