Paediatric Rheumatology European Society Research Articles

Comparison of EULAR/PRINTO/PReS Ankara 2008 and 2022 ACR/EULAR Classification Criteria for Granulomatosis with Polyangiitis in Children.

Granulomatosis with polyangiitis (GPA) is an antineutrophil cytoplasmic antibody-associated vasculitis. The 2022 American College of Rheumatology/European Alliance of Associations for Rheumatology (ACR/EULAR)-endorsed classification criteria for GPA was derived using data only from adult patients. We aimed to assess the performance of the ACR/EULAR classification criteria for GPA in pediatric patients and compare it with the EULAR/Pediatric Rheumatology International Trials Organization (PRINTO)/Pediatric Rheumatology European Society (PReS)-endorsed Ankara 2008 criteria for GPA. Retrospective data of pediatric patients with GPA in 20 centers from 9 countries were evaluated. The diagnosis of GPA was made according to the expert opinion. The sensitivity, specificity, positive predictive value, and negative predictive value of the criteria sets were evaluated. The study included 77 patients with GPA and 108 controls (immunoglobulin A vasculitis (n = 44), Takayasu's arteritis (n = 20), microscopic polyangiitis (n = 16), polyarteritis nodosa (n = 14), Behçet's disease (n = 12), eosinophilic granulomatosis with polyangiitis (n = 1), and Cogan's syndrome (n = 1)) with a median age of 17.8 and 15.2 years, respectively. Of patients with GPA, constitutional symptoms (85.7%) and ear-nose-throat involvement (79.2%) were the most common presentations. In the GPA group, 73 patients fulfilled the Ankara 2008 criteria and 69 the ACR/EULAR classification criteria. Sensitivities of the Ankara 2008 criteria and the ACR/EULAR classification criteria were 94.8% and 89.6%, while specificities were 95.3% and 96.3%, respectively. No significant difference was found between sensitivities and specificities of both classification criteria (p= 0.229 and p= 0.733, respectively). In children, both the ACR/EULAR and EULAR/PRINTO/PReS Ankara 2008 classification criteria for GPA perform well and similarly.

The impact of the COVID-19 pandemic on pediatric rheumatology practice: an international, cross-sectional survey study

ObjectiveThe COVID-19 pandemic has affected patient care in general. We aimed to analyze the impact of the pandemic on pediatric rheumatology practice. MethodsAn online survey including 22 questions was created by the representatives of the Emerging RheumatoloGists and rEsearchers (EMERGE) group of the Pediatric Rheumatology European Society (PReS) on SurveyMonkey. The descriptive analysis of the responses was performed on SurveyMonkey. ResultsOverall, 469 pediatric rheumatologists (F/M: 2.9) from 70 countries completed the survey. The practice of drug prescription is not affected by the pandemic, according to 65.3 % of the respondents, while 24.3 % and 16.5 % are prescribing biologic drugs and corticosteroids less often, respectively. Over 40 % of the respondents have seen an increased number of patients with vasculitis or chilblains during the pandemic. One-third of the respondents stated no adjustments in their clinical practice after 2.5 years of COVID-19 pandemic. The rest indicated implementing various changes, with an emphasis on incorporating telemedicine. Telemedicine constitutes ≥10 % of the clinical practice for one-third of the participants. Nonetheless, 35.5 % agree that there are still delays in patient care due to the pandemic. However, most (∼90 %) think our practice is returning to the pre-pandemic routine. ConclusionThe findings of our study indicate a significant alteration in pediatric rheumatology practice due to the pandemic. This includes increased caution when prescribing anti-rheumatic drugs, a transition towards telemedicine utilization, delays in routine care, and a rise in COVID-19-related inflammatory conditions. It is imperative to address these aspects in order to improve patient care in pediatric rheumatology.

Welcome to the New Issue (Vol: 29, No: 3, 2023) and Current News of the European Journal of Therapeutics

Welcome to the New Issue (Vol: 29, No: 3, 2023) and Current News of the European Journal of Therapeutics

OA39 Development of a Childhood Lupus Low Disease Activity State definition: recommendations from the International Childhood Lupus Treat-to-Target Task Force

Abstract Background/Aims International interest in development of treat-to-target (T2T) in both childhood-onset systemic lupus erythematosus (cSLE) and adult-onset SLE (aSLE) is increasing. T2T could facilitate more effective and structured use of treatments, aggressively controlling disease activity, preventing organ damage, and improving health-related quality of life. The first step is the selection of an appropriate target. Remission is deemed the ultimate target, but may not be attainable by all. Low disease activity (LDA), based on the principle of “tolerable” disease activity on stable treatment, with low corticosteroid dosage, may be more appropriate for some patients. The aim of this study was to derive a consensus-based cSLE appropriate definition of LDA, building upon existing aSLE definitions to improve applicability to cSLE, whilst maintaining sufficient unity to ensure that future T2T studies including adolescents and adults together are possible. Methods The International cSLE T2T Task Force, including 18 specialists from paediatric rheumatology/nephrology, and adult rheumatology undertook a series of Delphi surveys, exploring views on aSLE LDA targets. Two virtual consensus meetings were held, utilising a modified nominal group technique to debate, modify, and vote upon topics underpinning the cSLE LDA target and its criteria. Agreement of > 80% was considered consensus. Results The task force agreed that the LDA target should encompass cSLE as a whole and be based upon the aSLE Lupus Low Disease Activity State definition (LLDAS), with modifications to make it more applicable to cSLE (cLLDAS, all 100% agreement). A conceptual definition of cLLDAS was defined: ‘A state, which if sustained, is associated with a low likelihood of adverse outcome (considering disease activity, damage, and medication toxicity)’ (100% agreement). Five cLLDAS criteria were agreed, as detailed within Table 1. The final cLLDAS definition was endorsed by the Paediatric Rheumatology European Society (PReS) Executive Council and PReS cSLE Working Party Chair, on behalf of the Society. Conclusion A cSLE, age-appropriate definition of cLLDAS has been generated, preserving sufficient unity with the aSLE LLDAS definition to encourage life-course research. The development and validation of targets has been a key enabler for T2T trials, therefore this initiative represents a significant step forward for cSLE. Disclosure E.M.D. Smith: None. A. Aggarwal: None. J. Ainsworth: None. E. Al-Abadi: None. T. Avcin: None. L. Bortey: None. J. Burnham: None. C. Ciurtin: None. C.M. Hedrich: None. S. Kamphuis: None. D. Levy: None. L. Lewandowski: None. N. Maxwell: None. E. Morand: None. S. Ozen: None. C. Pain: None. A. Ravelli: None. C. Saad Magalhaes: None. C. Pilkington: None. D. Schonenberg: None. C. Scott: None. K. Tullus: None. M.W. Beresford: None.

OP0214-HPR EDUCATIONAL NEEDS AMONG HEALTH PROFESSIONALS IN RHEUMATOLOGY: LOW AWARENESS OF EULAR OFFERINGS AND UNFAMILIARITY WITH COURSE CONTENT AS A MAJOR BARRIER – A EULAR FUNDED EUROPEAN SURVEY

BackgroundHealth professionals in rheumatology (HPRs) should participate in post-graduate or continuous education to update and advance their knowledge and skills. This can improve patient outcomes and increase quality of care.1 EULAR aims to become a leading provider of postgraduate education for HPRs.ObjectivesThe aims of this study were to evaluate the current motivations for participating in postgraduate education of HPRs, identify barriers and facilitators for participation in postgraduate education, and evaluate participation in the current educational offerings of EULAR for HPRs across Europe.MethodsAn online survey was developed and distributed in collaboration with the EULAR Standing Committee of Education and Training (ESCET) and the Paediatric Rheumatology European Society (PReS). The questionnaire was translated by national HPR representatives in 24 languages to cover the 25 national member organisations. Barriers were assessed using 5-point Likert scales, higher scores representing higher barriers. Quantitative data were analysed using descriptive statistics. In addition, we ran the Latent Dirichlet Allocation (LDA) on the answers to the open questions. LDA is an unsupervised probabilistic topic modelling technique that extracts the meanings of a pre-defined number of topics. Design of the survey and reporting of results were done according to the Checklist for Reporting Results of Internet E-Surveys (CHERRIES).ResultsThe online questionnaire was accessed 3,589 times but only 667 complete responses were recorded. HPRs from 34 European countries responded to the survey; 80% of whom were women. The highest-ranked educational need was prevention, including lifestyle interventions and professional development. Although EULAR was well known among HPRs, only 32.1% of HPRs in adult care and 18.6% of HPRs in paediatric care have ever heard of the EULAR School of Rheumatology (Table 1 A).Table 1.A: Feedback on EULAR. Data are presented separately for HPRs in adult and paediatric care; except for the filter questions, no mandatory questions were included in the survey. To clarify the number of responses per question, the number of valid answers for each question was reported.VariablesHPRs in adult careHPRs in paediatric careHave you ever heard of the EULAR School of Rheumatology?61443 I am not sure, n(%)62 (10.1%)7 (16.3%) No, n(%)355 (57.8%)28 (65.1%) Yes, n(%)197 (32.1%)8 (18.6%)Are you aware of courses offered by the EULAR School of Rheumatology? (sub question)1978 I am not sure, n(%)30 (15.2%)2 (25.0%) No, n(%)63 (32.0%)5 (62.5%) Yes, n(%)104 (52.8%)1 (12.5%)Have you ever attended one of the EULAR School of Rheumatology courses? (sub question)1031 I am not sure, n(%)1 (1.0%)0 No, n(%)47 (45.6%)0 Yes, n(%)55 (53.4%)1 (100%)Have you ever participated in a EULAR annual congress meeting?61843 I am not sure, n(%)11 (1.8%)0 No, n(%)457 (73.9%)39 (90.7%) Yes, n(%)150 (24.3%)4 (9.3%)The main barriers to participation in EULAR’s educational offerings were identified by HPRs in adult care and in paediatric care (respectively) as: the unfamiliarity with the course content (3.48 [±1.50]; 3.92 [±1.46]), the associated costs (3.44 [±1.35]; 3.69 [±1.28]) and English language (2.59 [±1.50]; 2.80 [±1.34]).ConclusionEULAR is well-known by HPRs in Europe, however, awareness of educational offerings is low and barriers to participation are numerous. To become the leading provider of postgraduate training by 2023, EULAR could use a “franchise” model that can be tailored to local conditions. This could be achieved by strengthening national organizations by actively involving them in the development of training programs and disseminating these programs and offerings through their networks.

Current practice of transitional care for adolescents and young adults in Swiss paediatric and adult rheumatology centres.

About half of all children with rheumatic diseases need continuous medical care during adolescence and adulthood. A good transition into adult rheumatology is essential. Guidelines for a structured transition process have therefore been recommended by the European League Against Rheumatism (EULAR) and the Paediatric Rheumatology European Society (PReS). However, implementation of these guidelines requires resources often not available in a busy clinical practice. To assess the current practice of transitional care in Switzerland in relation to EULAR/PReS recommendations and to describe gaps and challenges in following the recommendations. All paediatric Swiss rheumatology centres and their collaborating adult centres offering a transition service to adult care were invited to participate in this survey. The responsible paediatric and adult rheumatologist of each centre was interviewed separately using a structured manual addressing the EULAR/PReS transitional care recommendations. All 10 paediatric and 9 out of 10 adult rheumatologists agreed to participate. Centres varied in the number of patients in transition, from n = 0 to n = 111. The following EULAR/PReS recommendations were implemented and applied in most centres: continuity in the healthcare team, consultations focused on adolescents and young adults, joint consultations between the paediatric and adult rheumatologist, and access to the EULAR website. Only rarely did a centre have a written transition policy or evaluate their transitional care programme. The vast majority of the interviewees had no specific training in adolescent health. Most centres rated their transitional care performance as very good. Transition in Switzerland is not uniform and consequently the implementation of the EULAR/PReS recommendations is variable in Swiss rheumatology centres. Skills of healthcare professionals, continuity between clinical settings, size of the centres, and hospital focus on the needs of adolescents and young adults may represent key predictors of successful transitional care for patients with chronic rheumatic diseases. Future studies should examine these variables.

Diagnosis and Treatment of Small Vessel Childhood Primary Angiitis of the Central Nervous System (sv-cPACNS): An International Survey.

Childhood primary angiitis of the Central Nervous System (cPACNS) is a rare autoimmune and inflammatory disease. It can result in significant neuronal damage, neurodevelopmental delay and potentially death. Childhood PACNS is divided into subcategories: angiography-positive p-cPACNS that affects medium and large vessels, and angiography-negative small vessel sv-cPACNS. Due to its rarity, variable clinical representation, and the lack of a diagnostic criteria and therapeutic plans, diagnosis and treatment of cPACNS is challenging and approaches vary. This survey collected information on diagnostic and therapeutic approaches to sv-PACNS. It was shared with international clinician networks, including the German Society for Paediatric Rheumatology, the Paediatric Rheumatology European Society, the “Network Paediatric Stroke,” and members of the American College of Rheumatology/CARRA Paediatric Rheumatology list server. This project has shown consensus in numerous diagnostic and therapeutic treatment approaches, highlighting key areas which will be utilised to develop statements in the use of expert consensus meetings to standardise diagnostic and therapeutic approaches in this rare inflammatory disease.

Diagnosis and Treatment of Angiography Positive Medium to Large Vessel Childhood Primary Angiitis of Central Nervous System (p-cPACNS): An International Survey.

Childhood Primary Angiitis of Central Nervous System (cPACNS) is rare, but can cause significant damage and result in disability or even death. Because of its rarity, the sometimes acute and variable presentation, limited awareness, and the absence of widely accepted diagnostic and therapeutic standards, cPACNS is a diagnostic and therapeutic challenge. Three subcategories of cPACNS exist, including angiography-positive non-progressive p-cPACNS, angiography-positive progressive p-cPACNS which both affects the medium to large vessels, and angiography-negative small vessel sv-cPACNS. Diagnosis and treatment of cPACNS relies on personal experience, expert opinion and case reports/case series. To collect information on diagnostic and therapeutic approaches to transient and progressive cPACNS, a survey was shared among international clinicians (German Society for Pediatric Rheumatology, the Pediatric Rheumatology European Society, the German speaking “Network Pediatric Stroke,” and members of the American College of Rheumatology/CARRA Pediatric Rheumatology list server). Results from this survey will be used to define statements toward a consensus process allowing harmonization of diagnostic and therapeutic approaches and the generation of evidence in a rare condition.

Predicting Macrophage Activation Syndrome in Childhood-onset Systemic Lupus Erythematosus Patients at Diagnosis.

Macrophage activation syndrome (MAS), a life-threatening inflammatory complication, is increasingly recognized in childhood-onset systemic lupus erythematosus (cSLE). It can be a challenge to differentiate active cSLE from MAS. We generated decision rules for discriminating MAS from active cSLE in newly diagnosed patients. We conducted a retrospective cohort study of consecutive, newly diagnosed, active cSLE patients with fever, requiring hospital admission to The Hospital for Sick Children from January 2003 to December 2007 (cohort 1) and January 2008 to December 2013 (cohort 2). All patients met ≥ 4 American College of Rheumatology or Systemic Lupus International Collaborating Clinics criteria, and were steroid-naïve and infection-free. MAS was diagnosed based on expert opinion. Recursive partitioning was applied to each cohort to derive a decision rule based on clinical and laboratory features, distinguishing MAS from non-MAS cSLE. Each decision rule was applied to the alternate, independent cohort. Sensitivity and specificity of these decision rules were compared to existing criteria. Cohort 1 (n = 34) and cohort 2 (n = 41) each had 10 patients with MAS. Recursive partitioning in cohort 1 identified ferritin ≥ 699 μg/L as the sole best discriminator between MAS and non-MAS patients (R2 = 0.48), and in cohort 2, ferritin ≥ 1107 μg/L was the best discriminator for MAS, followed by lymphocytes < 0.72 × 103/mm3 (R2 = 0.52). Cross-validation of our decision rules maintained 90-100% sensitivity and 65-85% specificity. Our decision rule demonstrated improved performance compared to preliminary guidelines for MAS in cSLE from the Lupus Working Group of the Paediatric Rheumatology European Society and familial hemophagocytic lymphohistiocytosis diagnostic criteria. Validation in independent cohorts is required.

How the COVID-19 pandemic has influenced pediatric rheumatology practice: Results of a global, cross-sectional, online survey

ObjectivesThe COVID-19 pandemic is a global health problem. We, as the EMERGE (EMErging RheumatoloGists and rEsearchers) group of PReS (Pediatric Rheumatology European Society) analyzed how the pandemic has affected pediatric rheumatology practice. MethodsAn online survey was developed to assess changes in pediatric rheumatology practice due to the pandemic. Results were analyzed using descriptive statistics. ResultsFrom 70 countries, 493 pediatric rheumatologists (80.3% in pediatric rheumatology practice for ≥5 years) responded to the survey. Around 70% disagreed that the pandemic led to reduced prescription of nonsteroidal anti-inflammatory drugs, conventional synthetic and biologic disease-modifying antirheumatic drugs. Almost half were more likely to taper corticosteroids faster. One-fifth hesitated to switch the major immunosuppressant during a flare. Patients encountering difficulties obtaining hydroxychloroquine and tocilizumab due to shortages were noted by 192 (38.9%) and 44 (8.9%), respectively. Twenty to 30% indicated that their patients had experienced a flare or delay in diagnosis/intervention due to postponed appointments.53% mentioned use of phone calls/smartphone applications while 47% shifted towards video consultations for patient care. Respondents indicated an increased number of patients with Kawasaki disease (30%), macrophage activation syndrome (15.6%), unusual vasculitic rashes (31.4%), and hyperinflammation (33.5%) during the pandemic. ConclusionThis is the largest survey to date addressing changes in pediatric rheumatology practice due to the COVID-19 pandemic. Primary changes were due to delays in clinic appointments, increase in use of virtual technologies, and concerns about the use of immunosuppressive therapies. An increased number of patients with Kawasaki disease/hyperinflammation mentioned by the respondents is noteworthy.

Multidisciplinary collaboration among young specialists: results of an international survey by the emerging EULAR network and other young organisations.

BackgroundMultidisciplinary collaboration is defined as a collective work involving multiple disciplines and is common in clinical care and research. Our aim was to describe current clinical and research collaboration among young specialists and to identify unmet needs in this area.MethodsAn online survey was disseminated by email and social media to members of the EMerging EUlar NETwork, the Young Nephrologists’ Platform, the Paediatric Rheumatology European Society Emerging Rheumatologists and Researchers and the European Academy of Allergy and Clinical Immunology Junior Members.ResultsOf 303 respondents from 36 countries, 61% were female, 21% were aged below 30 years and 67% were aged 31–40 years. Young rheumatologists were the most represented (39%), followed by young nephrologists (24%), young paediatricians (20%), young allergologists (11%) then young internists (3%) and 3% other specialities. Collaborations were reported frequently by phone and email, also by various combined clinics while common local multidisciplinary meetings were uncommon. 96% would like to develop clinical research collaborations and 69% basic research collaborations. The majority of young specialists would be interested in online (84%) and/or 1–2 days (85%) common courses including case discussion (81%) and training workshops (85%), as well as webinars recorded with several specialists on a specific disease (96%).ConclusionsThis collaborative initiative highlighted wishes from young specialists for developing (1) regular local multidisciplinary meetings to discuss complex patients, (2) clinical research collaboration with combined grants and (3) multidisciplinary online projects such as common courses, webinars and apps.

Ultrasound, X-ray, computed tomography and clinical tests for diagnosis of abdominal purpura in children: A retrospective study.

Imaging modalities are not included in The European League Against Rheumatism, The Pediatric Rheumatology International Trials Organization and The Pediatric Rheumatology European Society 2010 criteria for the evaluation of abdominal purpura. The objective of the present study was to compare diagnostic parameters of ultrasound, X-ray and computed tomography (CT) for diagnosis of abdominal purpura considering the American College of Rheumatology (ACR) criteria as ‘reference standard’ in children with acute abdominal pain. Medical records of 215 children with acute abdominal pain were reviewed. Data regarding demographics and clinical characteristics, laboratory tests, X-ray, ultrasound findings, and computed tomographic images were collected and analyzed. Decision curve analysis was used for evaluation of the beneficial score for each diagnostic modality. Among diagnostic modalities, CT had the highest sensitivity (0.939); however, ultrasound findings had the highest accuracy (0.861) for diagnosis of abdominal purpura. Unlike X-ray and laboratory tests, ultrasound and CT were successful at detecting abdominal purpura when children had only colicky pain and were aged <20 years; however, occult blood stool test and granulocytes in the walls of small venules and arterioles (biopsy results) were negative. With respect to the ACR criteria, there were seven and three inconclusive results for ultrasound and CT, respectively. Abdominal ultrasound is an easy, non-invasive and safe method for the detection of abdominal purpura in children.

Live attenuated MMR/V booster vaccines in children with rheumatic diseases on immunosuppressive therapy are safe: Multicenter, retrospective data collection

PurposeTo collect retrospective data of patients with Juvenile Idiopathic Arthritis (JIA) and other rheumatic diseases who received live attenuated booster measles-mumps-rubella (MMR) or measles-mumps-rubella-varicella (MMR/V) during treatment with immunosuppressive therapy. ResultsData from 13 pediatric rheumatology centers in 10 countries, including 234 patients, were collected. Mean age at diagnosis was 5 ± 2.7 years, 67% were girls. Among them, 211 (90.2%) had JIA and 110 (47%) were in remission on medication. Disease activity was low in 37%, high in 8%, and moderate in 8%. One hundred-twenty-four received MMR/V booster while on methotrexate (MTX); 3 reported local mild adverse events (AE). Among 62 on MTX + biologics and 9 patients who received a combination of 2 disease modifying antirheumatic drugs (DMARDs), 9 reported mild AE. Among 39 on biologics, 1 reported fever one day after booster vaccination. No vaccine-related infection of measles, rubella, mumps or varicella was reported, none of the patients developed disease flare, including those with high disease activity. ConclusionsIn this retrospective study, live-attenuated MMR/V booster vaccines were safe for children with rheumatic diseases, on immunosuppressive therapies. This strengthens the Paediatric Rheumatology European Society (PReS) recommendation that vaccination with live attenuated vaccines in patients on immunosuppressive therapies can be considered individually, weighing the benefit of vaccination against the risk of inducing infection through vaccination. These data provide the basis for a prospective data collection study, planned by the PReS vaccination study group.

Childhood-Onset Takayasu Arteritis (c-TA): Current and Future Drug Therapy

Childhood-onset Takayasu arteritis (c-TA) is the third most common systemic vasculitic disorder in children. Vascular stenosis is the main complication, and aneurysms are reported in 19-65% of cases, often in combination with stenotic lesions. Management of patients with c-TA is largely based on studies involving predominantly patients with adult-onset TA (a-TA). More widely used criteria for patients with c-TA have been devised by the joint European League Against Rheumatism, Pediatric Rheumatology International Trials Organization, and Pediatric Rheumatology European Society. Of the available imaging modalities, those that do not use radiation (color Doppler ultrasound and magnetic resonance angiogram) are preferred over 18F-labeled fluoro-2-deoxyglucose (18F-FDG) positron-emission tomography, computed tomography (CT), and CT angiogram in children. Remission rates have been reported to be lower in c-TA than in a-TA, and published mortality rates in c-TA range from 16 to 40%, which is much higher than reported in patients with a-TA. The usual drug therapy options include steroids plus steroid-sparing second-line immunosuppressants, such as mycophenolate, azathioprine, methotrexate, cyclophosphamide, and cyclosporine, along with antiplatelet agents. Interleukin-6 inhibitors such as tocilizumab, as well as the tumor necrosis factor inhibitors, are other aggressive therapeutic options. As yet, no randomized controlled trials have been conducted in c-TA.

Clinical course and prognostic factors of childhood Takayasu\u2019s arteritis: over 15-year comprehensive analysis of 101 patients

BackgroundChildhood Takayasu’s arteritis (c-TA) is scarcely reported but is characterized by devastating morbidity and mortality. This study aims to investigate the clinical course of c-TA and prognostic factors associated with rehospitalization and events including vascular complications, flares, and death.MethodsAn ambispective study of 101 c-TA patients satisfying the American College of Rheumatology (ACR) criteria and/or the European League against Rheumatism (EULAR)/Pediatric Rheumatology International Trials Organization (PRINTO)/Pediatric Rheumatology European Society (PReS) criteria was conducted from January 2002 to December 2017. Data on demographic, clinical, laboratory, imaging, and therapeutic features were collected. Event-free survival, complication-free survival, flare-free survival, rehospitalization-free survival, and associated prognostic factors were assessed by Kaplan-Meier survival curve and propensity score analysis.ResultsThe median age at c-TA onset was 14 (interquartile range (IQR) 12–16) years and 76.2% were female. Hypertension (70.3%), blood pressure discrepancy (55.4%), bruits (51.5%), and pulse deficits (37.6%) were core presentations. Major vascular involvement included the renal artery (62.4%), abdominal aorta (42.6%), subclavian artery (43.6%), and carotid artery (42.6%). Glucocorticoids (78.2%), antihypertensive drugs (72.3%), antiplatelet agents (72.3%), and revascularization (57.4%) were made up the majority administered. At a median 2.4 (IQR 0.7–6.1) years of follow-up, events, rehospitalization, vascular complications, flares and death were observed in 44.6%, 37.6%, 44.6%, 26.7%, and 3%, respectively. The 5-year event-free survival, rehospitalization-free survival, vascular complication-free survival, and flare-free survival were 42.8%, 55.8%, 45.9%, and 62.3%, respectively. Body mass index (BMI) (hazard ratio (HR) = 0.49, 95% confidence interval (CI) 0.30–0.81, p = 0.005), stroke (HR = 7.37, 95% CI 2.35–23.1, p = 0.001), and revascularization (HR = 0.51, 95% CI 0.27–0.94, p = 0.032) were independent prognostic predictors of events. Predictors for rehospitalization include age at admission (HR = 0.81, 95% CI 0.69–0.94, p = 0.006), renal artery involvement (HR = 0.49, 95% CI 0.25–0.96, p = 0.037), and elevated C-reactive protein (CRP; HR = 2.50, 95% CI 1.24–5.00, p = 0.01). BMI level (p = 0.024) and renal artery involvement (p = 0.015) were also associated with vascular complications, while revascularization (p = 0.002) independently correlated with re-flares.ConclusionsThis large ambispective study of c-TA revealed an early 3% mortality at the first year and around 50% morbidity within 5 years after diagnosis. Hypertension, renal artery involvement, and revascularization based on anti-inflammation, antihypertension, and antiplatelet medications dominated c-TA with indications for optimistic prognosis. Patients with initial lower BMI level, a younger age at admission, stroke, and elevated CRP have a high risk of poor outcomes, requiring close c-TA monitoring and more aggressive management.Trial registrationNCT03199183, unique protocol ID: 2016-ZX43. June 26, 2017

Multi-centre national audit of juvenile localised scleroderma: describing current UK practice in disease assessment and management

ObjectiveTo describe current United Kingdom practice in assessment and management of patients with juvenile localised scleroderma (JLS) compared to Paediatric Rheumatology European Society (PRES) scleroderma working party recommendations.MethodsPatients were included if they were diagnosed with JLS and were under the care of paediatric rheumatology between 04/2015–04/2016. Retrospective data was collected in eleven UK centres using a standardised proforma and collated centrally.Results149 patients were included with a median age of 12.5 years. The outcome measures recommended by the PRES scleroderma working party were not utilised widely. The localised scleroderma cutaneous assessment tool was only used in 37.6% of patients. Screening for extracutaneous manifestations did not meet recommendations that patients with head involvement have regular screening for uveitis and baseline magnetic resonance imaging (MRI) brain: only 38.5% of these patients were ever screened for uveitis; 71.2% had a MRI brain.Systemic treatment with disease-modifying anti-rheumatic drugs (DMARDs) or biologics was widely used (96.0%). In keeping with the recommendations, 95.5% of patients were treated with methotrexate as first-line therapy. 82.6% received systemic corticosteroids and 34.2% of patients required two or more DMARDs/biologics, highlighting the significant treatment burden. Second-line treatment was mycophenolate mofetil in 89.5%.ConclusionThere is wide variation in assessment and screening of patients with JLS but a consistent approach to systemic treatment within UK paediatric rheumatology. Improved awareness of PRES recommendations is required to ensure standardised care. As recommendations are based on low level evidence and consensus opinion, further studies are needed to better define outcome measures and treatment regimens for JLS.

Are diffuse and limited juvenile systemic sclerosis different in clinical presentation? Clinical characteristics of a juvenile systemic sclerosis cohort.

Juvenile systemic sclerosis is an orphan disease. Currently, the majority of juvenile systemic sclerosis cohort studies are retrospective in design without standardized assessment. This study was conducted prospectively to investigate the difference in manifestations of limited cutaneous juvenile systemic sclerosis and diffuse cutaneous juvenile systemic sclerosis subtypes. An additional aim was to compare these data to other juvenile systemic sclerosis cohorts and a large adult systemic sclerosis cohort. Patients fulfilling the Paediatric Rheumatology European Society juvenile systemic sclerosis classification criteria were included. Clinical characteristics and patient-related outcomes were assessed. In all, 88 patients with a mean disease duration of 3.5 years were enrolled, 72.5% with diffuse cutaneous juvenile systemic sclerosis with a mean modified Rodnan Skin score of 18 and 27.5% with limited cutaneous juvenile systemic sclerosis with mean modified Rodnan Skin score of 9. The mean age at the onset of Raynaud's and first non-Raynaud's symptoms was similar in both groups, approximately 9 and 10.5 years. Active digital tip ulcerations were present in 29% diffuse cutaneous juvenile systemic sclerosis and none in the limited cutaneous juvenile systemic sclerosis subjects (p = 0.005). Of those with cardiopulmonary testing, 3% of diffuse cutaneous juvenile systemic sclerosis and 23% of limited cutaneous juvenile systemic sclerosis group had cardiac involvement (p = 0.015), and 41% diffuse cutaneous juvenile systemic sclerosis and 22% of the limited cutaneous juvenile systemic sclerosis group had pulmonary involvement (p = 0.009). Physician global disease damage assessment was higher in the diffuse cutaneous juvenile systemic sclerosis group compared to the limited cutaneous juvenile systemic sclerosis group: 35 and 15 (p = 0.021). The majority of this international juvenile systemic sclerosis cohort had diffuse cutaneous juvenile systemic sclerosis (72.5%) with more frequent vascular and pulmonary involvement compared to the limited cutaneous group, who had increased cardiac involvement. Our cohort reflects prior findings of published juvenile systemic sclerosis cohorts and emphasizes a difference in the presentation compared to adult-onset systemic sclerosis.

Proceedings of the 24th Paediatric Rheumatology European Society Congress: Part two

Proceedings of the 24th Paediatric Rheumatology European Society Congress: Part two

Proceedings of the 24th Paediatric Rheumatology European Society Congress: Part one

Proceedings of the 24th Paediatric Rheumatology European Society Congress: Part one

Proceedings of the 24th Paediatric Rheumatology European Society Congress: Part three

Proceedings of the 24th Paediatric Rheumatology European Society Congress: Part three