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Made available in DSpace on 2022-04-28T19:49:00Z (GMT). No. of bitstreams: 0 Previous issue date: 2022-02-01 Arthritis Foundation Institute of Clinical and Translational Sciences Conselho Nacional de Desenvolvimento Científico e Tecnológico (CNPq) Fundação de Amparo à Pesquisa do Estado de São Paulo (FAPESP) National Institute of Arthritis and Musculoskeletal and Skin Diseases National Institute of Diabetes and Digestive and Kidney Diseases National Center for Advancing Translational Sciences Objective: To develop a standardized steroid dosing regimen (SSR) for physicians treating childhood-onset systemic lupus erythematosus (SLE) complicated by lupus nephritis (LN), using consensus formation methodology. Methods: Parameters influencing corticosteroid (CS) dosing were identified (step 1). Data from children with proliferative LN were used to generate patient profiles (step 2). Physicians rated changes in renal and extrarenal childhood-onset SLE activity between 2 consecutive visits and proposed CS dosing (step 3). The SSR was developed using patient profile ratings (step 4), with refinements achieved in a physician focus group (step 5). A second type of patient profile describing the course of childhood-onset SLE for ≥4 months since kidney biopsy was rated to validate the SSR-recommended oral and intravenous (IV) CS dosages (step 6). Patient profile adjudication was based on majority ratings for both renal and extrarenal disease courses, and consensus level was set at 80%. Results: Degree of proteinuria, estimated glomerular filtration rate, changes in renal and extrarenal disease activity, and time since kidney biopsy influenced CS dosing (steps 1 and 2). Considering these parameters in 5,056 patient profile ratings from 103 raters, and renal and extrarenal course definitions, CS dosing rules of the SSR were developed (steps 3–5). Validation of the SSR for up to 6 months post–kidney biopsy was achieved with 1,838 patient profile ratings from 60 raters who achieved consensus for oral and IV CS dosage in accordance with the SSR (step 6). Conclusion: The SSR represents an international consensus on CS dosing for use in patients with childhood-onset SLE and proliferative LN. The SSR is anticipated to be used for clinical care and to standardize CS dosage during clinical trials. University of Cincinnati College of Medicine Baylor College of Medicine The Hospital for Sick Children and The University of Toronto Emory University and Children's Healthcare of Atlanta Sanjay Gandhi Postgraduate Institute of Medical Sciences Medical University of South Carolina KK Women's and Children's Hospital Cincinnati Children's Hospital Medical Center Hospital for Special Surgery Albert Einstein College of Medicine SRCC Children's Hospital Capital Medical University and National Center for Children's Health Universidade Federal do Rio de Janeiro Nationwide Children's Hospital University College London Cerrahpasa Medical School Istanbul University-Cerrahpasa University of Zagreb School of Medicine IWK Health Centre and Dalhousie University Hacettepe University Northwestern University Feinberg School of Medicine and Ann & Robert H. Lurie Children's Hospital of Chicago University of Campinas Hospital das Clínicas da Universidade Federal de Pernambuco National and Kapodistian University of Athens University Teknologi MARA Instituto Mexicano del Seguro Social University of Washington and Seattle Children's Hospital São Paulo State University Amsterdam University Medical Center Red Cross War Memorial Children's Hospital and University of Cape Town Universidade de São Paulo Hospital de la Beneficencia Española Universidade Federal de São Paulo National Medical Center La Raza Aristotle University of Thessaloniki King Faisal Specialist Hospital and Research Center and Alfaisal University University of Cincinnati College of Medicine and Cincinnati Children's Hospital Medical Center São Paulo State University CNPq: 303422/2015-7 FAPESP: FAPESP 2015/03756-4 National Institute of Arthritis and Musculoskeletal and Skin Diseases: P30-AR-076316 National Institute of Diabetes and Digestive and Kidney Diseases: P50-DK-096418 National Institute of Arthritis and Musculoskeletal and Skin Diseases: R34-AR-071651 National Center for Advancing Translational Sciences: T32-AR-050958

We undertook this study to validate the Myositis Damage Index (MDI) in juvenile and adult myositis, to describe the degree and types of damage and to develop predictors of damage.Retrospective MDI evaluations and prospective assessment of disease activity and illness features were conducted. Patients with juvenile-onset disease (n = 143) were evaluated a median of 18 months after diagnosis; 135 patients were assessed 7-9 months later, and 121 were last assessed a median of 82 months after diagnosis. Ninety-six patients with adult-onset dermatomyositis or polymyositis had a baseline assessment a median of 30 months after diagnosis; 77 patients had a 6-month followup evaluation, and 55 had a final assessment a median of 60 months after diagnosis.Damage was present in 79% of juvenile patients and in 97% of adult patients. In juveniles, scarring, contractures, persistent weakness, muscle dysfunction, and calcinosis were most frequent (23-30%) at the last evaluation. In adults, muscle atrophy, muscle dysfunction, and muscle weakness were most frequent (74-84%). MDI severity correlated with physician-assessed global damage, serum creatinine, and muscle atrophy on magnetic resonance imaging, and in juveniles also with functional disability and weakness. MDI damage scores and frequency were highest in patients with a chronic illness course and in adult patients who died. Predictors of damage included functional disability, duration of active disease, disease severity at diagnosis, physician-assessed global disease activity, and illness features, including ulcerations in children and pericarditis in adults.Damage is common in myositis after a median duration of 5 years in patients with adult-onset disease and 6.8 years in patients with juvenile-onset disease. The MDI has good content, construct, and predictive validity in juvenile and adult myositis.