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

Abstract

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.

Criteria to define response to therapy in paediatric rheumatic diseases

“…Consensus formation techniques require that each step is based on the results of the previous steps. These techniques have been used to develop the outcome measures of several chronic rheumatic diseases, including JIA [9,10], adult rheumatoid arthritis [11], adult-onset SLE [12,13], and idiopathic inflammatory myopathies [14][15][16] as well as JSLE [17][18][19] and JDM [20].…”

Could C-reactive protein and erythrocyte sedimentation rate support monitoring of dermatomyositis and polymyositis activity?

“…8,9 However, the IMACS does not include erythrocyte sedimentation rate (ESR) or C-reactive protein (CRP), which have been used indiscriminately as possible markers of DM and PM activity. [10][11][12][13][14][15][16][17][18][19][20][21][22][23][24] High ESR levels have been correlated with neoplasia risk [11][12][13][14][15][16] and pulmonary involvement [17][18][19][20][21][22] in inflammatory myopathies.…”

H.P. Acthar® Gel in Dermatomyositis and Polymyositis Treatment Registry: An Interim Analysis

“…At baseline, and at months 3, 6, 9, and 12, a physical exam, manual muscle testing (MMT) of 18 muscle groups both proximal and distal and assessed bilaterally, as recommended by the International Myositis Assessment and Clinical Studies Group (IMACS) [21,22], assessment of the patient by both the inflammatory neuropathy cause and treatment (INCAT) disability scale [23] and the myositis activities profile (MAP) [24], and collection of both adverse events (AEs) and serious adverse events (SAEs) were conducted. Additionally, patient laboratory results were also reported, including myositis specific antibodies, glycated hemoglobin (HbA1c), and creatine phosphokinase (CPK).…”