Measurement of joint health in persons with haemophilia: A systematic review of the measurement properties of haemophilia‐specific instruments

Abstract

Introduction Accurate assessment of joint health in persons with haemophilia is crucial. Several haemophilia‐specific measurement tools are available, but an overview of the measurement properties is lacking. Aim To provide an overview of the measurement properties of haemophilia‐specific measurement tools to assess clinical joint health. Methods MEDLINE and EMBASE were searched for reports on reliability, validity or responsiveness of the World Federation of Haemophilia Orthopedic Joint Score (WFH), Colorado Physical Examination Score (CPE), joint examination score by Petrini (PJS) and Hemophilia Joint Health Score (HJHS). Methodological quality of the studies was assessed using an adapted COSMIN checklist. Results The search yielded 2905 unique hits, and 98 papers were included. The methodological quality of the included studies was limited. The HJHS was studied most extensively, which yielded limited evidence for good internal consistency and structural validity, moderate evidence for hypothesis testing in adults and conflicting evidence for hypothesis testing in children. Reliability, measurement error and responsiveness were rated unknown due to low COSMIN scores. For the CPE and PJS, we found limited to moderate evidence for good responsiveness and conflicting evidence for hypothesis testing. Conclusion Only patchy evidence is available on the quality of measurement properties of all haemophilia‐specific joint health scores. Although significant gaps in the evidence for all instruments remain, measurement properties of the HJHS were most extensively studied and show no drawbacks for use in clinical practice. This review forms the basis for further research aimed at the assessment of measurement properties of measurement tools to assess joint health.

Clinical outcomes in hemophilia: Towards development of a core set of standardized outcome measures for research

“…In those cases where a specific tool was considered to be mature and well‐validated, it was chosen or suggested (eg, EQ‐5D and HJHS). However, in some cases, this did not occur, despite the evidence presented from the systematic reviews 4,9,10 …”

“…As a follow‐up to the 2014 Outcome Measures meeting, 1 a 5 step, modified nominal groups process 19 was followed to promote the definition of a core set, guided by the findings of the results of that meeting, and especially the systematic reviews of tools for joint health scores, 4 activity/participation, 10 health related quality of life, 9 and radiology 20 …”

“…First, the results of the two pre‐meeting surveys (Steps 1 and 2) were presented as background. Second, key findings from the systematic reviews 4,9,10 were presented, along with other summary presentations from experts split into each major WHO‐ICF domain. In Step 3, the participants voted during the 2 day consensus meeting for those items they felt should be included as part of a core set for each ICF domain separately.…”

“…A problem for hemophilia researchers and healthcare professionals is redundancy among existing outcome measures. For example, within the body structures and function domain for joint examinations, there are at least 4 different tools that have been developed and extensively studied to measure the same construct, all with varying quality 4 . These include the World Federation of Hemophilia Score: Physical Joint Examination (the Gilbert Score), 5 the Colorado Physical Examination scales, 6 the Paediatric Orthopedic Joint Score, 7 and the Hemophilia Joint Health Score, v2.1 8 …”

“…In recent years, the advent of new therapies, and an emphasis on outcome measurement in clinical research and care mandated by regulatory bodies (eg the FDA, EAMA) and funding agencies, has resulted in unprecedented activity in outcome measures research specific to hemophilia. Following a meeting of interested stakeholders, held in 2014, which focused on the identification of outcome measures in hemophilia, 1 a series of systematic reviews were completed 4,9,10 . These reviews provided a critical overview of the outcome measures that have been developed for use in persons with hemophilia and point to gaps in information regarding the various outcome measures.…”