Objective
Repeated hemarthrosis in hemophilia causes arthropathy with pain and dysfunction. The Hemophilia Joint Health Score (HJHS) was developed to be more sensitive for detecting arthropathy than ...the World Federation of Hemophilia (WFH) physical examination scale, especially for children and those using factor prophylaxis. The HJHS has been shown to be highly reliable. We compared its validity and sensitivity to the WFH scale.
Methods
We studied 226 boys with mild, moderate, and severe hemophilia at 5 centers. The HJHS was scored by trained physiotherapists. Study physicians at each site blindly determined individual and total joint scores using a series of visual analog scales.
Results
The mean age was 10.8 years. Sixty‐eight percent were severe (93% of whom were treated with prophylaxis), 15% were moderate (24% treated with prophylaxis), and 17% were mild (3% treated with prophylaxis). The HJHS correlated moderately with the physician total joint score (rs = 0.42, P < 0.0001) and with overall arthropathy impact (rs = 0.42, P < 0.0001). The HJHS was 97% more efficient than the WFH at differentiating severe from mild and moderate hemophilia. The HJHS was 74% more efficient than the WFH at differentiating subjects treated with prophylaxis from those treated on demand. We identified items on the HJHS that may be redundant or rarely endorsed and could be removed from future versions.
Conclusion
Both the HJHS and WFH showed evidence of strong construct validity. The HJHS is somewhat more sensitive for mild arthropathy; its use should be considered for studies of children receiving prophylaxis.
The Hemophilia Joint Health Score (HJHS) was developed and validated to detect arthropathy in children. Additional evidence is required to show validity in adults. We studied the convergent and ...discriminant construct validity of the HJHS version 2.1(HJHSv2.1) in adults with hemophilia. A secondary aim was to define age‐related normative adult HJHSv2.1 reference values.
We studied 192 adults with hemophilia, and 120 healthy adults in four age‐matched groups—18 to 29, 30 to 40, 41 to 50, and >50 years—at nine centers. Trained physiotherapists scored the HJHS and World Federation of Hemophilia (WFH) joint score. Health history, the Functional Independence Scale of Hemophilia (FISH), Hemophilia Activities List (HAL), and Short‐Form McGill Pain Questionnaire (SF‐MPQ) were also collected.
The median age was 35.0 years. Of participants with hemophilia, 68% had severe, 14% moderate, and 18% mild disease. The HJHS correlated strongly with WFH score (Spearman’s rho rs = .95, P < .001). Moderate correlations were seen between the FISH (rs = .50, P < .001) and SF‐MPQ Present Pain Intensity (rs = .50, P < .001), while a modest correlation was found with the HAL (rs = −.37, P < .001). The HJHS significantly differentiated between age groups (Kruskal‐Wallis T = 35.02, P < .001) and disease severity in participants with hemophilia. The HJHS had high internal reliability (Cronbach’s α = .88). We identified duration of swelling as a redundant item in the HJHS.
The HJHS shows evidence of strong convergent and discriminant construct validity to detect arthropathy in adults with hemophilia and is well suited for use in this population.
For persons with hemophilia, optimization of joint outcomes is an important unmet need. The aim of this initiative was to determine use of ultrasound in evaluating arthropathy in persons with ...hemophilia, and to move toward consensus among hemophilia care providers regarding the preferred ultrasound protocols for global adaptation.
A global survey of hemophilia treatment centers was conducted that focused on understanding how and why ultrasound was being used and endeavored to move toward consensus definitions of both point‐of‐care musculoskeletal ultrasound (POC‐MSKUS) and full diagnostic ultrasound, terminology to describe structures being assessed by ultrasound, and how these assessments should be interpreted. Next, an in‐person meeting of an international group of hemophilia health care professionals and patient representatives was held, with the objective of achieving consensus regarding the acquisition and interpretation of POC‐MSKUS and full diagnostic ultrasound for use in the assessment of musculoskeletal (MSK) pathologies in persons with hemophilia.
The recommendations were that clear definitions of the types of ultrasound examinations should be adopted and that a standardized ultrasound scoring/measurement system should be developed, tested, and implemented. The scoring/measurement system should be tiered to allow for a range of complexity yet maintain the ability for comparison across levels.
Ultrasound is an evolving technology increasingly used for the assessment of MSK outcomes in persons with hemophilia. As adoption increases globally for clinical care and research, it will become increasingly important to establish clear guidelines for image acquisition, interpretation, and reporting to ensure accuracy, consistency, and comparability across groups.
Background
Intra-articular bleeds in patients with inherited bleeding disorders lead to active synovitis which may progress to a chronic state over time. We explored the diagnostic value of color ...Doppler ultrasound in detecting synovitis in boys with bleeding disorders.
Results
Sixty boys with hemophilia and 3 boys with type 3 von Willebrand disease aged 5 to 18 years (median 12.3 years) were imaged by gray-scale and color Doppler ultrasound (US) in three centers (Beijing, China
n
= 22, Guangzhou, China
n
= 12 and Toronto, Canada
n
= 29)) in this observational study. Images were independently reviewed by two radiologists blinded to clinical data using a subjective semi-quantitative scoring system and objective measurements of synovial thickness and vascularity. Inter-reader reliability for using subjective versus objective color Doppler US methods for assessing synovial vascularity was excellent for the subjective method and moderate/lower range of substantial for the objective method. Agreement between degree of vascularity on color Doppler and extent of synovial hypertrophy on gray-scale US was overall poor for Canada data and moderate for China data. Correlations between degree of vascularity on color Doppler and synovial hypertrophy on gray-scale US, and clinical constructs (total and itemized HJHS scores and total Pettersson X-ray scores) for assessment of blood-induced arthropathy were all poor.
Conclusion
Color Doppler US is a valuable scoring method for evaluating reactive synovitis in joints of subjects with inherited bleeding disorders and holds potential for assessing post-bleed reactive synovitis once further information on its association with timing of the joint bleed becomes available in the literature.
Patients with hemophilia may experience joint damage, which can impair participation, yet few studies have examined the impact hemophilia may have on social participation and quality of life.
The ...aims of this study are to assess the relationship between patient social participation and self‐perception, social support, and impact on the family.
A random representative sample of 50 boys with hemophilia from The Hospital for Sick Children, Toronto, Canada, completed measures of social participation (Participation Scale for kids), self‐perception (Self‐Perception Profile for children and adolescents), and social support (Social Support Scale for children). Participants' parents completed Family Impact Module of the Pediatric Quality of Life Inventory. Data were analyzed using Pearson product‐moment correlations.
Social participation was strongly correlated with self‐perception subscales Social Acceptance (r= −0.5, p= <0.001) and Global Self‐Worth (r= −0.6, p= <0.001) for all participants. The Athletic Competence subscale was strongly correlated for adolescents only (r= −0.6, p= <0.01). There were strong correlations between social participation and social support from parents (r= −0.6, p= <0.001), teachers (r= −0.5, p= <0.001), and classmates (r= −0.6, p= <0.001) and moderate correlations for support from close friends (r= −0.4, p= <0.01). There were no significant correlations with family impact.
In the context of a country with unlimited access to safe clotting factor concentrates, boys with hemophilia have few social participation restrictions. Although correlational findings do not represent causality, they suggest that encouragement of social participation may be beneficial in boys with hemophilia to increase self‐perception as well as strengthen their social support network.
In countries with restricted access to clotting factor concentrates, early implementation of low‐dose prophylaxis is recommended over episodic treatment.
The objective of this 1‐year prospective ...secondary prophylaxis study was to evaluate the efficacy of a dose/frequency escalating protocol in young boys with hemophilia A in China.
Boys were started on a low‐dose protocol (minimum 10–15 IU/kg of factor VIII FVIII twice weekly). Escalation was based on index joint bleeding, swelling/persistent joint swelling, and serial ultrasound (gray scale and color Doppler) examinations of index joints.
Thirty‐three boys, median age 4.8 years (interquartile range, 3.8‐6.1) were enrolled in a 3‐month observation period that preceded a 1‐year prophylaxis phase. A significant reduction in total bleeding events (43.0%, P= .001), index joint bleeds (53.2%, P= .002), and target index joint bleeds (70.0%, P= 0.02) was observed during the prophylaxis phase. During the prophylaxis period, 40% of target joints resolved. The percentage of boys with zero index joint bleeds increased significantly (P= .004) from 51.5% during the observation phase to 81.8% in last quarter of the prophylaxis phase (months 10‐12). There was no progression of arthropathy based on physical examination (Hemophilia Joint Health Score), X‐ray, and ultrasound obtained at entry into the prophylaxis phase and at study exit. The median FVIII consumption over the prophylaxis phase was 1786 IU/kg/y.
A low‐dose, individualized prophylaxis protocol, guided by individual bleeding profiles and serial assessment of joint status, enables escalation of treatment intensity in boys with severe hemophilia A, leading to a significant reduction in bleeding events and reduction in target joint bleeding.
OBJECTIVE. The purpose of this article is to assess the reliability of interpretation of ultrasound findings according to data blinding in maturing hemophilic joints and to determine the diagnostic ...accuracy of ultrasound compared with MRI for assessing joint components. SUBJECTS AND METHODS. Ankles (n = 34) or knees (n = 25) of boys with hemophilia or von Willebrand disease (median age, 13 years; range, 5-17 years) were imaged by ultrasound, MRI, and radiography in two centers (Toronto, Canada, and Vellore, India). Ultrasound scans were performed by two operators (one blinded and one unblinded to MRI data) and were reviewed by four reviewers who were unblinded to corresponding MRI findings according to a proposed 0- to 14-item scale that matches 14 of 17 items of the corresponding MRI scale. MRI examinations were independently reviewed by two readers. RESULTS. When data were acquired by radiologists, ultrasound was highly reliable for assessing soft-tissue changes (intraclass correlation coefficient ICC, 0.98 for ankles and 0.97 for knees) and substantially to highly reliable for assessing osteochondral changes (ICC, 0.61 for ankles and 0.89 for knees). Ultrasound was highly sensitive (> 92%) for assessing synovial hypertrophy and hemosiderin in both ankles and knees but had borderline sensitivity for detecting small amounts of fluid in ankles (70%) in contrast to knees (93%) and variable sensitivity for evaluating osteochondral abnormalities (sensitivity range, 86-100% for ankles and 12-100% for knees). CONCLUSION. If it is performed by experienced radiologists using a standardized protocol, ultrasound is highly reliable for assessing soft-tissue abnormalities of ankles and knees and substantially to highly reliable for assessing osteochondral changes in these joints.
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.
Introduction
In many countries, there is a shift from standard half‐life (SHL) to extended half‐life (EHL) clotting factor concentrates (CFCs).
Aim
To describe the experience of switching from SHL to ...an EHL FVIII CFC and the impact of this on frequency of infusions, factor consumption, bleeding rates and HRQoL using the Canadian Hemophilia Kids’ Life Assessment Tool (CHO‐KLAT).
Methods
A retrospective chart review was conducted at a single haemophilia treatment centre in 2018 that included boys (ages: 4‐18 years) with moderate/severe haemophilia A, without inhibitors, who switched from a SHL to an EHL FVIII CFC in the previous 2 years and for whom HRQoL data were available.
Results
The study cohort comprised 38 boys mean (SD) age: 11.0 (3.4) years with moderate (n = 5)/severe (n = 33) haemophilia A. The switch was associated with a 33% reduction in the number of weekly infusions from a median of 3.5 to 2.3 (P < .0001) and a 17% reduction in median FVIII consumption from 103 IU/kg/wk to 85.5 IU/kg/wk (P = .004). There was no significant change in annualized joint bleed rates or in CHO‐KLAT scores.
Conclusions
Despite documenting several benefits of switching to EHL FVIII (less infusions, lower factor consumption with no increase in bleeding), our study did not demonstrate any improvement in HRQoL. We conclude that either the current CHO‐KLAT tool is not optimized to measure burden of treatment administration in boys with low bleed rates switching from SHL to EHL FVIII CFCs or that a reduction of 1.2 infusions/week does not result in a meaningful change in HRQoL.
Introduction
The paediatric Haemophilia Activities List (pedHAL) was developed to measure activities and participation in children and youth with haemophilia. Results from international studies ...provide an opportunity to determine which items are universally important.
Aim
The aim of this study was to determine which items of the pedHAL are redundant to construct a shorter version of the pedHAL.
Methods
This study is a cross‐sectional multicentre secondary analysis on pooled data of published studies using the pedHAL (7 domains, 53 items, optimum score: 100) in children with haemophilia A/B aged 4–18 years. To identify redundant items, the following aspects were evaluated: floor and ceiling effects, proportions of missing and ‘not applicable’ responses, inter‐item correlations, component loadings in an exploratory factor analysis, internal consistency and item‐total correlations.
Results
Data on 315 patients with haemophilia from 6 studies were evaluated. Median age was 12.2 years) (range 4.0–18.0), 87.3% had severe haemophilia and 80.3% received prophylaxis. Median (IQR) pedHAL sum score was 96.7 (88.0–100). After a stepwise procedure, 31 items were removed, resulting in a pedHALshort of 22 items, representing all original 7 domains. Most remaining items belonged to the domains ‘sitting/kneeling/standing’ and ‘functions of the legs’. The pedHALshort sum score was similar to the original pedHAL sum score, with small differences in 5 domains.
Conclusion
This clinimetric study resulted in >50% reduction of the length of the pedHAL. The 22‐item pedHALshort reduces patient burden and is expected to capture the information on activities and participation. The pedHALshort needs validation in other populations.
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