Background JIA-associated uveitis (JIAU) is a serious, sight-threatening disease with significant long-term complications and risk of blindness, even with improved contemporary treatments. The MIWGUC was set up in order to propose specific JIAU activity and response items and to validate their applicability for clinical outcome studies. Methods The group consists of 8 paediatric rheumatologists and 7 ophthalmologists. A consensus meeting took place on November 2015 in Barcelona (Spain) with the objective of validating the previously proposed measures. The validation process was based on the results of a prospective open, international, multi-centre, cohort study designed to validate the outcome measures proposed by the initial MIWGUC group meeting in 2012. The meeting used the same Delphi and nominal group technique as previously described in the first paper from the MIWGUC group (Arthritis Care Res 64:1365–72, 2012). Patients were included with a diagnosis of JIA, aged less than 18 years, and with active uveitis or an uveitis flare which required treatment with a disease-modifying anti-rheumatic drug. The proposed outcome measures for uveitis were collected by an ophthalmologist and for arthritis by a paediatric rheumatologist. Patient reported outcome measures were also measured. Results A total of 82 patients were enrolled into the validation cohort. Fifty four percent (n = 44) had persistent oligoarthritis followed by rheumatoid factor negative polyarthritis (n = 15, 18%). The mean uveitis disease duration was 3.3 years (SD 3.0). Bilateral eye involvement was reported in 65 (79.3%) patients. The main findings are that the most significant changes, from baseline to 6 months, are found in the AC activity measures of cells and flare. These measures correlate with the presence of pre-existing structural complications and this has implications for the reporting of trials using a single measure as a primary outcome. We also found that visual analogue scales of disease activity showed significant change when reported by the ophthalmologist, rheumatologist and families. The measures formed three relatively distinct groups. The first group of measures comprised uveitis activity, ocular damage and the ophthalmologists’ VAS. The second comprised patient reported outcomes including disruption to school attendance. The third group consisted of the rheumatologists’ VAS and the joint score. Conclusions We propose distinctive and clinically significant measures of disease activity, severity and damage for JIAU. This effort is the initial step for developing a comprehensive outcome measures for JIAU, which incorporates the perspectives of rheumatologists, ophthalmologists, patients and families.

Background Systemic autoinflammatory diseases (SAID) are monogenic and polygenic inherited conditions characterized by dysregulation of the innate immune system. Objective We aimed to characterize the clinical features of patients with SAID. Methods This study was a retrospective chart review on the clinical and genetic features of the pediatric population with SAID observed from 1998 to 2020 in our center. Results A total of 54 patients were evaluated: 18 with periodic fever, aphthous stomatitis, pharyngitis and adenitis syndrome; 16 with Behçet disease; 13 with systemic juvenile idiopathic arthritis; 4 with syndrome of undifferentiated recurrent fever; 1 with cryopyrin associated periodic syndrome; 1 with chronic nonbacterial osteomyelitis; and 1 with Muckle-Wells syndrome. Conclusion The analysis of clinical features of our patients are similar to other studies. Our goal was to aware the medical community to early recognize and treat SAID to improve quality of life of pediatric patients.

No abstract

No abstract

BackgroundSystemic JIA (sJIA) treatment has changed dramatically with the introduction of biologic agents, although treatment approaches likely differ by region.ObjectivesWe compared the initial treatment of sJIA at pediatric rheumatology centers (PR) in the United States (US), United Kingdom (UK), Germany (GER), Portugal (POR), Canada (CAN), Sweden (SWE), and the Nordic countries (NORD) using prospectively collected registry data.MethodsData were extracted locally by the following JIA Registries: Childhood Arthritis and Rheumatology Research Alliance Registry (US); Childhood Arthritis Prospective Study (UK); National Pediatric Rheumatology Database (GER); Reuma.pt (POR); Research in Arthritis in Canadian Children Emphasizing Outcomes (CAN); the Swedish National JIA Registry (SWE) and the Nordic JIA Cohort (NORD; no duplicate patients with SWE). Prospectively collected treatment data covering the first year of disease (defined as 9 to 15 months following first encounter with PR) for children diagnosed with SJIA since 2009 were included. We also collected data on the presenting characteristics (defined as within 2 months of the first encounter with PR) of all children with sJIA within each registry. Data were compared across registries using ANOVA and chi-square.ResultsOverall, there were data available about the presenting characteristics for 486 patients and about the first year of treatment for 431 patients. There were differences in the sex distribution of the patients (more females in US) and in the elapsed time from symptom onset to evaluation by PR (shorter duration in GER). There were differences in presenting characteristics, including less frequent evanescent rash in GER and POR, less frequent lymphadenopathy in GER and CAN, and more frequent hepatosplenomegaly in GER. The proportion of patients receiving each medication in the first year of disease is shown in the Table. The use of all medication classes was significantly different among the registries (p<0.002 in all cases). Systemic glucocorticoids were less frequently used in UK. Methotrexate was more frequently used in UK and NORD. Biologics were more frequently used in US and NORD and less frequently used in GER.RegistryUSUKGERPORCANSWENORDN75212591414435Systemic Glucocorticoid80%57%71%71%93%95%100%Methotrexate61%76%42%57%64%40%100%Cyclosporine8%5%1%0%21%5%0%Any Biologic61%29%18%29%29%47%100%IL1 Inhibitor44%5%10%29%21%23%40%Tocilizumab12%24%5%0%0%21%60%TNF Inhibitor16%5%4%0%21%21%0%ConclusionsPresenting characteristics of children with sJIA were different among the registries. It is unknown how much of this variation may be attributable to differences in ascertainment and patient enrollment. There were marked differences in the treatment of sJIA in the first year of disease among the registries, particularly with respect to the use of biologic agents. The impact of these different treatment approaches on patient outcomes is not known, but is worthy of further investigation.Disclosure of InterestT. Beukelman Consultant for: Novartis; Genentech/Roche;...