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OBJECTIVES: The aim of the study was to establish an international multicenter registry to collect data on patients with Multisystem Inflammatory Syndrome in Children (MIS-C), in order to highlight a relationship between clinical presentation, age of onset and geographical distribution on the clinical outcome. STUDY DESIGN: Multicenter retrospective study involving different international societies for rare immunological disorders.1009 patients diagnosed with MIS-C between March and September 2022, from 48 centers and 22 countries were collected. Five age groups (<1, 1-4, 5-11, 12-16, >16 years) and four geographic macro-areas, Western Europe, Central-Eastern Europe, Latin America, Asian-African resource-limited countries (LRC), were identified. RESULTS: Time to referral was significantly higher in LRC. Intensive anti-inflammatory treatment, including biologics, respiratory support and mechanic ventilation were more frequently used in older children and in European countries. The mortality rate was higher in very young children (<1 year), in older patients (>16 years of age) and in LRC. Multivariate analysis identified the residence in LRC, presence of severe cardiac involvement, renal hypertension, lymphopenia and non-use of heparin prophylaxis, as the factors most strongly associated with unfavorable outcomes. CONCLUSIONS: The stratification of patients by age and geographic macro-area provided insights into the clinical presentation, treatment and outcome of MIS-C. The mortality and sequelae rates exhibited a correlation with the age and geographical areas. Patients admitted and treated in LRC displayed more severe outcomes, possibly due to delays in hospital admission and limited access to biologic drugs and to intensive care facilities.
RESUMO
BACKGROUND: Biological disease-modifying antirheumatic drugs (bDMARDs) are treatment options for refractory juvenile idiopathic arthritis (JIA) cases which cannot be afforded by the majority. Tofacitinib is a novel Janus kinase inhibitor, which is reported to be a cost-effective oral alternative to biologics. This prospective observational study was carried out to observe the efficacy and safety of tofacitinib in refractory polyarticular course JIA patients. MATERIALS AND METHODS: The study was conducted in the Department of Pediatrics, Bangabandhu Sheikh Mujib Medical University (BSMMU), Dhaka. Twenty-seven refractory polyarticular course JIA patients were included in this study. Patients received tofacitinib at recommended doses. Disease activity level was assessed by Juvenile Arthritis Disease Activity Score (JADAS) 27. Cases were evaluated at baseline and at the 6th, 12th and 24th weeks. Safety was monitored from history, examination findings and laboratory reports. Data were analyzed using appropriate statistical tests. RESULTS: Enthesitis-related arthritis was the commonest type (37%) followed by polyarticular (rheumatoid factor+) and systemic JIA. There was significant improvement in JADAS 27 in all the subtypes of JIA except oligoarticular extended type. Among 100% high disease activity state cases at baseline, 70.4% were inactive at 24 weeks. It was also possible to significantly reduce the dose of steroid. Few side effects like headache and vomiting, elevation of alanine aminotransferase and anemia were observed at 6 weeks. These side effects subsequently improved. CONCLUSION: Significant reduction of disease activity score was observed from baseline to follow up in this study. Tofacitinib was well tolerated with minimum side effects.
