Proposed preliminary core set measures for disease outcome assessment in adult and juvenile idiopathic inflammatory myopathies

128

115

Objective. To investigate the benefits and safety of an intensive muscular training program in patients with chronic polymyositis (PM) and dermatomyositis (DM).Methods. Nine patients with chronic PM or DM (median age 53 years, range 44 -61) were included. Assessments of impairment (10 -15 voluntary repetition maximum [VRM], the Functional Index 2 [FI-2], the Grippit, and pain rated on the Borg CR-10 scale), activity limitation (Myositis Activities Profile), and participation restriction (patients' disease impact on well-being) were performed 4 weeks prior to baseline, at baseline, and after 7 weeks of exercise. A 6-item core set of disease activity measures was administered and muscle biopsy samples of vastus lateralis were obtained at baseline and after 7 weeks of exercise. Response criteria at an individual level were set for disability and disease activity. The patients exercised 3 days per week for 7 weeks on loads allowing 10 VRM. Results. On a group level there were no significant differences between assessments at 4 weeks before baseline compared with baseline. The group improved significantly regarding 10 -15 VRM and FI-2 at 7 weeks compared with baseline (P < 0.05). All patients were responders with respect to impairment and 2 were activity limitation responders whereas participation restriction remained unchanged in all. Two patients were responders with reduced disease activity and no patient had signs of increased muscle inflammation in the muscle biopsy sample after 7 weeks of exercise. Conclusion. Patients with chronic, stable PM and DM can perform this intensive resistive exercise program with beneficial effects on impairment and activity limitation without increased muscle inflammation.

Section: Intensive Resistance Training In Pm and Dmmentioning

confidence: 99%

Benefits of intensive resistance training in patients with chronic polymyositis or dermatomyositis

Alexanderson¹,

Dastmalchi²,

Esbjörnsson-Liljedahl³

et al. 2007

128

115

“…Skin damage caused by chronic skin lesions, such as calcinosis, is an important source of morbidity in juvenile DM, and may be associated with pain, disfigurement, and reductions in quality of life and physical function (7). For these reasons, the assessment of skin disease is part of the core sets of outcomes recommended for studies in adult and juvenile DM (8,9). Both of these proposed core sets recommend the assessment of extramuscular disease in the overall assessment of disease activity and damage.…”

Section: Introductionmentioning

confidence: 99%

Preliminary validation and clinical meaning of the cutaneous assessment tool in juvenile dermatomyositis

Huber

Dugan

Lachenbruch

et al. 2008

Objective. To provide preliminary validation of the Cutaneous Assessment Tool (CAT), a new tool to assess cutaneous manifestations of juvenile dermatomyositis (DM), and to explore the clinical meaning of CAT scores. Methods. Children with juvenile DM (n ‫؍‬ 113) were assessed at baseline and 7-9 months later (n ‫؍‬ 94). Internal consistency, redundancy, construct validity, and responsiveness of the CAT were examined. CAT scores corresponding to ordinal global assessments were determined. Results. Item-total correlations ranged from 0.27-0.67 for activity lesions present in >10% of patients; item-domain and domain-total correlations ranged from 0.25-0.99. Cronbach's alpha was 0.79 for the CAT activity score and 0.74 for the CAT damage score. As predicted, the CAT activity score correlated strongly with both global disease activity and skin disease activity and moderately with the Childhood Myositis Assessment Scale, whereas the CAT damage score correlated moderately with the physician global disease and skin disease damage scores. Median CAT activity scores of 1, 7, 13, 18, and 31 corresponded to absent, mild, moderate, severe, and extremely severe skin disease activity, respectively. Median CAT damage scores of 0, 1, 2, and 5 correlated with the same descriptions of damage (severe and extremely severe combined). Conclusion. Preliminary validation of the CAT demonstrated good internal consistency, nonredundancy, good construct validity, and appropriate responsiveness. The CAT is a comprehensive, semiquantitative assessment tool for skin disease in juvenile DM.

“…Many of the previously used end points have been unidimensional, focused exclusively on improvement in muscle strength in these systemic rheumatic diseases. Also, end points in IIM therapeutic trials have not been adequately validated, and investigators have used nonstandardized assessment techniques or have reported on a degree of change that does not clearly represent clinically important improvement for the measures (3,4). This use of nonstandardized outcome assessments has yielded inconclusive results, insufficient power from multiple end points, an inability to compare the results of different trials, and, overall, an absence of proven therapies for these disorders.…”

mentioning

confidence: 99%

International consensus on preliminary definitions of improvement in adult and juvenile myositis

Rider

Giannini

Brunner

et al. 2004

215

164

Objective. To use a core set of outcome measures to develop preliminary definitions of improvement for adult and juvenile myositis as composite end points for therapeutic trials.Methods. Twenty-nine experts in the assessment of myositis achieved consensus on 102 adult and 102 juvenile paper patient profiles as clinically improved or not improved. Two hundred twenty-seven candidate definitions of improvement were developed using the experts' consensus ratings as a gold standard and their judgment of clinically meaningful change in the core set of measures. Seventeen additional candidate definitions of improvement were developed from classification and regression tree analysis, a data-mining decision tree tool analysis. Six candidate definitions specifying percentage change or raw change in the core set of measures were developed using logistic regression analysis. Adult and pediatric working groups ranked the 13 top-performing candidate definitions for face validity, clinical sensibility, and ease of use, in which the sensitivity and specificity were >75% in adult, pediatric, and combined data sets. Nominal group technique was used to facilitate consensus formation.Results. The definition of improvement (common to the adult and pediatric working groups) that ranked highest was 3 of any 6 of the core set measures improved by >20%, with no more than 2 worse by >25% (which could not include manual muscle testing to assess strength). Five and 4 additional preliminary definitions of improvement for adult and juvenile myositis, respectively, were also developed, with several definitions common to both groups. Participants also agreed to prospectively test 6 logistic regression definitions of improvement in clinical trials.Conclusion. Consensus preliminary definitions of improvement were developed for adult and juvenile myositis, and these incorporate clinically meaningful change in all myositis core set measures in a composite end point. These definitions require prospective validation, but they are now proposed for use as end points in all myositis trials.