Outcome measures monitoring physical function in children with haemophilia: a systematic review

Abstract

Our objective was to provide a synthesis of measurement properties for performance-based outcome measures used to evaluate physical function in children with haemophilia. A systematic review of articles published in English using Medline, PEDro, Cinahl and The Cochrane Library electronic databases was conducted. Studies were included if a performance-based method, clinical evaluation or measurement tool was used to record an aspect of physical function in patients with haemophilia aged ≤ 18 years. Recording of self-perceived or patient-reported physical performance, abstracts, unpublished reports, case series reports and studies where the outcome measure was not documented or cross-referenced was excluded. Description of outcome measures, patient characteristics, measurement properties for construct validity, internal consistency, repeatability, responsiveness and feasibility was extracted. Data synthesis of 41 studies evaluating 14 measures is reported. None of the outcome measures demonstrated the requirements for all the measurement properties. Data on validity and test-retest repeatability were most lacking together with studies of sufficient size. Measurement of walking and muscle strength demonstrated good repeatability and discriminative properties; however, correlation with other measures of musculoskeletal impairment requires investigation. The Haemophilia Joint Health Score demonstrated acceptable construct validity, internal consistency and repeatability, but the ability to discriminate changes in physical function is still to be determined. Rigorous evaluation of the measurement properties of performance-based outcome measures used to monitor physical function of children with haemophilia in larger collaborative studies is required.

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

“…9 Perspectives of PWH reported recommend simple, rapid measures are used routinely, and more detailed measures used in defined periods such as annual reviews. Participants also expressed con- common concern was however, raised regarding the time and effort required to complete measures.…”

Muscle function deterioration in patients with haemophilia: Prospective experience from Costa Rica

“…Haemophilia Joint Health Score includes evaluation of muscle trophism and strength for ankles and knees; nevertheless, hip joint and nearby muscles as gluteus are not included in this evaluation . HJHS has been validated and used to evaluate joint health status in patients with haemophilia of different ages across several studies showing good construct validity, internal consistency and test‐retest reliability . However, its ability in detecting significant differences between patients with different disease severity and, in particular, in discriminating early functional changes prior to structural damage onset is still to be determined …”

Recognizing the need for personalization of haemophilia patient‐reported outcomes in the prophylaxis era

“…However, neither the HJHS nor the WFH score correlate well with bleeding rates, and neither instrument is sensitive enough to discriminate change in patients at the extremes of the arthropathy spectrum (i.e. with late-stage or early-stage joint damage) [19]. More importantly, neither instrument measures functional status in daily life (e.g.…”

“…This limitation stems from the fact that improvements in ABR are comparable across clinical studies evaluating prophylaxis, with all studies showing low mean/median frequencies of bleeding [2,14,[17][18][19]. Thus, using ABR as the sole endpoint to discriminate among prophylactic approaches would require an impractically large patient enrolment to provide statistically meaningful power to discriminate efficacy.…”