Choosing outcome assessment tools in haemophilia care and research: a multidisciplinary perspective

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.

The Paediatric Haemophilia Activities List (pedHAL) in routine assessment: changes over time, child‐parent agreement and informative domains

“…2 It was directly derived from the Haemophilia Activities List for adults 3,4 and subsequently validated. 6 Regular outcome assessment should be part of regular care. The pedHAL is recommended for both research purposes and clinical management of individual patients.…”

Monitoring joint health in haemophilia: Factors associated with deterioration

“…However, early joint alterations remain undetected on the Pettersson score as X‐ray only shows osteochondral changes . Furthermore, in a paediatric population, the WFH physical examination score is less sensitive than the Haemophilia Joint Health Score (HJHS), which was developed more recently and is the recommended tool for routine patient follow‐up assessments of joint health . The WFH guidelines recommend annual use of the HJHS during regular evaluations, although assessment must be performed by a trained physiotherapist and is time consuming.…”

Physiotherapy outcome measures of haemophilia acute bleed episodes: What matters to patients?

“…10 Limited research has focussed on OM for recovery from acute bleeds or more generally for haemophilia. 10 Limited research has focussed on OM for recovery from acute bleeds or more generally for haemophilia.…”