A national registry for juvenile dermatomyositis and other paediatric idiopathic inflammatory myopathies: 10 years' experience; the Juvenile Dermatomyositis National (UK and Ireland) Cohort Biomarker Study and Repository for Idiopathic Inflammatory Myopathies

Objectives: The paediatric idiopathic inflammatory myopathies (IIMs) are a group of rare chronic inflammatory disorders of childhood, affecting muscle, skin and other organs. There is a severe lack of evidence base for current treatment protocols in juvenile myositis. The rarity of these conditions means that multicentre collaboration is vital to facilitate studies of pathogenesis, treatment and disease outcomes. We have established a national registry and repository for childhood IIM, which aims to improve knowledge, facilitate research and clinical trials, and ultimately to improve outcomes for these patients. Methods: A UK-wide network of centres and research group was established to contribute to the study. Standardized patient assessment, data collection forms and sample protocols were agreed. The Biobank includes collection of peripheral blood mononuclear cells, serum, genomic DNA and biopsy material. An independent steering committee was established to oversee the use of data/samples. Centre training was provided for patient assessment, data collection and entry. Results: Ten years after inception, the study has recruited 285 children, of which 258 have JDM or juvenile PM; 86% of the cases have contributed the biological samples. Serial sampling linked directly to the clinical database makes this a highly valuable resource. The study has been a platform for 20 sub-studies and attracted considerable funding support. Assessment of children with myositis in contributing centres has changed through participation in this study. Conclusions: This establishment of a multicentre registry and Biobank has facilitated research and contributed to progress in the management of a complex group of rare muscloskeletal conditions

The lived experience of juvenile idiopathic arthritis in young people receiving etanercept

BACKGROUND: This study explores young people's daily experiences of living with Juvenile Idiopathic Arthritis (JIA) and their thoughts, beliefs and feelings related to the biological drug Etanercept, prescribed as part of their treatment. METHODS: An Interpretive Phenomenological approach was used to allow in-depth examinations of the young people's personal accounts of their lived experiences. Data were obtained from 6 young people between the ages of 10-13 years, from one tertiary institution's Paediatric Rheumatology department using audio-taped open-ended interviews. RESULTS: The transcripts yielded seven thousand words of data and two hundred significant statements, which were reduced to five themes; 1) Who understands me, 2) Medicines and injections, 3) Challenges of schooling and friendships, 4) Being different, and 5) Exclusion from sports. There were marked similarities between the young people's statements; however, there were also some striking differences. The theme 'Who understands me' yielded the biggest section of data, but also produced the biggest disparity between the young people. Two patients were very clear that they thought everyone 'understands', whilst two other patients held the belief that 'no one understood'. This paper explores these statements in further detail. CONCLUSIONS: The findings from this study can give healthcare professionals novel insight into the likely reactions to treatment for JIA and, through this, enable them to offer improved support, education and early intervention before these issues become a concern. This study also provides insight into the emotional resilience of young people with JIA

The practical management of emergencies in primary care: Taking simulation out of the classroom and into real-life environments

Life threatening emergencies in the community are relatively infrequent and therefore provide a challenge for doctors in keeping up-to-date and maintaining confidence. Training in managing emergencies typically takes place through role play and classroom based simulation. In this project, we took simulation out of the classroom and into community environments where emergencies actually occur creating 'real-life' scenarios. These included the practical management of meningitis, anaphylaxis, hypoglycaemia, convulsions and cardiac arrest. Doctors had to find and utilize the equipment in their surgeries and were asked to physically draw up the appropriate medication. The simulation training was led by a GP and a Consultant in Intensive Care Medicine. Participants' confidence in managing emergencies significantly increased after the workshops. Qualitative feedback illustrated the need for more simulation based learning: "I hope this can be done regularly as it will make a huge difference to patient care", "Excellent - life like to make more memorable", "Good to use the actual surgery equipment". Many of the participants knew the theory of what to do but lacked the practical skills to efficiently manage emergency scenarios. Training doctors through simulation needs to be taken out of the classroom and into real life environments. This is particularly important for 'time critical illnesses' where delays can have a direct impact on morbidity and mortality

Update in juvenile myositis.

This update on childhood idiopathic inflammatory myopathies (IIMs) reviews recent progress in the field of translational science and clinical research over the past 12-18 months

CD161(+) Tconv and CD161(+) Treg Share a Transcriptional and Functional Phenotype despite Limited Overlap in TCR beta Repertoire

Human regulatory T cells (Treg) are important in immune regulation, but can also show plasticity in specific settings. CD161 is a lectin-like receptor and its expression identifies an effector-like Treg population. Here, we determined how CD161+ Treg relate to CD161+ conventional T cells (Tconv). Transcriptional profiling identified a shared transcriptional signature between CD161+ Tconv and CD161+ Treg, which is associated with T helper (Th)1 and Th17 cells, and tissue homing, including high expression of gut-homing receptors. Upon retinoic acid (RA) exposure, CD161+ T cells were more enriched for CCR9+ and integrin α4+β7+ cells than CD161− T cells. In addition, CD161+ Tconv and CD161+ Treg were enriched at the inflamed site in autoimmune arthritis, and both CD161+ and CD161− Treg from the inflamed site were suppressive in vitro. CD161+ T cells from the site of autoimmune arthritis showed a diminished gut-homing phenotype and blunted response to RA suggesting prior imprinting by RA in the gut or at peripheral sites rather than during synovial inflammation. TCRβ repertoires of CD161+ and CD161− Tconv and Treg from blood showed limited overlap whereas there was clear overlap between CD161+ and CD161− Tconv, and CD161+ and CD161− Treg from the inflamed site suggesting that the inflamed environment may alter CD161 levels, potentially contributing to disease pathogenesis

Managing emergencies in primary care: does real-world simulation-based training have any lasting impact?

General Practitioners (GPs) have a responsibility to provide prompt and effective care when attending to life threatening emergencies in their GP surgeries. Primary care staff undertake mandatory, annual basic life support training. However, most emergencies are peri-arrest situations, and this is an area where GPs lack confidence and competence [1, 2]. The importance of effective, early intervention in peri-arrest scenarios was highlighted by the NCEPOD report “Time to Intervene (2012)” [3]. This report suggested that better early assessment and intervention may have prevented progression to cardiorespiratory arrest. GPs need to be equipped to manage ‘time critical’ emergencies, particularly as GP surgeries are deemed a place of safety and 999 ambulances can be redirected to other emergencies, thereby delaying transfer to secondary care for patients in GP settings. In previous work, we demonstrated that GPs’ confidence in managing time critical emergencies was initially low, and significantly improved immediately after attending ‘real-world’, simulation based workshops [1]. The value of real-world, in-house simulation based training has also been shown to increase “practical preparedness” in the context of resuscitation training (4). However, there is relatively little data regarding the long term value of simulation based training in primary care (5). In the current paper, we assessed whether our workshops had any longer-term benefit on participants’ confidence in managing emergencies and if it led to any changes in clinical practice

Advances in the treatment of polyarticular juvenile idiopathic arthritis

Purpose of review: To review recent advances in the management strategies of polyarticular course juvenile idiopathic arthritis (JIA) and identify unanswered questions and avenues for further research. Recent findings: There is evidence for an early, aggressive, treat-to-target approach for polyarticular JIA. Clinical disease activity criteria have been recently defined and validated, including criteria for inactive disease and the juvenile arthritis disease activity score (JADAS). There is a need for evidence-based, defined disease targets and biomarkers for prediction of response, including targets for remission induction, and guidelines on drug withdrawal. Recent treatment consensus plans and guidelines are discussed and compared, including the 2015 NHS England clinical policy statement, the 2014 Childhood Arthritis and Rheumatology Research Alliance (CARRA) treatment plans and the 2011 American College of Rheumatology (ACR) guidelines. Evidence for new agents such as tocilizumab, rituximab, golimumab, ustekinumab, certolizumab and tofacitinib is promising: the recent clinical trials are summarized here. Stratification of individual patient treatment remains a goal, and predictive biomarkers have been shown to predict success in the withdrawal of methotrexate therapy. Summary: There are promising advances in the treatment approaches, disease activity criteria, clinical guidelines, pharmaceutical choices and individually stratified therapy choices for polyarticular JIA