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Updated: Mar 15, 2026
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Choosing outcome assessment tools in haemophilia care and research: a multidisciplinary perspective
K Fischer1, P Poonnoose2, A L Dunn3
1Van Creveldkliniek, University Medical Center Utrecht, Utrecht, The Netherlands.
Insights
Early clotting factor concentrate (CFC) prophylaxis offers near-normal lives for boys with hemophilia. This review identifies optimal outcome measures for assessing musculoskeletal health and treatment benefits in hemophilia care.
Area of Science:
- Medical Research
- Rehabilitation Medicine
- Hematology
Background:
- Early, regular clotting factor concentrate (CFC) prophylaxis improves quality of life for individuals with hemophilia.
- Optimal prophylactic regimens and their long-term benefits require further objective evidence.
- High costs necessitate robust data for treatment optimization and reimbursement.
Purpose of the Study:
- To critically review outcome measures for assessing musculoskeletal health in hemophilia.
- To align outcome measures with the International Classification of Functioning, Disability and Health (ICF) framework.
- To guide the selection of appropriate tools for clinical care and research.
Main Methods:
- Literature review combined with expert consensus from a multidisciplinary conference.
- Evaluation of existing hemophilia outcome tools for reliability, validity, and resource requirements.
- Assessment based on ICF domains, economic factors, and intended use (clinical vs. research).
Main Results:
- Recommendations for selecting outcome measures were developed based on ICF domains and setting.
- The review considered various patient groups and the practicality of tool implementation.
- Expert opinion guided the assessment of tool suitability for hemophilia care.
Conclusions:
- The study provides a framework for choosing outcome measures in hemophilia care.
- Identifying a 'core' set of outcome measures is the next crucial step.
- Standardized outcome assessment is vital for optimizing hemophilia treatment and research.
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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