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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 (r_s = 0.42, P < 0.0001) and with overall arthropathy impact (r_s = 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.

Summary. Background: Prophylactic treatment for severe hemophilia A is likely to be more effective than treatment when bleeding occurs, however, prophylaxis is costly. We studied an inception cohort of 25 boys using a tailored prophylaxis approach to see if clotting factor use could be reduced with acceptable outcomes. Methods: Ten Canadian centers enrolled subjects in this 5‐year study. Children were followed every 3 months at a comprehensive care hemophilia clinic. They were initially treated with once‐weekly clotting factor; the frequency was escalated in a stepwise fashion if unacceptable bleeding occurred. Bleeding frequency, target joint development, physiotherapy and radiographic outcomes, as well as resource utilization, were determined prospectively. Results: The median follow‐up time was 4.1 years (total 96.9 person‐years). The median time to escalate to twice‐weekly therapy was 3.42 years (lower 95% confidence limit 2.05 years). Nine subjects developed target joints at a rate of 0.09 per person‐year. There was an average of 1.2 joint bleeds per person‐year. The cohort consumed on average 3656 IU kg⁻¹year⁻¹ of factor (F) VIII. Ten subjects required central venous catheters (three while on study); no complications of these devices were seen. One subject developed a transient FVIII inhibitor. End‐of‐study joint examination scores – both clinically and radiographically – were normal or near‐normal. Conclusions: Most boys with severe hemophilia A will probably have little bleeding and good joint function with tailored prophylaxis, while infusing less FVIII than usually required for traditional prophylaxis.

Objective: Childhood dermatomyositis (DM) is often a chronic disease, lasting many years. It has traditionally been treated with long‐term corticosteroid therapy; side effects are often seen. For more than a decade, methotrexate (MTX) has been safely used for the treatment of juvenile arthritis. Here, we report use of MTX as first‐line therapy for DM, along with aggressively tapered corticosteroids, in an attempt to reduce treatment‐related side effects.Methods: We studied an inception cohort of 31 children with DM who were rigorously followed up in our myositis clinic, and compared them with a control group of 22 patients with incident cases of juvenile DM who received treatment just before we instituted a policy of first‐line therapy with MTX. The mean starting dosage of MTX in the study group was 15 mg/m²/week.Results: Both groups had similar improvement in strength and physical function; however, the median time during which patients in the study group received corticosteroids was 10 months, compared with 27 months for controls (P < 0.0001). As a result, the cumulative prednisone dose in the study group was approximately half that in the control group (7,574 mg versus 15,152 mg; P = 0.0006). The study group had greater height velocity during the first year of treatment and a smaller increase in the body mass index over the first 2 years. In the control group, the relative risk of cataracts developing was 1.95 (95% confidence interval 1.05–4.17). Side effects of MTX were rarely observed.Conclusion: Use of MTX in conjunction with an aggressively tapered course of prednisone may be as effective as traditional long‐term corticosteroid therapy for children with DM, while decreasing the cumulative dose of corticosteroids.

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 [r_s ] = .95, P < .001). Moderate correlations were seen between the FISH (r_s = .50, P < .001) and SF‐MPQ Present Pain Intensity (r_s = .50, P < .001), while a modest correlation was found with the HAL (r_s = −.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.