Abstract
Joint bleeds in persons with haemophilia (PWH) result in haemophilic arthropathy, which has an impact on daily activities and participation in school-, work- and leisure activities. Assessing outcomes is essential to evaluate any treatment at an individual patient level in day-to-day care or at group level to compare treatment strategies.
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The general aim of this thesis was to optimize outcome assessment of joint health, activities and participation in PWH. This thesis focused on measurement properties of the legacy instruments (paediatric) Haemophilia Activities List (pedHAL/HAL), as well as provided recommendations to reduce the time-investment of outcome assessment.
To assess joint structure and function by the Hemophilia Joint Health Score (HJHS), we recommend to monitor all six joints every five years in adult PWH on long-term prophylaxis with low bleeding rates. Single joints with multiple bleeding episodes and/or the presence of synovitis should be assessed more frequently. Furthermore, 2/9 HJHS items were identified as candidates for item reduction. In addition, use of the HJHSshort will lead to a small reduction in time. A screening examination of joints are also expected to reduce assessment time.
For the pedHAL, both child and parent proxy questionnaires should be reported. PedHAL assessment is recommended with an interval of ≥1 year. However, annual pedHAL assessment has limited clinical value in patients without limitations in activities and participation and without joint and/or muscle bleeds and should be assessed less frequently in these patients. We suggest an interval of three years based on stable high scores in children on prophylaxis with low bleeding rates. In addition, use of the pedHALshort with 22 items and HALshort with 18 items will lower burden for the patient too. The HAL is considered to be a reliable self-reported outcome measure for limitations in activities and participation. Score changes of the HAL sum score greater than the SDC 10.2 indicate that the change was not a result of measurement error.
Finally, we explored the advantages of Patient Reported Outcomes Measurement Information System (PROMIS®), which was recently introduced in the haemophilia field. PROMIS measures are available as Computer Adaptive Tests (CATs). In CATs the selection of the next item depends on the response on the earlier questions resulting in a selection of relevant questions. For PWH the PROMIS CATs ‘physical function’, ‘pain interference’, ‘satisfaction with social roles and activities’ and ‘fatigue’ are feasible, valid and attractive alternatives to legacy instruments. Especially the PROMIS CAT ‘physical function’ is more feasible than the legacy instruments and is relevant to PWH when assessing disabilities at group level.
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