The Paediatric Rheumatology International Trials Organisation provisional criteria for the evaluation of response to therapy in juvenile dermatomyositis

Abstract

Objective. To develop a provisional definition for the evaluation of response to therapy in juvenile dermatomyositis (DM) based on the Paediatric Rheumatology International Trials Organisation juvenile DM core set of variables. Methods. Thirty-seven experienced pediatric rheumatologists from 27 countries achieved consensus on 128 difficult patient profiles as clinically improved or not improved using a stepwise approach (patient's rating, statistical analysis, definition selection). Using the physicians' consensus ratings as the "gold standard measure," chi-square, sensitivity, specificity, false-positive and-negative rates, area under the receiver operating characteristic curve, and kappa agreement for candidate definitions of improvement were calculated. Definitions with kappa values >0.8 were multiplied by the face validity score to select the top definitions. Results. The top definition of improvement was at least 20% improvement from baseline in 3 of 6 core set variables with no more than 1 of the remaining worsening by more than 30%, which cannot be muscle strength. The second-highest scoring definition was at least 20% improvement from baseline in 3 of 6 core set variables with no more than 2 of the remaining worsening by more than 25%, which cannot be muscle strength (definition P1 selected by the International Myositis Assessment and Clinical Studies group). The third is similar to the second with the maximum amount of worsening set to 30%. This indicates convergent validity of the process. Conclusion. We propose a provisional data-driven definition of improvement that reflects well the consensus rating of experienced clinicians, which incorporates clinically meaningful change in core set variables in a composite end point for the evaluation of global response to therapy in juvenile DM.

Physical (in)activity and its influence on disease-related features, physical capacity, and health-related quality of life in a cohort of chronic juvenile dermatomyositis patients

“…In addition, Pearson's correlation coefficients were calculated to test possible associations between physical activity levels and disease-related parameters, physical capacity variables, and health-related quality-of-life scores in JDM patients. In order to shed light on the influence of disease activity on the results, we performed a sub-analysis by removing the patients with active disease and ran unpaired T-tests and Pearson's correlation coefficients between inactive JDM (i-JDM, n ¼ 17) (i.e., according to previously described criteria [11,12]) and CTRL. Data analyses were performed using SPSS (17.0) for Windows.…”

“…The sample consisted of 19 JDM patients (age between 8 and 22 years) and 19 healthy controls (CTRL) (age between 8 and 22 years) frequency-matched by physical activity levels, age, sex, and body mass index (BMI). JDM consisted of children with active (n ¼ 2) or nonactive (n ¼ 17) disease according to previously described criteria [11,12]. All patients fulfilled the Bohan and Peter criteria for the diagnosis of JDM [13], and had disease onset before age 18 years.…”

Networking in paediatrics: the example of the Paediatric Rheumatology International Trials Organisation (PRINTO)

“…With a second grant from the European Union (contract no. QLG1-CT-2000-00514), PRINTO has been able to propose validated core sets for the evaluation of disease activity and damage23–25 and also definitions of improvement to be used in future clinical trials in JSLE26 and JDM 25 27. Data on 294 patients with JDM and 557 patients with JSLE were collected from 41 countries.…”

“…Data on 294 patients with JDM and 557 patients with JSLE were collected from 41 countries. These projects have been officially endorsed by the ACR and the European League Against Rheumatism (EULAR) and are now known as the PRINTO core set24 and PRINTO/ACR definition of improvement for JSLE26 and the PRINTO/ACR/EULAR core set for JDM25 and PRINTO definition of improvement for JDM 27…”

Lack of Concordance in Interrater Scoring of the Provider's Global Assessment of Children With Juvenile Idiopathic Arthritis With Low Disease Activity

“…Examples include the American College of Rheumatology (ACR) Pediatric 30-90 response criteria (6), the ACR provisional criteria for clinical inactive disease (CID) in patients with select categories of juvenile idiopathic arthritis (JIA) (7), and the definition of clinical improvement in juvenile myositis and childhood systemic lupus erythematosus (8). Additionally, the PGA has served as the gold standard against which other, new descriptors of the patient's condition or state are compared to establish their criterion (concurrent) and construct validity (7,9,10).…”