Search

Dermatomyositis (DM) and juvenile dermatomyositis (JDM) are idiopathic inflammatory myopathies, which can be resistant and unresponsive to initial treatments, leading to severe complications and impaired quality of life. There are few randomised trials in dermatomyositis and the outcomes reported may not be consistent, which can limit decision-making. The aim of this study is to assess the scope and consistency of outcomes reported in randomised trials in dermatomyositis.MEDLINE, Embase, PsycINFO and clinicaltrials.gov were searched from 1993-2020 for randomised trials in children and adults with dermatomyositis. The frequency and characteristics of the outcomes reported were analysed and classified.20 trials were included. Across these trials, a total of 743 outcome measures were reported, which were grouped into 34 outcome domains; of which 17 were clinical, 13 were surrogate/biochemical, and 4 were patient-reported outcomes. The top five most frequently reported outcome domains were muscle inflammation (15 trials, 46 outcome measures), physical function (14 trials, 16 outcome measures), muscle strength (13 trials, 30 outcome measures), global health (12 trials, 33 outcome measures) and immunologic marker (11 trials, 91 outcomes).The majority of outcomes reported in trials in people with dermatomyositis and JDM are clinical and surrogate outcomes rather than patient-reported outcomes. The outcomes reported are very inconsistent across trials, with wide heterogeneity in the measures used. Standardised reporting of critically important outcomes is needed to strengthen the value of trials for decision-making.

Children and adolescents infected with severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) are predominantly asymptomatic or have mild symptoms compared with the more severe coronavirus disease 2019 (COVID-19) described in adults. However, SARS-CoV-2 is also associated with a widely reported but poorly understood paediatric systemic vasculitis. This multisystem inflammatory syndrome in children (MIS-C) has features that overlap with myocarditis, toxic-shock syndrome and Kawasaki disease. Current evidence indicates that MIS-C is the result of an exaggerated innate and adaptive immune response, characterized by a cytokine storm, and that it is triggered by prior SARS-CoV-2 exposure. Epidemiological, clinical and immunological differences classify MIS-C as being distinct from Kawasaki disease. Differences include the age range, and the geographical and ethnic distribution of patients. MIS-C is associated with prominent gastrointestinal and cardiovascular system involvement, admission to intensive care unit, neutrophilia, lymphopenia, high levels of IFNγ and low counts of naive CD4+ T cells, with a high proportion of activated memory T cells. Further investigation of MIS-C will continue to enhance our understanding of similar conditions associated with a cytokine storm., In this timely Review, the authors compare and contrast two forms of childhood inflammatory vasculitis: Kawasaki disease and the coronavirus disease 2019 (COVID-19)-associated multisystem inflammatory syndrome in children, highlighting epidemiological, clinical and immunological differences that suggest they should be classified as distinct syndromes., Key points Multisystem inflammatory syndrome in children (MIS-C) is characterized by exaggerated innate and adaptive immune responses following infection with severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) in predisposed children.Clinical presentation of MIS-C involves multiple organ systems, with prominent involvement of the gastrointestinal and cardiovascular systems.The factors that trigger the development of MIS-C in children exposed to or infected with SARS-CoV-2 are not yet known.Results from epidemiological, clinical and immunological investigations have revealed that although MIS-C has phenotypic similarities to Kawasaki disease, they are different syndromes.The approach to treatment of MIS-C aims to mute the augmented inflammatory response.

Importance Australian- and New Zealand-based, uveitis-specialized ophthalmologists have produced recommendations for the management of juvenile idiopathic arthritis (JIA)-type chronic anterior uveitis. Background Historically, the visual prognosis of JIA-type chronic anterior uveitis has been poor. New medical advances are likely to improve outcomes, but recently published guidelines are tailored for ophthalmic care in Europe and the United States. Design This work involved a consensus survey and a panel meeting. Participants The Australian and New Zealand JIA-Uveitis Working Group (29 ophthalmologists) participated in the work. Methods The Delphi technique was used to achieve consensus. Main outcome measures This work yielded consensus statements. Results The Working Group achieved consensus around 18 statements related to clinical evaluation, use of topical and regional corticosteroids, use of systemic corticosteroid and non-corticosteroid immunomodulatory drugs, and management of secondary cataract and glaucoma in childhood JIA-type uveitis. Conclusions and relevance Recommendations of the Australian and New Zealand JIA-Uveitis Working Group provide current and regionally applicable advice for managing chronic anterior uveitis in children with JIA.

Aim This study aimed to describe the current management practices for Kawasaki disease (KD) in Australia and New Zealand. Methods We performed a secondary analysis on the Australian and New Zealand responses to a large international survey of clinicians' perspectives on KD diagnosis and management. Results There was general consensus among Australian and New Zealand clinicians regarding the indications for intravenous immunoglobulin and aspirin in the management of acute KD. There was less consensus on the dose of these agents, the definition and management of treatment-resistant KD and the approach to long-term thromboprophylaxis. Conclusion Most clinicians use intravenous immunoglobulin for primary treatment of KD. There is variation regarding other aspects of KD diagnosis and important management issues. Future studies should confirm whether this reported variation occurs in real-world practice and assess potential impacts on patient outcome.

Background Kikuchi-Fujimoto disease (KFD) or necrotizing histiocytic lymphadenitis, was described separately by both Kikuchi and Fujimoto in Japan in the early 1970’s. Despite its rarity in the pediatric population, it is an important differential in persistent lymphadenopathy. Familial cases of KFD in the literature are rare. Here we describe the first reported case of KFD in non-identical twin sisters. Case presentation Twin 1 presented with a 3-week history of worsening right-sided cervical lymphadenopathy, daily fevers, significant lethargy, weight loss and arthralgia of her knees and ankles at the age of 12 years in 2015. She had had an unremarkable medical history. A biopsy of her lymph nodes showed histiocytic necrosis consistent with KFD. Twin 2 presented with a three-week history of lethargy, fatigue, weight loss and left-sided posterior cervical chain lymphadenopathy at 16 years of age in 2018. She had a history of frequently relapsing nephrotic syndrome and celiac disease. A biopsy of her lymph nodes was undertaken and showed histiocytic necrosis consistent with KFD. Conclusions KFD is a rare but self-limiting pathological process of necrotizing histiocytic lymphadenitis. Although further research is needed, there is an increasing amount of evidence which suggests a multifactorial pathological basis of disease. The two cases we document here are the first reported cases of familial KFD in dizygotic HLA-identical twins which reinforces the likely HLA-linkage in the etiology of KFD.

ObjectiveTo present Australia-wide data on paediatric COVID-19 and multisystem inflammatory syndromes to inform health service provision and vaccination prioritisation.DesignProspective, multicentre cohort study.SettingEight tertiary paediatric hospitals across six Australian states and territories in an established research surveillance network—Paediatric Active Enhanced Disease (PAEDS).ParticipantsAll children aged InterventionLaboratory-confirmed SARS-CoV-2 infection.Main outcomeIncidence of severe disease among children with COVID-19, PIMS-TS and KD-TS. We also compared KD epidemiology before and during the COVID-19 pandemic.ResultsAmong 386 children with SARS-CoV-2 infection, 381 (98.7%) had COVID-19 (median 6.3 years (IQR 2.1–12.8),53.3% male) and 5 (1.3%) had multisystem inflammatory syndromes (PIMS-TS, n=4; KD-TS, n=1) (median 7.9 years (IQR 7.8–9.8)). Most children with COVID-19 (n=278; 73%) were Australian-born from jurisdictions with highest community transmission. Comorbidities were present in 72 (18.9%); cardiac and respiratory comorbidities were most common (n=32/72;44%). 37 (9.7%) children with COVID-19 were hospitalised, and two (0.5%) required intensive care. Postinfective inflammatory syndromes (PIMS-TS/KD-TS) were uncommon (n=5; 1.3%), all were hospitalised and three (3/5; 60%) required intensive care management. All children recovered and there were no deaths. KD incidence remained stable during the pandemic compared with prepandemic.ConclusionsMost children with COVID-19 had mild disease. Severe disease was less frequent than reported in high prevalence settings. Preventative strategies, such as vaccination, including children and adolescents, could reduce both the acute and postinfective manifestations of the disease.

AIM The incidence of Kawasaki disease (KD) is reported to be increasing in some populations. We sought to describe long-term trends in the incidence and epidemiology of KD in Australia over 25 years. METHODS Two nationally complete administrative datasets relevant to KD in Australia were analysed and compared. The Australian Red Cross Lifeblood Supply Tracking Analysis Reporting System (STARS) recorded all doses of immunoglobulin (IVIG) approved in Australia between January 2007 and June 2016. The Australian Institute of Health and Welfare National Hospital Morbidity Database (NHMD) records all episodes of care in hospitals across Australia. Data relevant to KD were extracted an analysed, with comparisons made for the period of data overlap. RESULTS During the period of data overlap (2007-2015) the IVIG treatment rate in the 0- to 4-year age group (calculated from STARS) was 14.31 per 100 000 person-years (95% confidence interval 13.67-14.97). The hospitalisation rate in the same age group (calculated from the NHMD) was 14.99 per 100 000 person-years (95% confidence interval 14.33-15.66). Hospitalisation rates rose at an average rate of 3.54% annually over the 25 years to 2017 in the 0- to 4-year age group, almost exclusively in the 1- to 4-year age group. CONCLUSIONS There is evidence of increasing KD diagnosis in Australia. Similar trends have also been reported in Asia but not in North America or Europe. Increasing diagnosis may reflect a true increase in disease incidence, increasing recognition or overdiagnosis. Further research is needed to determine the cause for these trends.

Objectives The aim of this study is to investigate the effect of customized preformed foot orthoses on pain, quality of life, swollen and tender lower joints and foot and ankle disability in children with JIA. Methods Parallel group design. Children diagnosed with JIA were recruited from the three children’s hospitals in New South Wales, Australia. Participants were randomly assigned to a control group receiving a standard flat innersole (sham) with no corrective modifications. The trial group were prescribed a preformed device that was customized based on biomechanical assessments. Pain was the primary outcome and was followed up to 12 months post intervention. Secondary outcomes include quality of life, foot and ankle disability and swollen and tender joints. A linear mixed model was used to assess the impact of the intervention at each time point. Results Sixty-six participants were recruited. Child-reported pain was reduced statistically and clinically significant at 4 weeks and 3 months post intervention in favour of the trial group. Statistical significance was not reached at 6 and 12-month follow-ups. Quality of life and foot and ankle disability were not statistically significant at any follow-up; however, tender midfoot and ankle joints were significantly reduced 6 months post intervention. Conclusion Results of this clinical trial indicate customized preformed foot orthoses can be effective in reducing pain and tender joints in children with JIA exhibiting foot and ankle symptoms. Long-term efficacy of foot orthoses remains unclear. Overall, the trial intervention was safe, inexpensive and well tolerated by paediatric patients. Trial registration Australian New Zealand Clinical Trials Registry (ANZCTR): 12616001082493.

Children with juvenile idiopathic arthritis (JIA) can experience significant physical impairment of the lower extremity. Prolonged joint disease and symptoms may cause gait alterations such as reduced walking speed and increased plantar pressures in diseased areas of their feet. There is limited robust clinical trials investigating the effect of non-invasive mechanical therapies such as foot orthoses (FOs) on improving gait parameters in children with JIA.Are customised preformed FOs effective in improving gait parameters in children with JIA?A multicentre, parallel design, single-blinded randomised clinical trial was used to assess the gait impacts of customised preformed FOs on children with JIA. Children with a diagnosis of JIA, exhibiting lower limb symptoms and aged 5-18 were eligible. The trial group received a low-density full length, Slimflex Simple device which was customised chair side and the control group received a sham device. Peak pressure and pressure time integrals were used as the main gait outcomes and were measured using portable Tekscan gait analysis technology at baseline, 3 and 6 months. Differences at each follow-up were assessed using the Wilcoxon rank sum test.66 participants were recruited. Customised preformed FOs were effective in altering plantar pressures in children with JIA versus a control device. Reductions of peak pressures and pressure time integrals in the heel, forefoot and 5th metatarsophalangeal joint were statistically significant in favour of the trial group. This was associated with statistically significant increased midfoot contact with the trial device at baseline, 3 and 6-month data collections. The trial intervention was safe and well accepted by participants, which is reflected in the high retention rate (92%).Clinicians may prescribe customised preformed FOs in children with JIA to deflect pressure from painful joints and redistribute from high pressure areas such as the rearfoot and forefoot.

Background Juvenile idiopathic arthritis (JIA) management aims to promote remission through timely, individualized, well-coordinated interdisciplinary care using a range of pharmacological, physical, psychological, and educational interventions. However, achieving this goal is workforce-intensive. Harnessing the burgeoning eHealth and mobile health (mHealth) interventions could be a resource-efficient way of supplementing JIA management. Objective This systematic review aims to identify the eHealth and mHealth interventions that have been proven to be effective in supporting health outcomes for children and young people (aged 1-18 years) living with JIA. Methods We systematically searched 15 databases (2018-2021). Studies were eligible if they considered children and young people (aged 1-18 years) diagnosed with JIA, an eHealth or mHealth intervention, any comparator, and health outcomes related to the used interventions. Independently, 2 reviewers screened the studies for inclusion and appraised the study quality using the Downs and Black (modified) checklist. Study outcomes were summarized using a narrative, descriptive method and, where possible, combined for a meta-analysis using a random-effects model. Results Of the 301 studies identified in the search strategy, 15 (5%) fair-to-good–quality studies met the inclusion criteria, which identified 10 interventions for JIA (age 4-18.6 years). Of these 10 interventions, 5 (50%) supported symptom monitoring by capturing real-time data using health applications, electronic diaries, or web-based portals to monitor pain or health-related quality of life (HRQoL). Within individual studies, a preference was demonstrated for real-time pain monitoring over recall pain assessments because of a peak-end effect, improved time efficiency (P=.002), and meeting children’s and young people’s HRQoL needs (P Conclusions Evidence that supports the inclusion of eHealth and mHealth interventions in JIA management is increasing. However, this evidence needs to be considered cautiously because of the small sample size, wide CIs, and moderate to high statistical heterogeneity. More rigorous research is needed on the longitudinal effects of real-time monitoring, web-based pediatric rheumatologist–children and young people interactions, the comparison among different self-management programs, and the use of wearable technologies as an objective measurement for monitoring physical activity before any recommendations that inform current practice can be given.

BACKGROUND Juvenile idiopathic arthritis (JIA) management aims to promote remission through timely, individualized, well-coordinated interdisciplinary care using a range of pharmacological, physical, psychological, and educational interventions. However, achieving this goal is workforce-intensive. Harnessing the burgeoning eHealth and mobile health (mHealth) interventions could be a resource-efficient way of supplementing JIA management. OBJECTIVE This systematic review aims to identify the eHealth and mHealth interventions that have been proven to be effective in supporting health outcomes for children and young people (aged 1-18 years) living with JIA. METHODS We systematically searched 15 databases (2018-2021). Studies were eligible if they considered children and young people (aged 1-18 years) diagnosed with JIA, an eHealth or mHealth intervention, any comparator, and health outcomes related to the used interventions. Independently, 2 reviewers screened the studies for inclusion and appraised the study quality using the Downs and Black (modified) checklist. Study outcomes were summarized using a narrative, descriptive method and, where possible, combined for a meta-analysis using a random-effects model. RESULTS Of the 301 studies identified in the search strategy, 15 (5%) fair-to-good–quality studies met the inclusion criteria, which identified 10 interventions for JIA (age 4-18.6 years). Of these 10 interventions, 5 (50%) supported symptom monitoring by capturing real-time data using health applications, electronic diaries, or web-based portals to monitor pain or health-related quality of life (HRQoL). Within individual studies, a preference was demonstrated for real-time pain monitoring over recall pain assessments because of a peak-end effect, improved time efficiency (P=.002), and meeting children’s and young people’s HRQoL needs (PI2=79%; P=.04). The final 30% (3/10) of interventions supported self-management development through web-based programs, or apps, facilitating a small effect, reducing pain intensity (SMD −0.14, 95% CI −0.43 to 0.15; I2=53%; P=.33), and increasing disease knowledge and self-efficacy (SMD 0.30, 95% CI 0.03-0.56; I2=74%; P=.03). These results were not statistically significant. No effect was seen regarding pain interference, HRQoL, anxiety, depression, pain coping, disease activity, functional ability, or treatment adherence. CONCLUSIONS Evidence that supports the inclusion of eHealth and mHealth interventions in JIA management is increasing. However, this evidence needs to be considered cautiously because of the small sample size, wide CIs, and moderate to high statistical heterogeneity. More rigorous research is needed on the longitudinal effects of real-time monitoring, web-based pediatric rheumatologist–children and young people interactions, the comparison among different self-management programs, and the use of wearable technologies as an objective measurement for monitoring physical activity before any recommendations that inform current practice can be given.

ObjectivesTo describe the experiences, priorities, and needs of patients with rheumatic disease and their parents during transition from paediatric to adult healthcare.SettingFace-to-face and telephone semistructured interviews were conducted from December 2018 to September 2019 recruited from five hospital centres in Australia.ParticipantsFourteen young people and 16 parents were interviewed. Young people were included if they were English speaking, aged 14–25 years, diagnosed with an inflammatory rheumatic disease (eg, juvenile idiopathic arthritis, juvenile dermatomyositis, systemic lupus erythematosus, panniculitis, familial Mediterranean fever) before 18 years of age. Young people were not included if they were diagnosed in the adult setting.ResultsWe identified four themes with respective subthemes: avoid repeat of past disruption (maintain disease stability, preserve adjusted personal goals, protect social inclusion); encounter a daunting adult environment (serious and sombre mood, discredited and isolated identity, fear of a rigid system); establish therapeutic alliances with adult rheumatology providers (relinquish a trusting relationship, seek person-focused care, redefine personal–professional boundaries, reassurance of alternative medical supports, transferred trust to adult doctor) and negotiate patient autonomy (confidence in formerly gained independence, alleviate burden on patients, mediate parental anxiety).ConclusionsDuring transition, patients want to maintain disease stability, develop a relationship with their adult provider centralised on personal goals and access support networks. Strategies to comprehensively communicate information between providers, support self-management, and negotiate individualised goals for independence during transition planning may improve satisfaction, and health and treatment outcomes.

Background: Juvenile idiopathic arthritis (JIA) is the most common rheumatic disease in children, with lower limb involvement highly prevalent. Recent evidence has highlighted the lack of specific lower limb physical examination (PE) tools for clinicians assisting the paediatric rheumatology team in identifying lower extremity disease in patients with JIA. Early clinical detection may lead to more prompt and targeted interventions to reduce lower limb problems in children with JIA. The aim of this pilot study is to provide preliminary data on the diagnostic accuracy of a lower limb PE tool in JIA. Methods: Children with JIA requiring magnetic resonance imaging (MRI) on their lower limb joints per their usual care were eligible. Lower limb joint counts were conducted clinically by a podiatrist and paediatric rheumatologist using the proposed twenty joint per side, PE tool. The PE were compared to MRI assessments completed by two independent paediatric radiologists. Data were analysed using agreement (observed, positive and negative) and Cohen’s kappa with 95% CIs. Results: Fifteen participants were recruited into the study in which 600 lower limb joints were clinically examined. Statistical analysis showed excellent inter-rater reliability between podiatrist and paediatric rheumatologist for both joint swelling and tenderness. Results of the intra-rater reliability of the podiatrist using the PE tool indicated excellent percentage agreements (98.5–100%) and substantial kappa coefficients (0.93–1). The inter-rater reliability between radiological assessments contrasted the PE results, showing low agreement and poor reliability. Comparisons between PE and MRI resulted in poor kappa coefficients and low agreement percentages. The most agreeable joint between MRI and PE was the ankle joint, while the worst performing joint was the sub-talar joint. Conclusion: Results indicate potential clinical reliability; however, the validity and diagnostic accuracy of the proposed PE tool remains unclear due to low kappa coefficients and inconsistent agreements between PE and MRI results. Further research will be required before the tool may be used in a clinical setting.

Background Considering the changing landscape of internet use and rising ownership of digital technology by young people, new methods could be considered to improve the current model of juvenile idiopathic arthritis (JIA) management. Objective This systematic review aims to evaluate the usability of eHealth and mobile health (mHealth) interventions currently available for young people living with JIA. Methods The PRISMA (Preferred Reporting Items for Systematic Reviews and Meta-Analyses) guidelines were used to oversee this review. We systematically searched 15 databases for 252 potential studies; 2 authors independently screened all quantitative studies reporting the use of eHealth and mHealth interventions for young people (aged 1-18 years) diagnosed with JIA. Studies were excluded if they did not report outcome measures or were reviews, commentaries, or qualitative studies. Study methodological quality was scored using the Down and Black (modified) checklist. A narrative descriptive methodology was used to quantify the data because of heterogeneity across the studies. Results A total of 11 studies were included in this review, reporting 7 eHealth and mHealth interventions for young people (aged 4-18 years) living with JIA, targeting health issues such as pain, health-related quality of life, physical activity, and chronic disease self-management. The usability of the interventions was facilitated through training and ongoing support. The engagement was promoted by a combination of persuasive influences, and barriers preventing adherence were removed through personal reminders and flexible program schedules to cater to JIA and non-JIA illnesses or other commonly seen activities in childhood. The feedback obtained was that most young people and their parents liked the interventions. Conclusions The results of this review need to be considered cautiously because of the lack of rigorous testing and heterogeneity, which limits the detailed descriptions of data synthesis. Further research is needed to consider gender differences, associated costs, and the effectiveness of the interventions on health outcomes to better support young people living with JIA.

Aim To describe the contemporary experience of Kawasaki Disease (KD) in a tertiary paediatric hospital. Methods This was a retrospective analysis of admissions with suspected/confirmed KD to John Hunter Children’s Hospital from 1 January 2015 to 31 December 2016, with follow-up. Patients were stratified into classical, incomplete, unlikely and uncertain KD cohorts based on the 2004 American Heart Association (AHA) guidelines. Results Forty (40) patients had 45 admissions with suspected KD. Twenty-four (24) patients (60%) had complete and incomplete KD. Twenty-nine per cent (29%) (7/24) were not diagnosed at presentation. Thirty per cent (30%) (12/40) were incompletely assessed according to the AHA pathway. Seventy-one per cent (71%) of KD patients (17/24) were treated with aspirin and intravenous immunoglobulin. The incidence was 22–26 per 100,000 in children less than 5 years during the study period, higher compared to prior data. Conclusion The diagnosis of KD remains challenging, with some children not diagnosed at the time of initial presentation. The increase in KD incidence is hypothesis generating and requires further investigation.

We provide an update on the state of play with regards a newly described inflammatory condition which has arisen during the current SARS‐CoV‐2 pandemic. The condition has been named paediatric inflammatory multisystem syndrome temporally associated with SARS‐CoV‐2 or multisystem inflammatory syndrome in children. This condition has shown significant similarities to Kawasaki disease and toxic shock syndrome.

BackgroundChronic conditions are the leading cause of mortality, morbidity and disability in children. However, children and caregivers are rarely involved in identifying research priorities, which may limit the value of research in supporting patient-centred practice and policy.ObjectiveTo identify priorities of patients, caregivers and health professionals for research in childhood chronic conditions and describe the reason for their choices.SettingAn Australian paediatric hospital and health consumer organisations.MethodsRecruited participants (n=73) included patients aged 8 to 14 years with a chronic condition (n=3), parents/caregivers of children aged 0 to 18 years with a chronic condition (n=19), representatives from consumer organisations (n=13) and health professionals including clinicians, researches (n=38) identified and discussed research priorities. Transcripts were thematically analysed.ResultsSeventy-eight research questions were identified. Five themes underpinned participants’ priorities: maintaining a sense of normality (enabling participation in school, supporting social functioning, promoting understanding and acceptance), empowering self-management and partnership in care (overcoming communication barriers, gaining knowledge and skills, motivation for treatment adherence, making informed decisions, access and understanding of complementary and alternative therapies),strengthening ability to cope (learning to have a positive outlook, preparing for home care management, transitioning to adult services), broadening focus to family (supporting sibling well-being, parental resilience and financial loss, alleviating caregiver burden), and improving quality and scope of health and social care (readdressing variability and inequities, preventing disease complications and treatment side effects, identifying risk factors, improving long-term outcomes, harnessing technology, integrating multidisciplinary services).ConclusionResearch priorities identified by children, caregivers and health professionals emphasise a focus on life participation, psychosocial well-being, impact on family and quality of care. These priorities may be used by funding and policy organisations in establishing a paediatric research agenda.

There is accumulating evidence that patients with functional neurological symptom disorder (FND) show activation of multiple components of the stress system—the hypothalamic–pituitary–adrenal axis, autonomic nervous system, and brain regions involved in arousal- and emotion-processing. This study aims to examine whether the immune-inflammatory component of the stress system is also activated. C-reactive protein (CRP) blood titre levels were measured in 79 children and adolescents with FND. CRP values ≥ 2 mg/L suggest low-grade inflammation. CRP values > 10 mg/L suggest a disease process. Sixty-six percent of subjects (n = 52) had CRP titres ≥ 2 mg/L. The upward shift in the distribution of CRP levels suggested low-grade inflammation (median CRP concentration was 4.60 mg/L, with 75th and 90th percentiles of 6.1 and 10.3 mg/L, respectively). Elevated CRP titres were not explained by sex, pubertal status, BMI, or medical factors. Confounder analyses suggested that history of maltreatment (χ2 = 2.802, df = 1, p = 0.094, φ = 0.190; β = 2.823, p = 0.04) and a diagnosis of anxiety (χ2 = 2.731, df = 1, p = 0.098, φ = 0.187; β = 4.520, p = 0.061) contributed to elevated CRP levels. Future research will need to identify the origins and locations of immune cell activation and the pathways and systems contributing to their activation and modulation. Because functional activity in neurons and glial cells—the brain’s innate effector immune cells—is tightly coupled, our finding of elevated CRP titres suggests activation of the immune-inflammatory component of the brain’s stress system. A more direct examination of inflammation-related molecules in the brain will help clarify the role of immune-inflammatory processes in FND.

Intravenous immunoglobulin (IVIG) reduces coronary aneurysms in patients with Kawasaki disease (KD), but additional management options remain challenging, with no generalisable evidence-based recommendations. We performed a survey of 724 physicians from 73 countries to assess variation in practice. IVIG was the preferred initial treatment by 659 (91%) of respondents. Criteria for adjunctive primary treatment varied considerably and definitions of IVIG resistance varied markedly by geographical continent, Human Development Index tiers and medical specialty. A second dose of IVIG was used most often for patients with coronary aneurysm non-responsive to initial treatment (572, 79%), but corticosteroids (379, 52%) and tumour necrosis factor alpha inhibitors (208, 29%) were also frequently used. Our findings highlight the need for international collaborative efforts to optimise management of patients with KD worldwide.

Objective Patients with systemic sclerosis (SSc) experience severe physical limitations and psychological morbidity, but their lived experience remains underrepresented and is reflected in the scarcity of evidence-based patient-centered interventions. We aimed to describe patients' perspectives of SSc to inform strategies to improve their care. Methods Face-to-face semistructured interviews were conducted with 30 adult patients with limited cutaneous or diffuse cutaneous SSc in Australia. Transcripts were thematically analyzed using HyperRESEARCH software. Results Six themes were identified: bodily malfunction (restrictive pain, debilitating physical changes, pervasive exhaustion), deprivation of social function (loss of work and career, social isolation, threat to traditional roles, loss of intimacy), disintegration of identity (stigmatizing physical changes, disassociated self-image, extinguished hopes, alone and powerless, invisibility of illness), insecurity of care (unrecognized disease, ambiguity around diagnosis and cause, information insufficiency, resigning to treatment limitations, seeking reassurance, fear of progression), avoiding the sick role (evading thoughts of sickness, protecting family, favorable comparison), and perseverance and hope (positive stoicism, optimism about treatment and monitoring, taking control of own health, pursuing alternative treatments, transcending illness through support). Conclusion SSc inflicts major bodily and social restrictions that crush patients' identity and self-image. Uncertainties about the cause, diagnosis, and prognosis can undermine confidence in care, leading to anxiety and therapeutic nihilism. Access to psychosocial care to support the patients' role and functioning capacity, as well as communication and education that explicitly address their concerns regarding management may potentially improve treatment satisfaction, self-efficacy, adherence, and outcomes in patients with SSc.

Background Intravenous (IV) cyclophosphamide has been first-line treatment for inducing disease remission in lupus nephritis. The comparative efficacy and toxicity of newer agents such as mycophenolate mofetil (MMF) and calcineurin inhibitors are uncertain. Study Design Network meta-analysis. Setting & Population Patients with proliferative lupus nephritis. Selection Criteria for Studies Randomized trials of immunosuppression to induce or maintain disease remission. Interventions IV cyclophosphamide, oral cyclophosphamide, MMF, calcineurin inhibitor, plasma exchange, rituximab, or azathioprine, alone or in combination. Outcomes Complete remission, end-stage kidney disease, all-cause mortality, doubling of serum creatinine level, relapse, and adverse events. Results 53 studies involving 4,222 participants were eligible. Induction and maintenance treatments were administered for 12 (IQR, 6-84) and 25 (IQR, 12-48) months, respectively. There was no evidence of different effects between therapies on all-cause mortality, doubling of serum creatinine level, or end-stage kidney disease. Compared to IV cyclophosphamide, the most effective treatments to induce remission in moderate- to high-quality evidence were combined MMF and calcineurin inhibitor therapy, calcineurin inhibitors, and MMF (ORs were 2.69 [95% CI, 1.74-4.16], 1.86 [95% CI, 1.05-3.30], and 1.54 [95% CI, 1.04-2.30], respectively). MMF was significantly less likely than IV cyclophosphamide to cause alopecia (OR, 0.21; 95% CI, 0.12-0.36), and MMF combined with calcineurin inhibitor therapy was less likely to cause ovarian failure (OR, 0.25; 95% CI, 0.07-0.93). Regimens generally had similar odds of major infection. MMF was the most effective strategy to maintain remission. Limitations Outcome definitions not standardized, short duration of follow-up, and possible confounding by previous or subsequent therapy. Conclusions Evidence for induction therapy for lupus nephritis is inconclusive based on treatment effects on all-cause mortality, doubling of serum creatinine level, and end-stage kidney disease. MMF, calcineurin inhibitors, or their combination were most effective for inducing remission compared to IV cyclophosphamide, while conferring similar or lower treatment toxicity. MMF was the most effective maintenance therapy.

Paediatric Respiratory Reviews - In Press.Proof corrected by the author Available online since vendredi 15 juillet 2016

Background Children and adolescents with Juvenile Idiopathic Arthritis (JIA) typically have reduced physical activity level and impaired aerobic and anaerobic exercise capacity when compared to their non-JIA counterparts. Low intensity exercise regimens appear to be safe in children with JIA and may results in improvements in overall physical function. Poor adherence to paediatric rheumatology treatment may lead to negative clinical outcomes and possibly increased disease activity. This includes symptoms such as pain, fatigue, quality of life, longer term outcomes including joint damage, as well as increase of healthcare associated costs. Low adherence to medications such as methotrexate and biological-drugs remains a significant issue for paediatric rheumatologists, with alarming reports that less than half of the children with JIA are compliant to drug-therapy. Main body The recent advances in interactive technology resulting in a variety of wearable user-friendly smart devices may become a key solution to address important questions in JIA clinical management. Fully understanding the impact that arthritis and treatment complications have upon individual children and their families has long been a challenge for clinicians. Modern interactive technologies can be customised and accessed directly in the hands or wrists of children with JIA. These secured networks could be accessible ‘live’ at anytime and anywhere by the child, parents and clinicians. Multidisciplinary teams in paediatric rheumatology may benefit from adopting these technologies to better understand domains such as patient biological parameters, symptoms progression, adherence to drug-therapy, quality of life, and participation in physical activities. Most importantly the use of smart devices technologies may also facilitate more timely clinical decisions, improve self-management and parents awareness in the progression of their child’s disease. Paediatric rheumatology research could also benefit from the use of these smart devices, as they would allow real-time access to meaningful data to thoroughly understand the disease-patterns of JIA, such as pain and physical activity outcomes. Data collection that typically occurs once every 1 or 3 months in the clinical setting could instead be gathered every week, day, minute or virtually live online. Arguably, few limitations in wearing such interactive technologies still exist and require further developments. Conclusion Finally, by embracing and adapting these new and now highly accessible interactive technologies, clinical management and research in paediatric rheumatology may be greatly advanced.

Objective.Managing juvenile-onset systemic lupus erythematosus (SLE) is particularly challenging. The disease may be severe, adolescent patients have complex medical and psychosocial needs, and patients must navigate the transition to adult services. To inform patient-centered care, we aimed to identify the healthcare and research priorities of young patients with SLE and describe the reasons underpinning their priorities.Methods.Face-to-face, semistructured interviews and focus groups were conducted with patients with SLE, aged from 14 to 26 years, from 5 centers in Australia. For each of the 5 allocation exercises, participants allocated 10 votes to (1) research topics; research questions on (2) medical management, (3) prevention and diagnosis, (4) lifestyle and psychosocial; and (5) healthcare specialties, and discussed the reasons for their choices. Descriptive statistics were calculated for votes and qualitative data were analyzed thematically.Results.The 26 participants prioritized research that alleviated the psychological burden of SLE. They allocated their votes toward medical and mental health specialties in the management of SLE, while fewer votes were given to physiotherapy/occupational therapy and dietetics. The following 7 themes underpinned the participants’ priorities: improving service shortfalls, strengthening well-being, ensuring cost efficiency, minimizing family/community burden, severity of comorbidity or complications, reducing lifestyle disruption, and fulfilling future goals.Conclusion.Young patients with SLE value comprehensive care with greater coordination among specialties. They prioritized research focused on alleviating poor psychological outcomes. The healthcare and research agenda for patients with SLE should include everyone involved, to ensure that the agenda aligns with patient priorities, needs, and values.

Objectives We aimed to describe patients’ attitudes and experiences of transition from paediatric to adult healthcare in rheumatology to inform patient-centred transitional care programmes. Methods We searched MEDLINE, EMBASE, PsycINFO and CINAHL to August 2019 and used thematic synthesis to analyse the findings. Results From 26 studies involving 451 people with juvenile-onset rheumatic conditions we identified six themes: a sense of belonging (comfort in familiarity, connectedness in shared experiences, reassurance in being with others of a similar age, desire for normality and acceptance); preparedness for sudden changes (confidence through guided introductions to the adult environment, rapport from continuity of care, security in a reliable point of contact, minimizing lifestyle disruptions); abandonment and fear of the unknown (abrupt and forced independence, ill-equipped to hand over medical information, shocked by meeting adults with visible damage and disability, vulnerability in the loss of privacy); anonymous and dismissed in adult care (deprived of human focus, sterile and uninviting environment, disregard of debilitating pain and fatigue); quest for autonomy (controlled and patronized in the paediatric environment, liberated from the authority of others, freedom to communicate openly); and tensions in parental involvement (overshadowed by parental presence, guilt of excluding parents, reluctant withdrawal of parental support). Conclusion Young people feel dismissed, abandoned, ill-prepared and out of control during transition. However, successful transition can be supported by preparing for changes, creating a sense of belonging and negotiating parental involvement and autonomy. Incorporating patient-identified priorities into transitional services may improve satisfaction and outcomes in young people with juvenile-onset rheumatic conditions.

Background Patients with coronary artery aneurysms (CAA) after Kawasaki disease (KD) are at risk of thrombosis, which can lead to myocardial infarction or sudden death. Clinical practice guidelines recommend anticoagulation for high-risk patients. Methods Web-based worldwide survey of physicians completed between 2016 and 2017 investigating anti-thrombotic management after KD. We compared management of patients by geographic location, Human Development Index (HDI) tier, and medical specialty. Results The survey was completed by 603 physicians from 63 countries. In patients with normal coronaries, 95 (25%) of physicians recommended low-dose aspirin during long-term follow-up (>3 months after diagnosis). In patients with non-giant CAA, dual antiplatelet (e.g. aspirin and clopidogrel) was used by 121 (32%) of physicians, and anticoagulation by 72 (19%) of physicians. In patients with giant CAA, dual antiplatelet was used by 39 (10%) of physicians and anticoagulation by 285 (74%). In multivariable analysis, cardiology (OR 6.4 [95% CI 2.7, 16.1]) and rheumatology (OR 4.3 [95% CI 1.6, 12.6]) specialty (versus general pediatrics) were the only independent predictors of anticoagulant use in patients with giant CAA. Conclusion There is significant variation in anti-thrombosis management of patients with CAA after KD, with 26% of physicians not recommending anticoagulation of patients with giant CAA. Further studies are needed to evaluate the drivers of this practice variation to inform educational initiatives and to ascertain impact on long-term outcomes.

BACKGROUND Considering the changing landscape of internet use and rising ownership of digital technology by young people, new methods could be considered to improve the current model of juvenile idiopathic arthritis (JIA) management. OBJECTIVE This systematic review aims to evaluate the usability of eHealth and mobile health (mHealth) interventions currently available for young people living with JIA. METHODS The PRISMA (Preferred Reporting Items for Systematic Reviews and Meta-Analyses) guidelines were used to oversee this review. We systematically searched 15 databases for 252 potential studies; 2 authors independently screened all quantitative studies reporting the use of eHealth and mHealth interventions for young people (aged 1-18 years) diagnosed with JIA. Studies were excluded if they did not report outcome measures or were reviews, commentaries, or qualitative studies. Study methodological quality was scored using the Down and Black (modified) checklist. A narrative descriptive methodology was used to quantify the data because of heterogeneity across the studies. RESULTS A total of 11 studies were included in this review, reporting 7 eHealth and mHealth interventions for young people (aged 4-18 years) living with JIA, targeting health issues such as pain, health-related quality of life, physical activity, and chronic disease self-management. The usability of the interventions was facilitated through training and ongoing support. The engagement was promoted by a combination of persuasive influences, and barriers preventing adherence were removed through personal reminders and flexible program schedules to cater to JIA and non-JIA illnesses or other commonly seen activities in childhood. The feedback obtained was that most young people and their parents liked the interventions. CONCLUSIONS The results of this review need to be considered cautiously because of the lack of rigorous testing and heterogeneity, which limits the detailed descriptions of data synthesis. Further research is needed to consider gender differences, associated costs, and the effectiveness of the interventions on health outcomes to better support young people living with JIA.

IMPORTANCE Paediatric uveitis is a severe sight-threatening uveitis due to disease progression and treatment failure. Biological agents are a promising new treatment. This study provides real-world data on their use from Sydney, Australia. BACKGROUND Traditionally corticosteroids and non-biological immunosuppressive agents were used to treat paediatric uveitis, often with poor outcomes. DESIGN Retrospective, chart review over an 8-year period at a tertiary referral eye hospital. PARTICIPANTS A total of 27 paediatric uveitis patients treated with biological agents. METHODS Chart review of demographic data and treatment outcomes. MAIN OUTCOME MEASURES Treatment efficacy (corticosteroid-sparing effect, topical steroid cessation/reduction, reduction in systemic-steroid sparing agents, change in intraocular inflammation, visual acuity and central macular thickness); treatment failure; and adverse events. Data were collected at biological initiation, 6 weeks, 6 months and 12 months. RESULTS Biological therapy over 1 year was effective with prednisolone dose reduced to

AIM To define the clinical characteristics, investigations, management and outcomes of lymphoedema in a paediatric cohort. METHODS A retrospective chart review of children with lymphoedema seen at two tertiary paediatric hospitals since 1998. Telephone interviews with parents were performed when information was missing. Information recorded included demographic data, features of diagnosis and clinical presentation, symptoms, complications and treatment. RESULTS A total of 86 patients with lymphoedema were identified. Eighty cases (93%) were primary and six cases (7%) were secondary. Most were female (60%). Location of swelling was most commonly the lower limbs (94%). There were 13 cases (15%) of genital involvement. Swelling presented in the first 12 months of life in 60% of primary lymphoedema patients. Complications of lymphoedema occurred in 73% of patients. Lymphoscintigraphy was the most common investigation used (65%), followed by ultrasound (57%) and magnetic resonance imaging (MRI) (35%). Eight of the 48 (17%) lymphoscintigraphs produced a false negative result or were inconclusive with a correct diagnosis subsequently made clinically and using MRI. Average time to diagnosis was 9 months. Lymphoedema was managed with compression garments (99%), manual lymph drainage (97%) and multilayered bandaging (68%). Eight patients had an operative procedure as a part of management. CONCLUSIONS Primary lymphoedema is more common than secondary lymphoedema in children. Onset tends to be during infancy for both males and females, and the lower limb is typically involved. Causes of secondary lymphoedema are diverse and rare. Diagnosis in children is often delayed but is possible based on history and physical examination alone and when further investigation is necessary MRI is effective.

Aim Cryopyrin-associated periodic syndromes (CAPS) encapsulate three auto-inflammatory conditions, ranging in severity from mild (familial cold auto-inflammatory syndrome: FCAS), moderate (Muckle-Wells syndrome: MWS) and severe (neonatal onset multi-inflammatory disorder: NOMID). We aimed to describe the epidemiology, clinical features and outcomes of Australian children and adults with CAPS. Methods Patients were identified and clinical data collected through a questionnaire sent during 2012–2013 to clinicians reporting to the Australian Paediatric Surveillance Unit and subscribing to the Australasian Societies for Allergy/Immunology, Rheumatology and Dermatology. Results Eighteen cases of CAPS were identified (8 NOMID; 8 MWS, 2 FCAS); 12 in children

Recurrent fever is a common presentation in paediatric practice and can be caused by a wide variety of diseases including autoinflammatory conditions. The innate immune system plays an essential role in the 'first line' response to infection through mediation of inflammatory responses. Inflammasomes are part of the regulatory process for this system and result in the production of the powerful pro-inflammatory cytokine interleukin-1B. Dysregulation of inflammasomes, and Interleukin 1 production, contributes to the pathogenesis of autoinflammatory diseases. This review focuses on described periodic fever syndromes (PFS) which are now collectively referred to as autoinflammatory syndromes. Conditions discussed include periodic fever aphthous stomatitis pharyngitis and cervical adenopathy, familial Mediterranean fever, tumour necrosis factor receptor-associated periodic syndromes, hyperimmunoglobulinaemia D and the cryopyrin-associated periodic syndromes. Presenting features, complications, diagnostic and treatment approaches for these conditions are discussed. Nonetheless, as most of these conditions are rare and may have significant long-term complications, it is recommended that they be managed in consultations with a physician experienced in managing PFS.

Objective.Systemic sclerosis (SSc) is a chronic, progressive autoimmune disease with major end-organ involvement. Much attention has been focused on the management of physical and clinical manifestations; however, the effect of the disease and treatment on the patient’s identity, relationships, functioning, and mental well-being are less known. We aimed to describe the patients’ perspectives and experiences of living with SSc.Methods.Electronic databases were searched to October 2014. Thematic synthesis was used to analyze the findings.Results.We included 26 studies involving 463 patients. Six key themes were identified: distressing appearance transformation (disturbing facial changes, stigmatizing sickness, unrecognizable self), palpable physical limitations (bodily restrictions, frustrating mind-body disconnect, pervasive fatigue, disabling pain), social impairment (breaking intimacy, struggling to fulfill family responsibilities, maintaining work, losing independence), navigating uncertainty (diagnostic ambiguity, medically fending for oneself, unpredictable course of illness), alone and misunderstood (fearful avoidance of fellow patients, invisible suffering), and gradual acceptance and relative optimism (adapting to change and accepting limitations, taking a positive spin, cautious hoping, empowering relationships, valuing medical support).Conclusion.SSc is a rare and unpredictable illness that undermines patients’ sense of certainty and control and impairs their self-image, identity, and daily functioning. Patient-centered care that encompasses strategies to promote self-esteem, resilience, and self-efficacy may help to improve treatment satisfaction and health and quality of life outcomes for patients with SSc.

The lysosomal storage disorders are a collection of progressive, multisystem disorders that frequently present in childhood. Their timely diagnosis is paramount as they are becoming increasingly treatable. Musculoskeletal manifestations often occur early in the disease course, hence are useful as diagnostics clues. Non-inflammatory joint stiffness or pain, carpal tunnel syndrome, trigger fingers, unexplained pain crises and short stature should all prompt consideration of a lysosomal storage disorder. Recurrent ENT infections, hepatosplenomegaly, recurrent hernias and visual/hearing impairment - especially when clustered together - are important extra-skeletal features. As diagnostic and therapeutic options continue to evolve, children with lysosomal storage disorders and their families are facing more sophisticated options for screening and treatment. The aim of this article is to highlight the paediatric presentations of lysosomal storage disorders, with an emphasis on the musculoskeletal features.

Aim There have been no formal assessments of current levels of the pediatric rheumatology workforce in Australia and New Zealand. Despite this it is felt that we fall well behind international guidelines placing children and adolescents with rheumatic diseases at risk of suboptimal care. Overcoming this shortfall in specialist pediatric rheumatology care requires documentation and recognition of the shortfall and a commitment from the health system to support improvements to supplement the current specialist workforce. The purpose of this survey was to assess the current state of play of the pediatric rheumatology workforce in Australia and New Zealand. Methods The Australian Paediatric Rheumatology Group (APRG) conducted a survey, which examined the current pediatric rheumatology workforce in Australia and New Zealand. The survey was sent via email link to a survey hosted by Zoomerang™ to 49 physicians known to treat patients with pediatric rheumatic diseases and they were asked to forward the survey to any others who they knew saw children with rheumatic disease. Results Currently there is a shortfall in the pediatric rheumatology workforce of 68% based on minimum requirements and a shortfall of 225% based on an ideal scenario. Conclusion Currently in Australia and New Zealand we fail to provide the level of care to children with pediatric rheumatic diseases comparable to other developed health economies worldwide. The current deficiency requires an increase in resource allocation to clinical service and speciality training to overcome this disparity and ensure children in Australia and New Zealand receive internationally recognized standards of care.

Background Cyclophosphamide, in combination with corticosteroids, has been first-line treatment for inducing disease remission for proliferative lupus nephritis, reducing death at five years from over 50% in the 1950s and 1960s to less than 10% in recent years. Several treatment strategies designed to improve remission rates and minimise toxicity have become available. Treatments, including mycophenolate mofetil (MMF) and calcineurin inhibitors, alone and in combination, may have equivalent or improved rates of remission, lower toxicity (less alopecia and ovarian failure) and uncertain effects on death, end-stage kidney disease (ESKD) and infection. This is an update of a Cochrane review first published in 2004 and updated in 2012. Objectives Our objective was to assess the evidence and evaluate the benefits and harms of different immunosuppressive treatments in people with biopsy-proven lupus nephritis. The following questions relating to management of proliferative lupus nephritis were addressed: 1) Are new immunosuppressive agents superior to or as effective as cyclophosphamide plus corticosteroids? 2) Which agents, dosages, routes of administration and duration of therapy should be used? 3) Which toxicities occur with the different treatment regimens? Search methods We searched the Cochrane Kidney and Transplant Specialised Register up to 2 March 2018 with support from the Cochrane Information Specialist using search terms relevant to this review. Studies in the Specialised Register are identified through searches of CENTRAL, MEDLINE, and EMBASE, conference proceedings, the International Clinical Trials Register (ICTRP) Search Portal and ClinicalTrials.gov. Selection criteria Randomised controlled trials (RCTs) and quasi-RCTs comparing any immunosuppressive treatment for biopsy-proven class III, IV, V+III and V+VI lupus nephritis in adult or paediatric patients were included. Data collection and analysis Data were abstracted and the risks of bias were assessed independently by two authors. Dichotomous outcomes were calculated as risk ratio (RR) and measures on continuous scales calculated as mean differences (MD) with 95% confidence intervals (CI). The primary outcomes were death (all causes) and complete disease remission for induction therapy and disease relapse for maintenance therapy. Evidence certainty was determined using GRADE. Main results In this review update, 26 new studies were identified, to include 74 studies involving 5175 participants overall. Twenty-nine studies included children under the age of 18 years with lupus nephritis, however only two studies exclusively examined the treatment of lupus nephritis in patients less than 18 years of age. Induction therapy Sixty-seven studies (4791 participants; median 12 months duration (range 2.5 to 48 months)) reported induction therapy. The effects of all treatment strategies on death (all causes) and ESKD were uncertain (very low certainty evidence) as this outcome occurred very infrequently. Compared with intravenous (IV) cyclophosphamide, MMF may have increased complete disease remission (RR 1.17, 95% CI 0.97 to 1.42; low certainty evidence), although the range of effects includes the possibility of little or no difference. Compared to IV cyclophosphamide, MMF is probably associated with decreased alopecia (RR 0.29, 95% CI 0.19 to 0.46; 170 less (129 less to 194 less) per 1000 people) (moderate certainty evidence), increased diarrhoea (RR 2.42, 95% CI 1.64 to 3.58; 142 more (64 more to 257 more) per 1000 people) (moderate certainty evidence) and may have made little or no difference to major infection (RR 1.02, 95% CI 0.67 to 1.54; 2 less (38 less to 62 more) per 1000 people) (low certainty evidence). It is uncertain if MMF decreased ovarian failure compared to IV cyclophosphamide because the certainty of the evidence was very low (RR 0.36, 95% CI 0.06 to 2.18; 26 less (39 less to 49 more) per 1000 people). Studies were not generally designed to measure ESKD. MMF combined with tacrolimus may have increased complete disease remission (RR 2.38, 95% CI 1.07 to 5.30; 336 more (17 to 1048 more) per 1000 people (low certainty evidence) compared with IV cyclophosphamide, however the effects on alopecia, diarrhoea, ovarian failure, and major infection remain uncertain. Compared to standard of care, the effects of biologics on most outcomes were uncertain because of low to very low certainty of evidence. Maintenance therapy Nine studies (767 participants; median 30 months duration (range 6 to 63 months)) reported maintenance therapy. In maintenance therapy, disease relapse is probably increased with azathioprine compared with MMF (RR 1.75, 95% CI 1.20 to 2.55; 114 more (30 to 236 more) per 1000 people (moderate certainty evidence). Multiple other interventions were compared as maintenance therapy, but patient-outcome data were sparse leading to imprecise estimates. Authors' conclusions In this review update, studies assessing treatment for proliferative lupus nephritis were not designed to assess death (all causes) or ESKD. MMF may lead to increased complete disease remission compared with IV cyclophosphamide, with an acceptable adverse event profile, although evidence certainty was low and included the possibility of no difference. Calcineurin combined with lower dose MMF may improve induction of disease remission compared with IV cyclophosphamide, but the comparative safety profile of these therapies is uncertain. Azathioprine may increase disease relapse as maintenance therapy compared with MMF.

Background Dermatomyositis – an idiopathic inflammatory myopathy- can be resistant and unresponsive to initial treatments and require chronic therapy. Symptoms including muscle weakness and skin rash can lead to severe complications and impair quality of life. There are a paucity of randomised trials in dermatomyositis, which may not consistently report outcomes that are important to patients and their clinicians. Objectives To assess the scope and consistency of outcomes reported in randomised trials in dermatomyositis. Methods MEDLINE, Embase, CINAHL, PsycINFO were searched to February 2017 for randomised trials in children and adults with dermatomyositis. The frequency and characteristics of the outcome domains and measures reported were then analysed. Similar outcomes were grouped into outcome domains and classified as surrogate, clinical or patient-reported outcomes. The frequency and characteristics of the outcome domains and measures reported were then analysed. Results We included 18 trials (n=844 participants), reporting 680 outcome measures. These were grouped into 27 different outcome domains; including 12 (44%) clinical outcomes, 9 (33%) surrogate/biochemical outcomes, and 6 (22%) patient-reported outcomes. The 5 most frequently reported outcome domains were: physical function (13 trials, 72%), muscle strength (12, 67%), muscle inflammation (12, 67%), biomarkers (11, 61%), and composite i.e. myositis disease activity (10, 56%). Conclusions The majority of outcomes reported in trials in dermatomyositis are clinical outcomes and few are patient-reported. Establishing a core set of patient-important outcomes may improve the consistency and relevance of outcomes reported in trials in dermatomyositis to inform decision-making. Disclosure of Interest None declared

Objective. Interprofessional collaboration is a crucial component of care for children with rheumatic disease. Interprofessional care, when delivered appropriately, prevents disability and improves long-term prognosis in this vulnerable group. Methods. The aim of this survey was to explore allied health professionals’ and nurses’ confidence in treating paediatric rheumatology patients. Results. Overall, 117 participants were recruited, 77.9% of participants reported being “not confident at all,” “not confident,” or “neutral” in treating children with rheumatic diseases (RD) despite 65.1% of participants reporting having treated >1 paediatric rheumatology case in the past month. Furthermore, 67.2% of participants felt their undergraduate education in paediatric rheumatology was inadequate. “Journals” or “texts books” were used by 49.3% of participants as their primary source of continuing professional development (CPD) and 39.3% of participants indicated that they did not undertake any CPD related to paediatric rheumatology. Small group and online education were perceived to be potentially of “great benefit” for CPD. Conclusion. This paper highlights allied health professionals’ and nurses’ perceived inadequacy of their undergraduate education in paediatric RD and their low confidence in recognising and treating RD. Undergraduate and postgraduate education opportunities focusing on interprofessional collaboration should be developed to address this workforce deficiency.