BackgroundThe advent of new treatments for Juvenile Idiopathic Arthritis (JIA) has prompted interest in systematically studying the outcomes of patients treated in the ‘modern era’. Such data provide both benchmarks for assessing local outcomes and important information for use in counselling families of newly diagnosed patients. While data are available for cohorts in Europe and North America, no such data exist for Australian patients.The aim was to examine the demographics, treatment and outcomes at 12 months of an inception cohort of newly diagnosed patients with JIA at a single tertiary referral paediatric rheumatology centre in Australia.MethodsRetrospective review of prospectively collected data from patients newly diagnosed with JIA between 2010 and 2014 at the Royal Children’s Hospital in Melbourne.ResultsOne hundred thirty four patients were included (62% female). Oligoarthritis was the single largest category of JIA (36%) and rheumatoid factor positive polyarthritis the least common (2%). Undifferentiated JIA accounted for 13% of patients and was the third largest category. Across the cohort 94% received NSAIDs, 53% oral steroids, 62% methotrexate and 15% a biologic DMARD. Intra-articular steroids were used in 62%, most commonly in the oligoarticular subtype (94%). 95% of patients achieved a joint count of zero at a median of 4.1 months, however flares occurred in 42%. At 12 months 65% had no active joint disease, though more than half remained on medication.ConclusionAustralian children with JIA managed in the modern era have similar characteristics and achieve short term outcomes comparable to cohorts in Europe and North America, with high rates of joint remission in the first 12 months of follow-up but with a significant relapse rate and requirement for ongoing medication.

CLIPPERS (chronic lymphocytic inflammation with pontine perivascular enhancement responsive to steroids) is an extremely rare neurologic inflammatory condition. Fewer than 10 pediatric cases have been described. Debate persists as to whether it is a distinct disease or a clinical, radiologic, and histologic phenotype evolving into another disorder. We propose that CLIPPERS may be a clinical manifestation of an underlying state of immune-dysregulation. We describe the case of the youngest known report of CLIPPERS, an 18-month-old infant from Melbourne, Australia. Reviewing the literature for all reported pediatric cases, we identified that robust investigation and whole exome sequencing was underutilized and proposed diagnostic criteria were frequently unmet. Particular focus should be paid to genes known to cause familial hemophagocytic lymphohistiocytosis (HLH), with the CLIPPERS phenotype manifesting as a form of isolated central nervous system (CNS)-HLH in some patients. Curative treatment options such as hematopoietic stem cell transplantation may be appropriate for some patients and should be considered early.

Background Juvenile Idiopathic Arthritis (JIA) is the most common rheumatic inflammatory disease in childhood. Optimal management requires clinicians to be up to date with the rapidly evolving evidence base. ‘Living’ evidence-based clinical practice guidelines, which integrate new evidence as soon as it is available, are a novel method to enhance the translation of research into practice. To determine the most relevant questions that should be prioritised in national Australian JIA living guidelines, we invited Australian and New Zealand paediatric rheumatologists and other relevant health professionals to identify and rank their most important questions in order of priority. Methods All 47 members of the Australian Paediatric Rheumatology Group (APRG) were invited to participate in a modified Delphi study comprising two rounds. The first round identified demographic information of respondents, current attitudes to guideline use and invited submission of priority management questions. The second round asked respondents to rank 27 collated and refined questions identified in round one in order of priority. Results There were 29 (62%) and 28 (60%) responses to the first and second survey rounds respectively. About two thirds were rheumatologists or trainees (66, 68%), nearly half had more than 10 years of experience (45, 46%) and practice setting was largely hospital (79, 86%) and urban (86, 75%). Most respondents used clinical guidelines in their practice (72% sometimes, 24% often), most frequently American College of Rheumatology (ACR) (66%) and European Alliance of Associations for Rheumatology (EULAR) (59%) guidelines. Reported barriers to guideline use included that they are not up to date and access difficulties. Most respondents (83%) considered Australian guidelines were necessary and two-thirds indicated they would use them if integrated into practice software. The highest ranked topics were down-titration and discontinuation of disease modifying anti-rheumatic drugs (ranked first), best outcome measures (second) and treatment targets in JIA (third). Conclusions There is strong clinician support for the development of Australian living guidelines for JIA. Consensus was reached on the ten top-ranked priority questions. Our guidelines will develop evidence-based recommendations for these high priority questions that will be updated in real time as needed to facilitate rapid translation of evidence into clinical practice.