OBJECTIVE: To quantify the impact of inflammatory brain diseases in the pediatric population on health-related quality of life, including the subdomains of physical, emotional, school and social functioning. METHODS: This was a multicenter, observational cohort study of children (< 18 years of age) diagnosed with inflammatory brain disease (IBrainD). Patients were included if they had completed at least one Health Related Quality of Life Questionnaire (HRQoL). HRQoL was measured using the Pediatric Quality of Life Inventory Version 4.0 (PedsQL) Generic Core Scales, which provided a total score out of 100. Analyses of trends were performed using linear regression models adjusted for repeated measures over time. RESULTS: In this study, 145 patients were included of which 80 (55%) were females. Cognitive dysfunction was the most common presenting symptoms (63%), and small vessel childhood primary angiitis of the CNS was the most common diagnosis (33%). The mean child's self-reported PedsQL total score at diagnosis was 68.4, and the mean parent's proxy-reported PedsQL score was 63.4 at diagnosis. Child's self-reported PedsQL scores reflected poor HRQoL in 52.9% of patients at diagnosis. Seizures or cognitive dysfunction at presentation was associated with statistically significant deficits in HRQoL. CONCLUSION: Pediatric IBrainD is associated with significantly diminished health-related quality of life. Future research should elucidate why these deficits occur and interventions should focus on improving HRQoL in the most affected subdomains, in particular for children presenting with seizures and cognitive dysfunction.

• Klíčová slova
• CNS vasculitis, Health-related quality of life, Inflammatory brain disease, Pediatrics, Quality of life,
• MeSH
• dítě MeSH
• kohortové studie MeSH
• kojenec MeSH
• kvalita života * MeSH
• lidé MeSH
• mladiství MeSH
• nemoci mozku diagnóza   psychologie   MeSH
• předškolní dítě MeSH
• průzkumy a dotazníky MeSH
• psychometrie metody   MeSH
• rizikové faktory MeSH
• rodiče psychologie   MeSH
• Check Tag
• dítě MeSH
• kojenec MeSH
• lidé MeSH
• mladiství MeSH
• mužské pohlaví MeSH
• předškolní dítě MeSH
• ženské pohlaví MeSH
• Publikační typ
• časopisecké články MeSH
• multicentrická studie MeSH
• pozorovací studie MeSH

OBJECTIVE: The objective was to describe the methodology used to develop new response criteria for adult DM/PM and JDM. METHODS: Patient profiles from prospective natural history data and clinical trials were rated by myositis specialists to develop consensus gold-standard ratings of minimal, moderate and major improvement. Experts completed a survey regarding clinically meaningful improvement in the core set measures (CSM) and a conjoint-analysis survey (using 1000Minds software) to derive relative weights of CSM and candidate definitions. Six types of candidate definitions for response criteria were derived using survey results, logistic regression, conjoint analysis, application of conjoint-analysis weights to CSM and published definitions. Sensitivity, specificity and area under the curve were defined for candidate criteria using consensus patient profile data, and selected definitions were validated using clinical trial data. RESULTS: Myositis specialists defined the degree of clinically meaningful improvement in CSM for minimal, moderate and major improvement. The conjoint-analysis survey established the relative weights of CSM, with muscle strength and Physician Global Activity as most important. Many candidate definitions showed excellent sensitivity, specificity and area under the curve in the consensus profiles. Trial validation showed that a number of candidate criteria differentiated between treatment groups. Top candidate criteria definitions were presented at the consensus conference. CONCLUSION: Consensus methodology, with definitions tested on patient profiles and validated using clinical trials, led to 18 definitions for adult PM/DM and 14 for JDM as excellent candidates for consideration in the final consensus on new response criteria for myositis.

• Klíčová slova
• 1000Minds software, conjoint analysis, dermatomyositis, hybrid measure, juvenile dermatomyositis, outcome assessment, polymyositis, response criteria,
• MeSH
• dermatomyozitida terapie   MeSH
• lidé MeSH
• logistické modely MeSH
• minimální klinicky významný rozdíl MeSH
• plocha pod křivkou MeSH
• polymyozitida terapie   MeSH
• výsledek terapie MeSH
• Check Tag
• lidé MeSH
• Publikační typ
• časopisecké články MeSH
• konsensus - konference MeSH

To develop response criteria for juvenile dermatomyositis (DM). We analysed the performance of 312 definitions that used core set measures from either the International Myositis Assessment and Clinical Studies Group (IMACS) or the Paediatric Rheumatology International Trials Organisation (PRINTO) and were derived from natural history data and a conjoint analysis survey. They were further validated using data from the PRINTO trial of prednisone alone compared to prednisone with methotrexate or cyclosporine and the Rituximab in Myositis (RIM) trial. At a consensus conference, experts considered 14 top candidate criteria based on their performance characteristics and clinical face validity, using nominal group technique. Consensus was reached for a conjoint analysis-based continuous model with a total improvement score of 0-100, using absolute per cent change in core set measures of minimal (≥30), moderate (≥45), and major (≥70) improvement. The same criteria were chosen for adult DM/polymyositis, with differing thresholds for improvement. The sensitivity and specificity were 89% and 91-98% for minimal improvement, 92-94% and 94-99% for moderate improvement, and 91-98% and 85-86% for major improvement, respectively, in juvenile DM patient cohorts using the IMACS and PRINTO core set measures. These criteria were validated in the PRINTO trial for differentiating between treatment arms for minimal and moderate improvement (p=0.009-0.057) and in the RIM trial for significantly differentiating the physician's rating for improvement (p<0.006). The response criteria for juvenile DM consisted of a conjoint analysis-based model using a continuous improvement score based on absolute per cent change in core set measures, with thresholds for minimal, moderate, and major improvement.

• Klíčová slova
• Dermatomyositis, Polymyositis, Treatment,
• MeSH
• dermatomyozitida terapie   MeSH
• dítě MeSH
• dospělí MeSH
• hodnocení výsledků zdravotní péče normy   MeSH
• konsensus MeSH
• lidé MeSH
• mladiství MeSH
• předškolní dítě MeSH
• randomizované kontrolované studie jako téma MeSH
• senzitivita a specificita MeSH
• stupeň závažnosti nemoci * MeSH
• Check Tag
• dítě MeSH
• dospělí MeSH
• lidé MeSH
• mladiství MeSH
• předškolní dítě MeSH
• Publikační typ
• časopisecké články MeSH
• konsensus - konference MeSH
• směrnice pro lékařskou praxi MeSH
• validační studie MeSH

OBJECTIVE: To develop response criteria for juvenile dermatomyositis (DM). METHODS: We analyzed the performance of 312 definitions that used core set measures from either the International Myositis Assessment and Clinical Studies Group (IMACS) or the Paediatric Rheumatology International Trials Organisation (PRINTO) and were derived from natural history data and a conjoint analysis survey. They were further validated using data from the PRINTO trial of prednisone alone compared to prednisone with methotrexate or cyclosporine and the Rituximab in Myositis (RIM) trial. At a consensus conference, experts considered 14 top candidate criteria based on their performance characteristics and clinical face validity, using nominal group technique. RESULTS: Consensus was reached for a conjoint analysis-based continuous model with a total improvement score of 0-100, using absolute percent change in core set measures of minimal (≥30), moderate (≥45), and major (≥70) improvement. The same criteria were chosen for adult DM/polymyositis, with differing thresholds for improvement. The sensitivity and specificity were 89% and 91-98% for minimal improvement, 92-94% and 94-99% for moderate improvement, and 91-98% and 85-86% for major improvement, respectively, in juvenile DM patient cohorts using the IMACS and PRINTO core set measures. These criteria were validated in the PRINTO trial for differentiating between treatment arms for minimal and moderate improvement (P = 0.009-0.057) and in the RIM trial for significantly differentiating the physician's rating for improvement (P < 0.006). CONCLUSION: The response criteria for juvenile DM consisted of a conjoint analysis-based model using a continuous improvement score based on absolute percent change in core set measures, with thresholds for minimal, moderate, and major improvement.

• MeSH
• alanintransaminasa metabolismus   MeSH
• aldolasa metabolismus   MeSH
• antirevmatika terapeutické užití   MeSH
• aspartátaminotransferasy metabolismus   MeSH
• cyklosporin terapeutické užití   MeSH
• dermatomyozitida farmakoterapie   metabolismus   patofyziologie   MeSH
• dítě MeSH
• glukokortikoidy terapeutické užití   MeSH
• hodnocení výsledků péče pacientem MeSH
• hodnocení výsledků zdravotní péče MeSH
• kreatinkinasa metabolismus   MeSH
• L-laktátdehydrogenasa metabolismus   MeSH
• lidé MeSH
• logistické modely MeSH
• methotrexát terapeutické užití   MeSH
• mladiství MeSH
• prednison terapeutické užití   MeSH
• průzkumy a dotazníky MeSH
• reprodukovatelnost výsledků MeSH
• revmatologie MeSH
• rituximab terapeutické užití   MeSH
• společnosti lékařské MeSH
• svalová síla MeSH
• výsledek terapie MeSH
• Check Tag
• dítě MeSH
• lidé MeSH
• mladiství MeSH
• Publikační typ
• časopisecké články MeSH
• konsensus - konference MeSH
• Geografické názvy
• Evropa MeSH
• Spojené státy americké MeSH
• Názvy látek
• alanintransaminasa MeSH
• aldolasa MeSH
• antirevmatika MeSH
• aspartátaminotransferasy MeSH
• cyklosporin MeSH
• glukokortikoidy MeSH
• kreatinkinasa MeSH
• L-laktátdehydrogenasa MeSH
• methotrexát MeSH
• prednison MeSH
• rituximab MeSH