Pediatric Rheumatology Centers Research Articles

The place of JAK inhibitors in systemic juvenile idiopathic arthritis with lung disease (SJIA-LD): French experience.

A new form of systemic juvenile idiopathic arthritis (SJIA) with associated lung disease (SJIA-LD) has recently been described. Multiple lines of treatment have failed to yield satisfactory results for this disorder. JAK inhibitors (JAKis) have recently been approved for the treatment of JIA, but clinical evidence of their efficacy in SJIA-LD is still weak. Here we describe and assess real-life experience of SJIA-LD treatment with JAKis in France. This is a retrospective study based on information gathered from patients' medical records. Systemic and pulmonary symptoms, biological data including CRP, ferritin, IL18, chest CT scan, and functional respiratory tests were collected. Eight patients with SJIA-LD were identified in French pediatric rheumatology centers. All received at least one JAKi (baricitinib, ruxolitinib, and/or tofacitinib). Complete disease control was obtained in four patients. Steroids were tapered in four patients and stopped in two. Three patients presented an episode of MAS shortly after anti-IL1s were stopped when JAKis were introduced. Two patients had other serious side effects (viral reactivation-EBV, BK virus, cytopenia). At last follow-up, one patient had died from severe MAS, two patients had undergone hematopoietic stem cell transplantation, four were in complete response (two of them free of steroids), and one in partial response with JAKis. Lung response to JAKi was not clearly linked to disease duration. JAKis offer another therapeutic option for patients with SJIA-LD. However, the risk of MAS argues for caution about stopping anti-IL1s when introducing JAKis. Tolerance needs careful monitoring in larger studies.

Diagnostic and Therapeutic Insights Into Pediatric Neurosarcoidosis: Observations From French Pediatric Rheumatology Centers

BackgroundThe diagnosis and management of neurosarcoidosis (NS) in pediatric patients remain challenging, with limited case documentation to guide clinicians. Most existing reports focus on initial presentations. This study aimed to outline the clinical features, management, and medium-term outcomes of pediatric NS MethodsIn this retrospective, multicentric, observational study, we collected data from pediatric patients followed in French pediatric rheumatology centers with a diagnosis of NS between January 2001 and June 2023. ResultsWe identified 11 patients diagnosed with NS, comprising eight girls and three boys. The mean age at diagnosis of sarcoidosis was 10 (5 to 15) years, and the mean age of diagnosis of NS was 11.5 (5 to 17) years. Predominant neurological symptoms included headache (nine of 11 patients), papilledema (6 of 11 patients), facial palsy (two patients), seizures (one patient), and motor deficit (two patients). Nine of 11 patients had eye involvement, which consisted of granulomatous and bilateral uveitis. All patients exhibited meningitis, with cerebrospinal fluid white blood cell counts ranging from 6 to 70 cells/mm3. Six individuals presented neurological abnormalities on imaging, detailed in this study. Treatment primarily involved corticosteroids, methotrexate, and tumor necrosis factor alpha (TNF-alpha) inhibitors. Biologics targeting TNF-alpha were necessary to achieve remission in eight of 11 patients. In two patients who did not receive this treatment initially, it was required later in the course of evolution. ConclusionsThis study enhances understanding of the clinical course of pediatric NS and supports the early use of TNF-alpha biologics for improved management in affected children.

Transition Guide Dissemination to Foster Patient-Care Team Conversations: A Childhood Arthritis Rheumatology Research Alliance Transition Learning Collaborative Pilot Implementation Study.

Uptake of evidence-informed health care transition processes among pediatric rheumatologists is low despite poor outcomes of transition from pediatric to adult care. We piloted a learning collaborative model to implement transition guides. We dually assessed implementation outcomes and changes in reported patient-care team discussions about transition. This was a type II hybrid effectiveness-implementation pilot study of transition guide dissemination to patients at least 14 years old with rheumatic conditions across nine pediatric rheumatology centers in the Childhood Arthritis Rheumatology Research Alliance Transition Learning Collaborative. We evaluated implementation outcomes (feasibility, adaptations, and fidelity) and the proportion of patients surveyed that reported having discussions with their care team regarding transfer planning. Six sites were retained through the COVID-19 pandemic (below 70% target). Five out of six sites contributed outcome data (met 80% target) but with substantial heterogeneity in how transition guides were shared (eg, in-person, electronic messages, and posted flyers), and data were collected. The pooled proportion of respondents having discussed transfer planning with their care team was 39% preimplementation (n = 239; 95% confidence interval [CI] 32%-46%) and 55% postimplementation (n = 864; 95% CI 36%-73%). After implementation, there were significant increases in the likelihood of respondents recalling receiving a transition guide (odds ratio [OR] 2.58, 95% CI 1.35-4.92) and discussing transfer planning (OR 2.14, 95% CI 1.30-3.52), adjusted for age and site of care. Transition guide dissemination is a simple intervention associated with increased awareness among young people with rheumatic conditions and discussions with their care team about transition preparation. The learning collaborative model facilitated identification of several dissemination strategies adaptable to site-specific resources.

Automated mental health screening in pediatric lupus: associations with disease features and treatment.

Patients with childhood-onset systemic lupus erythematosus (c-SLE) have higher rates of depression than their peers, which has been associated with worse medical outcomes. Therefore, it is imperative that their mental health be addressed. We utilized quality improvement (QI) methodology to automate mental health screening for patients with lupus within a pediatric rheumatology clinic. The retrospective cohort study aims to evaluate the association between mental health screening outcomes and demographics, medications, and disease activity measures in patients with childhood lupus. The mental health QI team at a quaternary pediatric rheumatology center implemented an automated process for mental health screening in patients with c-SLE. Patients seen between 2017 and June 2023 with a diagnosis of c-SLE were identified using International Classification of Disease -Clinical Modification (ICD-CM) codes. Disease activity was assessed with the Systemic Lupus Erythematosus Disease Activity Index 2000 (SLEDAI 2K). Medications were identified on outpatient and inpatient orders for conventional synthetic and biologic disease-modifying anti-rheumatic drugs, hydroxychloroquine, corticosteroids, and aspirin. Mental health screening was accomplished with the Patient Health Questionnaire (PHQ). Descriptive statistics, univariate and multivariate linear regression were used. Between January 2017 and June 2023, 117 patients with c-SLE (41% with lupus nephritis) completed 534 total screenings. Each patient completed PHQ screenings, a median of 5 [interquartile range 2, 6] times. Screening increased when the screening process was automated. Those who were Black, female, or prescribed leflunomide, mycophenolate, and corticosteroids had higher PHQ scores. Mental health support is essential for patients with chronic rheumatologic diseases such as SLE. Sustainable processes for quickly identifying depression are needed for optimal care of patients with SLE. Our process of automated, streamlined mental health screening successfully increased the screening of patients with SLE at every visit and led to timely interventions for positive PHQ scores. Higher PHQ scores were correlated with patients on leflunomide, mycophenolate, and corticosteroids. Future research should identify modifiable risk factors for high PHQ scores that the medical team can target.

A rare disease with many faces: A multicenter registry of IgG4-related disease in children

Abstract Objectives We aimed to report the characteristics of pediatric IgG4-related disease (IgG4-RD) through a multicentre registry, to assess disease clusters, and to evaluate the performances of the 2019 American College of Rheumatology and European League Against Rheumatism (ACR/EULAR) classification criteria and the 2020 revised comprehensive diagnostic (RCD) criteria in this cohort. Methods Data of IgG4-RD patients in 13 pediatric rheumatology centers were recorded to a web-based registration system. The diagnosis of IgG4-RD was made according to the 2011 comprehensive diagnostic criteria. Results Thirty-five children (19 females and 16 males) with IgG4-RD were enrolled. The median age at diagnosis was 13.3 (25p-75p; 9.9–15.2) years. The most common organ involvement was the eye (n = 21, 60%), followed by lymph nodes (n = 12, 34.3%), musculoskeletal system (n = 12, 34.3%), and neurological system (n = 9, 25.7%). We identified three clusters in our study cohort: those with eye involvement (n = 11, 31.4%), those with eye involvement and neurological findings (n = 15, 42.9%), and those with pancreato-hepatobiliary disease and lymph node involvement (n = 9, 25.7%). Serum IgG4 levels were high in 19 out of 28 patients (67.8%). All patients except one received corticosteroid treatment, and azathioprine was the most preferred drug as a steroid-sparing agent. The sensitivities of the 2019 ACR/EULAR classification criteria and the 2020 RCD criteria were 5.7% and 88.5%, respectively. Conclusion IgG4-RD has a wide variety of clinical manifestations, however in children the most common presentation was orbital involvement. The 2020 RCD criteria had a better performance whereas the 2019 ACR/EULAR classification criteria performed poorly in pediatric patients.

Efficacy and safety of neridronate in paediatric type I complex regional pain syndrome: a multicentre experience.

Evidence regarding the efficacy of neridronate in the treatment of complex regional pain syndrome type I (CRPS I) is increasing, however, very little data are available in paediatric age. Our aim was to analyse the safety and the efficacy of neridronate in a case series of children with CRPS I, according to the Budapest criteria, who did not respond to previous pharmacological and physical therapy. We collected data of children affected by CRPS I from three paediatric rheumatology centres who received neridronate. Efficacy was evaluated by changes in pain intensity, vasomotor changes, physical function, need for pain medications and MRI findings. Adverse effects were also documented. Five children (3 females and 2 males, mean age 10.4 years, range 7-13 years) who received neridronate (4 intravenous, 1 intramuscular) were included. All patients had failed previous medical treatments (NSAIDs in 4, local steroids in 2, gabapentin, vitamin D and calcium supplementation in 1) and non-medical therapies (physiotherapy in 4, magnetotherapy in 2, laser in 1). Four out of five patients reported a significant improvement in pain (average VAS pre-treatment 9.6, post-treatment 2.6), recovery of physical function, and a reduced need for pain medications. Before treatment, all patients underwent MRI which revealed bone oedema that disappeared in the three of them after treatment. Neridronate was well-tolerated as only one patient experienced mild flu-like symptoms. Our data suggest that in children as in adults with CRPS I, neridronate may represent an effective and safe treatment option, particularly in those who do not respond to other pain treatments.

Diagnosis journey for children with juvenile idiopathic arthritis: a qualitative study

ObjectiveThe objective is to explore the journey to diagnosis and referral pathway from the onset of symptoms to the initial assessments at paediatric rheumatology (PR) centres, based on the experience...

Exploring factors for predicting colchicine responsiveness in children with PFAPA

Periodic fever, aphthous stomatitis, pharyngitis, and cervical adenitis syndrome (PFAPA) are the most common autoinflammatory syndromes in children. This study aimed to evaluate the clinical and laboratory parameters that may predict colchicine responsiveness.This retrospective, multicenter, cross-sectional study involved nine pediatric rheumatology centers from our country., The patients diagnosed with PFAPA were compared on the basis of their responses to colchicine. In the 806 (42.3% female 57.7% male) patients, the most common clinical findings were fever (100%), exudative tonsillitis (86.5%), pharyngitis (80.9%), and aphthous stomatitis (50.5%). The mean attack frequency was 13.5 ± 6.8 attacks per year lasting for a mean of 3.9 ± 1.1 days. Colchicine treatment was attempted in 519 (64.4%) patients, with 419 (80.7%) showing a favorable response. In patients who underwent MEFV gene analysis (70.8%), the most common variant was M694V heterozygous (16.8%). The presence of pharyngitis (p = 0.03, 95% CI 0.885 to 0.994), the presence of arthralgia (p = 0.04, 95% CI 0.169 to 0.958), and having more frequent attacks (p = 0.001, 95% CI 0.028 to 0.748) were found to be associated with colchicine unresponsiveness, whereas the carriage of the M694V variant (p = 0.001, 95% CI 0.065 to 0.242) was associated with colchicine responsiveness.Conclusion: This study identified the presence of pharyngitis, arthralgia, and increased attack frequency in patients with PFAPA as factors predicting colchicine unresponsiveness, whereas the carriage of the M694V variant emerged as a predictor of colchicine responsiveness. Predicting colchicine response at disease onset may facilitate a more effective management of PFAPA.What is Known:• Colchicine treatment can be used in the prophylaxis of PFAPA disease.• Having the MEFV variant is the most commonly known factor in predicting response to colchicine.What is New:• The presence of pharyngitis or arthralgia, and more frequent attacks in PFAPA disease were found to be independently associated with colchicine unresponsiveness. • Carrying the M694V variant was identified as the sole factor predicting colchicine responsiveness.

Clinical characteristics and prognostic factor in juvenile dermatomyositis: data of the Spanish registry

BackgroundJuvenile Dermatomyositis (JDM) is the most common chronic idiopathic inflammatory myopathy in children. The diagnosis is clinical. Baseline laboratory and complementary studies trace the phenotype of these patients. The objective of this study was to describe epidemiological, clinical and laboratory characteristics at diagnosis of JDM patients included in the Spanish JDM registry, as well as to identify prognostic factors on these patients.MethodsWe retrospectively reviewed clinical features, laboratory tests, and complementary studies at diagnosis of JDM patients included on the Spanish JDM registry. These data were analyzed to assess whether there was a relationship with the development of complications and time to disease inactivity.ResultsOne hundred and sixteen patients from 17 Spanish paediatric rheumatology centres were included, 76 girls (65%). Median age at diagnosis was 7.3 years (Interquartile range (IQR) 4.5–10.2). All patients had pathognomonic skin lesions at the beginning of the disease. Muscle weakness was present in 86.2%. Median Childhood Muscle Assessment Scale was 34 (IQR 22–47). Twelve patients (34%) had dysphagia and 3,5% dysphonia. Anti-p155 was the most frequently detected myositis specific antibody, followed by anti-MDA5. Twenty-nine patients developed calcinosis and 4 presented with macrophage activation syndrome. 70% reached inactivity in a median time of 8.9 months (IQR 4.5–34.8). 41% relapsed after a median time of 14.4 months (IQR 8.6–22.8) of inactivity. Shorter time to treatment was associated with better prognosis (Hazard ratio (HR) = 0.95 per month of evolution, p = 0.02). Heliotrope rash at diagnosis correlates with higher risk of development complications.ConclusionsWe describe heliotrope rash as a risk factor for developing complications in our cohort of JDM patients, an easy-to-evaluate clinical sign that could help us to identify the group of patients we should monitor closely for this complication.

Defining Criteria for Disease Activity States in Systemic Juvenile Idiopathic Arthritis Based on the Systemic Juvenile Arthritis Disease Activity Score.

Our objective was to develop and validate cutoff values in the systemic Juvenile Arthritis Disease Activity Score 10 (sJADAS10) that distinguish the states of inactive disease (ID), minimal disease activity (MDA), moderate disease activity (MoDA), and high disease activity (HDA) in children with systemic juvenile idiopathic arthritis, based on subjective disease state assessment by the treating pediatric rheumatologist. The cutoff definition cohort was composed of 400 patients enrolled at 30 pediatric rheumatology centers in 11 countries. Using the subjective physician rating as an external criterion, six methods were applied to identify the cutoffs: mapping, calculation of percentiles of cumulative score distribution, the Youden index, 90% specificity, maximum agreement, and receiver operating characteristic curve analysis. Sixty percent of the patients were assigned to the definition cohort, and 40% were assigned to the validation cohort. Cutoff validation was conducted by assessing discriminative ability. The sJADAS10 cutoffs that separated ID from MDA, MDA from MoDA, and MoDA from HDA were ≤2.9, ≤10, and >20.6, respectively. The cutoffs discriminated strongly among different levels of pain, between patients with and without morning stiffness, and among patients whose parents judged their disease status as remission or persistent activity or flare or were satisfied or not satisfied with current illness outcome. The sJADAS cutoffs revealed good metrologic properties in both definition and validation cohorts and are therefore suitable for use in clinical trials and routine practice.

The transition process for paediatric rheumatology clinic patients at a single tertiary paediatric rheumatology centre in Australia.

This study aimed to examine the transition process of paediatric rheumatology patients from the Monash Children's Hospital (MCH) in Melbourne in order to identify areas that could be improved. Retrospective review of clinical data from the rheumatology database of paediatric rheumatology patients eligible for transition between January 2015 and September 2020. One hundred and sixty-five patients were included; 57 patients were transitioned. Of patients transitioned to an adult service, 38 (88%) were on medication and 14 (33%) had active disease. All patients transitioned to the general practitioner (GP) had inactive disease off medication. Juvenile idiopathic arthritis (JIA) (non-systemic) was the most common diagnosis in patients transitioned. The mean age at which transition was first discussed was 18.0 years; the first referral was made at a mean of 18.3 years. The mean age at the first adult appointment was 18.5 years. Thirty-nine (91%) patients had a referral completed and 8 (19%) had a transfer letter. Thirteen (93%) patients transferred to the GP had a transfer letter. Transfer documents to an adult public rheumatology service rated 4.3 for quality, compared to 5.5 to the GP. Transfer of care was confirmed in 40 (93%) patients transitioned to an adult service; however, correspondence was available for only 3 (7%). Although the transition process at MCH was adequate, it could be improved through earlier discussion of the process and improved referrals and documentation. A readiness-to-transfer checklist and a young adult clinic have the potential to improve the process of transition to adult rheumatology care.

P090 Real-world data on immuno-modulation therapies and body mass index in children and young people with juvenile idiopathic arthritis

Abstract Background/Aims There is some evidence that children and young people (CYP) with Juvenile Idiopathic Arthritis (JIA) who are overweight/obese have less favourable disease control and treatment outcomes. The aim of this study was to investigate any association between BMI and total number of immune modulation therapies used in CYP with JIA. Methods This was a retrospective observational study at a tertiary paediatric rheumatology centre. Cross-sectional data was captured in April 2022 from CYP receiving immunomodulation therapies for JIA. Data on diagnosis, treatments and weight/height at last routine follow-up was included. BMI was calculated for each patient and centile assigned using Royal College of Paediatrics and Child Health electronic BMI charts as follows: BMI ≥85th to 94th centile - overweight, ≥95th centile - obese, as per population monitoring British 1990 growth reference (UK90). Results 187 CYP were identified and 180 included (seven were excluded due to missing BMI data). Baseline demographic, disease and treatment data are summarised in Table A. 67% were female. Median (interquartile range (IQR)) age in years at diagnosis and follow-up was 4.3 (2.7, 8) and 11.9 (8.5, 14.4) respectively. At follow-up, 41.1% of CYP were either obese or overweight. The median (IQR) number of total immunomodulation therapies per patient according to BMI categories were: under/normal weight 2 (1, 3); overweight 3 (2, 3) and obese 3 (2, 5). The median (IQR) years of follow-up in under/normal weight, overweight and obese groups were 4.77 (2, 8.74); 5.27 (1.86, 7) and 5.5 (3, 9.1) respectively. Conclusion CYP that were overweight/obese received more therapies compared to under/normal weight. The high proportion (41%) of CYP in this study who were either overweight or obese is similar to prevalence (44.2%) for children in year six at school in this region. Corticosteroid use and limited physical activity is associated with inadequate control of joint inflammation and increased BMI but this data was not collected and is an important limitation, in addition to cross-sectional and retrospective study design. The overweight/obese group had longer follow-up and may have accumulated more therapies. Future research to evaluate optimal dosing of immunomodulation in CYP with high BMI is warranted. Disclosure K. Hartley: Grants/research support; NIHR BRC Internship. S. Jandial: None. F. McErlane: None. E. Sen: None. S. Sampath: None.

P033 Online educational resources in paediatric and adolescent rheumatology- what’s new?

Abstract Background/Aims Paediatric and adolescent rheumatology (PAR) is a small sub-specialty, and due to the rarity of some conditions, UK trainees find it difficult to access relevant educational materials. Furthermore, due to the wide geographical distribution of paediatric rheumatology centres, regional educational opportunities are limited. A 2022 survey of the UK PAR trainee group confirmed that the creation of more PAR specific online educational materials and signposting of current guidelines/ resources was needed. Survey participants were supportive of a range of delivery methods, including webinars, e-learning, case studies and podcasts. Methods A PAR trainee education team was formed in 2022, with the aim of promoting learning opportunities, by developing and collating educational resources. The PAR trainee education team has been working both independently and in conjunction with the British Society of Rheumatology (BSR) education team and Digital Learning Board (DLB) to achieve this aim. Activities have included the following: a) Creation of a BSR hosted Teams channel to store PAR specific educational information; b) PAR trainee inclusion on the BSR DLB, which creates digital education resources including podcasts and the monthly blended learning Spotlight topic series; c) Creation of PAR focused podcasts within the BSR Talking Rheumatology podcast series; d) Organisation of webinars on PAR relevant topics; e) Creation of a PAR podcast playlist on the Spotify platform; f) Production of a resource list and education newsletter to signpost relevant existing/upcoming learning opportunities and events. Results A 2023 follow up survey of the PAR trainee group (n = 16 respondents), showed the most used resources were podcasts (69%), the newsletter (63%) and resource list (43%). Webinars (38%), the Teams channel (38%) and the BSR Spotlight series (31%) had been accessed less. BSR podcast download data illustrated that PAR relevant podcasts from the Talking Rheumatology/ Talking Rheumatology Spotlight series have a UK and international audience. There have been over 2500 downloads from six PAR relevant podcasts covering myositis guidelines, biologics in PAR, HLH, antiphospholipid syndrome and ultrasound in PAR. Feedback from those who have used the various educational resources has been extremely positive, although comments have indicated a barrier in terms of awareness of the materials and how to access them. Conclusion Good progress has been made, however maximising means and opportunities to disseminate available resources within the PAR community is vital. Work to develop, collate and signpost more PAR educational content is ongoing. A project is in progress to create condition-specific lists of classification criteria, guidelines and key studies. The PAR trainee group are very grateful to all of those who have supported this work including the professionals who have shared their knowledge and expertise via podcasts and webinars, members of the BSR DLB and the education team at BSR. Disclosure J. Lemon: Other; Unpaid member of the BSR Digital Learning Board. R. Close: None. G. Dobson: None. K. Maxey: Other; Works for the BSR as Events and Education Officer. J. May: None. D. McLaughlin: None. I. Rajarathinam: None. S. Saadalla: None. L. Yeo: None. P.A. Watson: Other; Works for the BSR in a paid role (2PA per week ) as digital learning editor.

An “On Demand” canakinumab regimen for treating children with Colchicine-Resistant familial Mediterranean fever – A multicentre study

ObjectivesCanakinumab, a human monoclonal antibody targeted at interleukin-1 beta, has demonstrated safety and efficacy in preventing familial Mediterranean fever (FMF) attacks among individuals with colchicine-resistant (crFMF). The manufacturer orders prescribe monthly subcutaneous injections. However, a subset of our patients is treated with an “canakinumab on demand ” (COD) strategy, with wider intervals between drug administrations. Therefore, we aimed to compare disease activity and drug safety between COD and “canakinumab fixed frequency” (CFF) policies. MethodsThis retrospective study collected data from three Israeli paediatric rheumatology centres, of children with crFMF who were treated with canakinumab. Epidemiological and clinical parameters, cumulative drug dosages, and adverse events were compared between children treated by both policies. ResultsTwenty-five (49 %) children were treated according to COD policy and 26 according to CFF policy. Demographic parameters and most of the disease features did not differ significantly between the groups. Both groups showed significant reduction in attacks after canakinumab introduction. The median number (interquartile range) of attacks per month did not differ significantly between the COD and CFF groups (0.33 (0.08, 0.58) and 0.13 (0, 0.5), respectively, p = 0.485 (even though, per definition, COD patients presumably had an attack before receiving the second canakinumab dose). The mean monthly dose was lower for the COD than the CFF group (1.13 ± 1.13 vs. 3.16 ± 1.46 mg/kg, p < 0.001). Adverse events were similar between the groups. ConclusionFor individuals with crFMF, COD compared to CFF policy can achieve similar efficacy and safety, with a lower accumulated canakinumab dose, rendering it less immunosuppressive and less expensive.

Economic evaluation of a trial exploring the effects of a web-based support tool for parents of children with juvenile idiopathic arthritis.

To explore the cost-effectiveness of a web-based support tool for parents of children with Juvenile idiopathic arthritis. A multi-centred randomized controlled trial was conducted in paediatric rheumatology centres in England. The WebParC intervention consisted of online information about JIA and its treatment and a toolkit using cognitive-behavioural therapy principles to support parents manage their child's JIA. An economic evaluation was performed alongside the trial involving 220 parents. The primary outcome was the self-report Pediatric Inventory for Parents measure of illness-related parenting stress, with two dimensions: difficulty and frequency. These measures along with costs were assessed post intervention at 4 and 12 months. Costs were calculated for healthcare usage using a UK NHS economic perspective. Data was collected and analysed on the impact of caring costs on families. Uncertainty around cost-effectiveness was explored using bootstrapping and cost-effectiveness acceptability curves. The intervention arm showed improved average Pediatric Inventory for Parents scores for the dimensions of frequency and difficulty, of 1.5 and 3.6 respectively at 4 months and 0.35 and 0.39 at 12 months, representing improved PIP scores for the intervention arm. At both 4 and 12 month follow-up, the average total cost per case was higher in the control group when compared with the intervention arm with mean differences of £360 (95% CI £29.6 to £691) at 4 months and £203 (95% CI £16 to £390) at 12 months. The probability of the intervention being cost-effective ranged between 49% and 54%. The WebParC intervention led to reductions in primary and secondary healthcare resource use and costs at 4 and 12 months. The intervention demonstrated particular savings for rheumatology services at both follow-ups. Future economies of scale could be realised by health providers with increased opportunities for cost-effectiveness over time. ISRCTN, ISRCTN13159730.

Macrophage activation syndrome in patients with systemic juvenile idiopathic arthritis on anti-interleukin-1 or -6 therapy.

The aim of this study is to investigate the effect of anti-interleukin (IL)-1/-6 biologics on systemic juvenile idiopathic arthritis (sJIA)-associated macrophage activation syndrome (MAS). Demographic, clinical, and laboratory data of patients followed up with a diagnosis of sJIA-associated MAS assessed from sixteen pediatric rheumatology centers across the country. The clinical and laboratory features of MAS developing while on biological drugs were compared with those without this treatment. One hundred and sixty-two patients were included in the study. 45 of the MAS events were detected under the effect of anti-IL-1/-6 biologics, while the patients experiencing the remaining 155 events have not received biological treatment in the last three months. Platelet count [128 (72-232) vs 199 (130-371) 109/l], ferritin level on admission [1107 (676-2050) vs 2863 (1193-9562) ng/ml], C-reactive protein level [15.4 (2.9-56) vs 90 (32-160) mg/l], erythrocyte sedimentation rate [13 (3-36) vs 43.5 (13-77) mm/h] and fever duration [5 (4-7.5) vs 10 (7-14.3) days] were found lower in the group under the impact of anti-IL-1/-6 biologics. Among patients treated with biologics, 26.6% did not meet the published 2016 MAS classification criteria at presentation. The rates of hepatomegaly and splenomegaly were relatively lower in the canakinumab-treated group when compared with those receiving other biologicals or to patients, not on biologicals. Anti-IL-1/-6 therapies can mask the clinical and laboratory features of MAS, and proposed guidelines for MAS classification criteria may not be met.

Transition From Pediatric to Adult Rheumatology Care: An Exploratory Study From Latin America.

Adequate transition from pediatric to adult care is associated with better adherence to treatment and better outcomes in pediatric patients with chronic diseases. There are little data on transition programs, outcomes, use of transition guidelines, and available tools in pediatric rheumatology centers from Latin America (LATAM). In this study, we described the characteristics of transition programs from 3 pediatric rheumatology centers. We also introduced results of the first survey examining the transition experience in countries from LATAM. The experience and implementation process of transition programs from 3 pediatric rheumatology centers were described. A survey based on a questionnaire created by Chira et al (J Rheumatol. 2014;41:768-779) from the Childhood Arthritis and Rheumatology Research Alliance was also administrated to pediatric rheumatology centers from LATAM. A total of 49 (68%) pediatric rheumatologists answered the survey. Most centers do not have an official and written transition program and reported a need for more tools and resources in their services to facilitate the transition experience. Transition guidelines culturally tailored to developing countries are needed in LATAM.

A rare case of severe isolated temporomandibular joint involvement in juvenile idiopathic arthritis

Background: Juvenile idiopathic arthritis is an inflammatory condition of unknown etiology. Early temporomandibular joint involvement may be asymptomatic or with mild symptomatology, despite radiological signs of substantial joint damage. Early diagnosis and management is of substantial importance, because orofacial symptoms due to temporomandibular joint arthritis may be disabling and interfere with impaired quality of life or even dentofacial abnormalities. When temporomandibular joint involvement is the first and only manifestation - a very rare event – it is easy to remain undiagnosed. It is noteworthy that the current body of published material on isolated arthritis of the temporomandibular joint is limited to 2 case reports and a multinational cohort of pediatric rheumatology centers.

Clinical characteristics and predictors for recurrence in chronic nonbacterial osteomyelitis: a retrospective multicenter analysis.

Chronic nonbacterial osteomyelitis (CNO) is a rare disease of unknown etiology and most commonly occurs during childhood or adolescence. The purpose of this study is to collect data on the clinical features, outcomes, and management of the disease and to identify the factors affecting recurrence. This is a retrospective multicenter cross-sectional study of pediatric patients diagnosed with CNO. A total of 87 patients with a diagnosis of CNO followed for at least 6 months in 8 pediatric rheumatology centers across the country between January 2010 and December 2021 were included in this study. The study included 87 patients (38 girls, 49 boys; median age: 12.5 years). The median follow-up time was 20 months (IQR: 8.5-40). The median time of diagnostic delay was 9.9 months (IQR: 3-24). Arthralgia and bone pain were the most common presenting symptoms. Multifocal involvement was detected in 86.2% of the cases and a recurrent course was reported in one-third of those included in the study. The most commonly involved bones were the femur and tibia. Vertebrae and clavicles were affected in 19.5% and 20.6% of cases, respectively. The erythrocyte sedimentation rate (ESR) values of 60.9% of the patients were above 20 mm/h and the C-reactive protein values of 44.8% were above 5 mg/L. The remission rate was 13.3% in patients using nonsteroidal antiinflammatory drugs and 75.0% in those using biological drugs. Vertebral and mandibular involvement and high ESR values at the time of diagnosis were associated with recurrence. In this multicenter study, CNO with vertebral and mandibular involvement and high ESR at diagnosis were associated with recurrence.

Majority of new patient referrals to a large pediatric rheumatology center result in non-rheumatic diagnosis

ObjectivePediatric rheumatology faces a looming supply-demand crisis. While strategies have been proposed to address the supply shortfall, investigation into the increased demand for pediatric rheumatic care has been limited. Herein, we analyze new patient visits to a large tertiary care pediatric rheumatology center to identify emerging trends in referrals and areas for potential intervention to meet this increased demand.MethodsAll patients referred to and seen by the University of Alabama at Birmingham Pediatric Rheumatology Division between January 2019 and December 2021 for a new patient evaluation were identified. Patient data was retrospectively abstracted, de-identified, and analyzed to develop trends in referrals and frequency of rheumatic disease, non-rheumatic disease, and specific diagnoses.ResultsDuring the study period, 2638 patients were referred to and seen in by the pediatric rheumatology division. Six hundred and ten patients (23.1%) were diagnosed with rheumatic disease. The most common rheumatic disease was juvenile idiopathic arthritis (JIA) at 45.6%, followed by primary Raynaud phenomenon (7.4%), recurrent fever syndromes (6.9%), vasculitides (6.7%), and inflammatory eye disease (6.2%). Of the 2028 patients (76.9%) diagnosed with a non-rheumatic condition, benign musculoskeletal pain was the most common (61.8%), followed by a combination of somatic conditions (11.6%), and non-inflammatory rash (7.7%).ConclusionIn this analysis of new patient referrals to a large pediatric rheumatology center, the majority of patients were diagnosed with a non-rheumatic condition. As a worsening supply-demand gap threatens the field of pediatric rheumatology, increased emphasis should be placed on reducing non-rheumatic disease referrals.