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Mental health problems are more common in children with pediatric rheumatologic diseases (PRDs) than healthy peers. Mental health problems affect disease-related outcomes and health-related quality of life (HRQOL), so addressing these problems can improve clinical and psychosocial outcomes. Mental health screening tools are available, and there are resources available to aid in integrating mental health care into the clinical setting. By implementing these tools, mental health problems can be recognized and addressed.

Implementation science is the study of processes that promote reliable uptake of evidence-based practices into clinical care. The integration of implementation science and health disparities research approaches has been proposed as a method to reduce health inequity through detection, understanding, and implementation of health equity-focused interventions. In this review, we provide an argument for the study of implementation science in pediatric rheumatology in light of previously observed health disparities, present a framework for the study of health equity and implementation science in pediatric rheumatology, and propose next steps to accelerate action.

The advent of social media has revolutionized and empowered communities of people living with rare diseases. Social media has enabled families of children with similar pediatric rheumatic diseases (PRDs) to meet regardless of geographic distance, allowing them to support each other and join together to advocate on behalf of their children. Researchers have also leveraged social media to learn about the health of patients and their communities. In this manuscript, we review the promises and perils of social media related to health, focusing on its potential use to support research and care of children with PRD.

Pediatric rheumatology subspecialists treat chronic autoimmune diseases with onset in childhood. Prompt diagnosis and ongoing management of these conditions are imperative to prevent damage from ongoing inflammation. Here, we aim to describe the current landscape of pediatric rheumatic disease in lower to middle-income countries (LMICs) and explore current barriers to understanding global disease burden. We then examine innovative strategies to promote a more equitable future for children and young people living with rheumatic diseases worldwide.

The Childhood Arthritis & Rheumatology Research Alliance (CARRA) launched in 2000 as a small network of pediatric rheumatologists and investigators dedicated to promoting collaborative research to improve the care and outcomes of childhood-onset rheumatic diseases. Over the past 2 decades, CARRA has grown to become a major driver of advances in evidence-based medicine and career development in pediatric rheumatology. Its research approach has transformed pediatric rheumatology. CARRA is a vibrant organization that will continue to facilitate impactful research in the care of children, adolescents, and young adults with rheumatic disease in the years to come.

Background: Down Syndrome (DS) is one of the most common birth conditions in the United States of America, with approximately 5300 births annually, resulting in an estimated birth prevalence of 12.6 per 10,000 live births and a population prevalence in the USA since 2010 of 6.7 per 10,000 inhabitants. Children with DS have complex medical challenges that present due to changes in their immune system that results in increased rates of infection, malignancy, and autoimmune disease. Juvenile Dermatomyositis (JDM) is a rare, autoimmune disease, and the most common inflammatory myopathy of childhood. Reports suggest an increased incidence of arthritis in children with DS, but there have been no reports of JDM in children with DS. Additionally, those with DS pose unique challenges with an increase in adverse effects and ineffectiveness of immunosuppressive therapy. Case : We describe the first case of an adolescent female with DS who developed JDM with a positive anti-p155/140 antibody and characteristic clinical phenotype. We discuss increased awareness of autoimmune disease in adolescents with DS and clinical considerations for therapy with immunosuppression. Conclusion: Adolescents with DS are at increased risk for autoimmune disease, including JDM. Awareness, early recognition of the signs and symptoms of autoimmune disease in those with DS is important, so appropriate therapy can be implemented.

Objective The optimal time to start biologics in polyarticular juvenile idiopathic arthritis (JIA) remains uncertain. The Childhood Arthritis and Rheumatology Research Alliance (CARRA) developed 3 consensus treatment plans (CTPs) for untreated polyarticular JIA to compare strategies for starting biologics. Methods Start Time Optimization of Biologics in Polyarticular JIA (STOP‐JIA) was a prospective, observational, CARRA Registry study comparing the effectiveness of 3 CTPs: 1) the step‐up plan (initial nonbiologic disease‐modifying antirheumatic drug [DMARD] monotherapy, adding a biologic if needed, 2) the early combination plan (DMARD and biologic started together), and 3) the biologic first plan (biologic monotherapy). The primary outcome measure was clinically inactive disease according to the provisional American College of Rheumatology (ACR) criteria, without glucocorticoids, at 12 months. Secondary outcome measures included Patient‐Reported Outcomes Measurement Information System (PROMIS) pain interference and mobility scores, inactive disease as defined by the clinical Juvenile Arthritis Disease Activity Score in 10 joints (JADAS‐10), and the ACR Pediatric 70 criteria (Pedi 70). Results Of 400 patients enrolled, 257 (64%) began the step‐up plan, 100 (25%) the early combination plan, and 43 (11%) the biologic first plan. After propensity score weighting and multiple imputation, clinically inactive disease according to the ACR criteria was achieved in 37% of those on the early combination plan, 32% on the step‐up plan, and 24% on the biologic first plan (P = 0.17). Inactive disease according to the clinical JADAS‐10 (score ≤2.5) was also achieved in more patients on the early combination plan than the step‐up plan (59% versus 43%; P = 0.03), as was ACR Pedi 70 (81% versus 62%; P = 0.008), but generalizability was limited by missing data. PROMIS measures improved in all groups, but without significant differences. Twenty serious adverse events were reported (mostly infections). Conclusion Achievement of clinically inactive disease without glucocorticoids did not significantly differ between groups at 12 months. While there was a significantly higher likelihood of early combination therapy achieving inactive disease according to the clinical JADAS‐10 and ACR Pedi 70, these results require further exploration.

OBJECTIVE To study disease course and long-term outcome in children with linear scleroderma (SSc) treated with methotrexate (MTX) since diagnosis. METHODS The present study was retrospective and cross-sectional and included consecutive children with linear SSc who were treated with MTX for >1 year and were followed up for at least 2 years. Disease course was analyzed by the number of relapses and treatment changes. Relapse-free survival was examined by Kaplan-Meier analysis, comparing patients with linear SSc and those with other juvenile localized scleroderma (JLS) disease subtypes. Disease activity and damage were assessed by the Localized Scleroderma Cutaneous Assessment Tool and thermography. RESULTS Fifty patients with a mean follow-up duration of 7.8 years and a mean MTX treatment duration of 3.1 years were included. Sixteen percent of patients did not respond to the first course of MTX, and 16% had at least 1 flare. Complete remission was observed in 18.2% of patients who were followed up for 2-5 years, in 80.0% of patients followed up for 10 years, and in 87.5% of patients followed up for >10 years. No significant difference in relapse-free survival between patients with linear SSc and in 17 patients with other JLS disease subtypes was observed. Tissue damage was mild in 42% of patients, moderate in 32%, and severe in 26%. The correlations between severity of tissue damage and linear SSc subtype, disease duration, relapses, and remission were not significant. The relationships between treatment duration and disease relapses (P < 0.05) and severity of tissue damage (P < 0.005) were significant. CONCLUSION Most patients with linear SSc who are treated with MTX achieve complete and long-lasting remission. Overall aesthetic and functional sequelae are moderate, most likely because tissue damage is established early and treatment likely stabilizes the damage. Early diagnosis and MTX treatment, as well as long-term monitoring, are crucial to improve outcome and promptly identify flares.

Introduction The search for biomarkers in juvenile idiopathic arthritis (JIA) is a promising and rapidly expanding field of investigation. The biomarkers identified so far may help to dissect the clinical heterogeneity of the illness, measure the level of disease activity, predict clinical remission, relapse, response to medications, course over time, complications, and forestall disease flares. Areas covered We provide a summary of the most recent advances in the development and application of biomarkers in JIA. We performed a PubMed search for significant articles combining predetermined keywords related to biomarkers in non-systemic and systemic JIA, chronic uveitis, and macrophage activation syndrome (MAS).The biomarkers available or under study are presented and discussed separately for non-systemic and systemic subtypes and for the two main disease complications, uveitis and MAS. Expert opinion The incorporation of valid and reliable biomarkers in standard clinical care may help to design better patient-tailored treatment regimens and to improve the therapeutic strategies based on the treat-to-target approach. The establishment of biomarkers that predict the risk of disease flare may lead to define the optimal modalities for treatment discontinuation after the achievement of clinical remission.

Lupus nephritis (LN) is a life-threatening manifestation of systemic lupus erythematosus (SLE) and is more common in children than adults. The epidemiology and management of childhood-onset SLE (cSLE) have changed over time, prompting the need to reassess expected outcomes. The purpose of this study is to use the Childhood Arthritis and Rheumatology Research Alliance (CARRA) prospective registry to validate historical principles of LN in a contemporary, real-world cohort. After an extensive literature review, six principles of LN in cSLE were identified. The CARRA registry was queried to evaluate these principles in determining the rate of LN in cSLE, median time from cSLE diagnosis to LN, short-term renal outcomes, and frequency of rituximab as an induction therapy. Of the 677 cSLE patients in the CARRA registry, 32% had documented LN. Decline in kidney function was more common in Black cSLE patients than non-Black patients ( p = 0.04). Black race was associated with worse short-term renal outcomes. In short-term follow up, most children with LN had unchanged or improved kidney function, and end stage kidney disease (ESKD) was rare. Ongoing follow-up of cSLE patients in the CARRA registry will be necessary to evaluate long-term outcomes to inform risk, management, and prognosis of LN in cSLE.

Background The transition of health care from Pediatric to Adult providers for adolescents and young adults with chronic disease is associated with poor outcomes. Despite the importance of this transition, over 80% of these patients do not receive the services necessary to transition to Adult health care. In 2018, we initiated a transition clinic structure, integrating an Internal Medicine - Pediatrics trained Adult Rheumatologist in a Pediatric Rheumatology clinic to guide this transition. Our goal was to improve transition outcomes. We report the methods of this clinic and its preliminary outcomes. Methods For patients referred to the transition clinic, the Adult Rheumatologist assumed medical management and implemented a six-part modular transition curriculum. This curriculum included a Transition Policy, Transition Readiness Assessment, medication review and education, diagnosis review and education, and counseling on differences between Pediatric and Adult-oriented care. Eligible patients and their families were enrolled in a prospective observational outcomes research registry. Initial data from this transition clinic is reported including adherence with certain aspects of the transition curriculum and clinic utilization. Results The transition clinic Adult Rheumatologist saw 177 patients in 2 years, and 57 patients were eligible for, approached, and successfully enrolled in the registry. From this registry, all patients reviewed the Transition Policy with the Adult Rheumatologist and 45 (78.9%) completed at least one Transition Readiness Assessment. Of the 22 patients for whom transition was indicated, all were successfully transitioned to an Adult Rheumatologist. 17 (77.3%) continued care post-transition with the transition clinic Adult Rheumatologist, and 5 (22.7%) continued care post-transition with a different Adult Rheumatologist. The median time between the last transition clinic visit and first Adult clinic visit was 5.1 months. Conclusions Our experience demonstrated the success of our clinic model regarding participation in the transition curriculum and improved clinic utilization data. Our results are an improvement over transition rates reported elsewhere that did not implement our model. We believe that this structure could be applied to other primary care and subspecialty clinics. Trial registration This research was approved by the University of Utah Institutional Review Board (IRB) in January 2019 (IRB_00115964). Patients were retrospectively registered if involved prior to this date.

PURPOSE OF REVIEW: The purpose of this review is to discuss the clinical management of children with pediatric rheumatic disease (PRD) during the Coronavirus disease of 2019 (COVID-19) pandemic, as well as the unique role of the pediatric rheumatologist during a time of emerging post-COVID inflammatory sequelae including, multisystem inflammatory syndrome in children (MIS-C). RECENT FINDINGS: To date, there has been little evidence to suggest that children with PRD, including those on immunomodulatory therapies, are at increased risk for severe COVID-19. Clinical guidance statements have been created to support clinical providers in providing care to children with PRD during the COVID-19 pandemic. Pediatric rheumatologists have also been called upon to assist in the identification and management of post-COVID sequelae, including the rapidly emerging inflammatory illness, MIS-C. SUMMARY: The COVID-19 era has been defined by a rapid expansion in scientific knowledge and a time of extraordinary local and worldwide collaboration, both within the pediatric rheumatology community, as well as across multiple disciplines. Through collective efforts, we have learned that children with PRD, including those on immunomodulatory therapies, are not at increased risk for severe COVID-19. Pediatric rheumatologists have also worked alongside other disciplines to develop guidance for the management of MIS-C, with the majority of patients experiencing excellent clinical outcomes.

Background: To explore the long-term safety and dynamics of the immune response induced by the second and third doses of the BNT162b2 mRNA COVID-19 vaccine in adolescents with juvenile-onset autoimmune inflammatory rheumatic diseases (AIIRDs) compared with healthy controls. Methods: This international prospective study included adolescents with AIIRDs and controls vaccinated with two (AIIRDs n = 124; controls n = 80) or three (AIIRDs n = 64; controls n = 30) doses of the BNT162b2 vaccine, evaluated for vaccine side-effects, disease activity, COVID-19 breakthrough infection rates and severity, and anti-spike S1/S2 IgG antibody titers in a sample from both groups. Results: The vaccination safety profile was favorable, with most patients reporting mild or no side-effects. The rheumatic disease remained stable at 98% and 100% after the second and third doses, respectively. The two-dose vaccine induced comparable seropositivity rates among patients (91%) and controls (100%), (p = 0.55), which declined within 6 months to 87% and 100%, respectively (p = 0.3) and increased to 100% in both groups after the third vaccine dose. The overall post-vaccination COVID-19 infection rate was comparable between patients and controls, 47.6% (n = 59) and 35% (n = 28), respectively; p = 0.5278, with most infections occurring during the Omicron surge. In relation to the last vaccination, time-to-COVID-19 infection was similar between patients and controls, at a median of 5.5 vs. 5.2 months, respectively (log-rank p = 0.1555). Conclusion: The safety profile of three doses of the BNT162b2 mRNA vaccine was excellent, with adequate humoral response and similar efficacy among patients and controls. These results support the recommendation for vaccinating adolescents with juvenile-onset AIIRDs against COVID-19.

Small, noncoding sequences of ribonucleic acid called microRNAs (miRNAs, miR) are functioning as posttranscriptional regulators of gene expression. As they draw increasing attention of rheumatologists, there is a growing body of evidence concerning specific molecules that may affect the long-term care of patients with inflammatory arthritides. Findings involving children with juvenile idiopathic arthritis (JIA) are still limited though. The aim of the study was to browse the available data on microRNAs which may be utilized as potential biomarkers helpful in diagnosing and monitoring JIA patients. The review contains a brief summary on the most studied sequences: miR-16, miR-125a-5p, miR-146a, miR-155, and miR-223. It is complemented with other miRNAs possibly relevant for JIA (miR-145, miR-23b, miR-27a, and miR-204) and discussion on challenges for using miRNAs in pediatric rheumatology (particularly, issues regarding specificity of biomarkers and measurements involving synovial fluid).

Introduction Transition is a planned process of pediatric patients from child-centered to adult-oriented treatment. Transitional care for patients with chronic diseases is essential. The present study aimed to evaluate the readiness of patients with rheumatic diseases and their parents for transition process. Method This is a cross-sectional, single-center study. All patients and their parents were questioned about their awareness of and willingness to undergo transitional care. Transition Readiness Assessment Questionnaire (TRAQ) was applied to all the participants. TRAQ is a tool for measuring readiness for transitional care in adolescents with chronic diseases. TRAQ includes 20 items that are divided into 2 domains: self-management and self-advocacy. Results A total of 157 (87 girls/70 boys) patients and their parents were enrolled. Of them 64 were diagnosed with familial Mediterranean fever, 52 with juvenile idiopathic arthritis, 21 with systemic lupus erythematosus, and 20 with Behcet's disease. The median age of the patients was 16 years (15-18). However, all patients and parents accepted that transition to adult-oriented care is necessary; only one-third of them were aware about transitional care. Eighty (50.9%) patients and 147 (93.6%) of the parents stated that they were wishing to continue pediatric rheumatology treatment. The mean TRAQ self-management domain and self-advocacy domain total scores in the patients were 1.76 ± 0.51 and 1.72 ± 0.49, respectively (P = .48). The mean TRAQ total score was not different between patients and parents. When we assessed the factors affecting transition process, the TRAQ score was lower among patients with active disease, and requiring hospitalization during the previous year. Conclusion Assessment od the readiness of patients with chronic rheumatic diseases for transition care will increase the awareness of patients and their parents as well, and provide determination of the optimal time for transition.

ObjectiveMental health problems are prevalent in youth with rheumatologic disease. Gaps in knowledge exist regarding their effect, as well as strategies for detection and effective treatment. To address these gaps, the Childhood Arthritis and Rheumatology Research Alliance (CARRA) Mental Health Workgroup developed and prioritized an agenda of research topics.MethodsWe systematically reviewed the literature and identified 5 major research domains in further need of study: (A) mental health burden and relationship to pediatric rheumatologic disease, (B) effect of mental health disorders on outcomes, (C) mental health awareness and education, (D) mental health screening, and (E) mental health treatment. Research topics within these areas were developed by workgroup leaders and refined by the workgroup. Members were surveyed to prioritize the topics by importance, feasibility of study, and actionability.ResultsFifty-nine members (57%) completed the survey. Among the proposed research topics, 31/33 were rated as highly important and 4/33 were rated highly for importance, feasibility, and actionability. Topics rated most important related to (A) mental health burden and relationship to rheumatologic disease, and (B) the effect of mental health on outcomes. Topics rated most feasible and actionable were related to (D) mental health screening.ConclusionAddressing gaps in knowledge regarding mental health in youth with rheumatologic disease is essential for improving care. We have identified high priority research topics regarding mental health of pediatric rheumatology patients in need of further investigation that are feasible to study and believed to lead to actionable results in patient care.

Background During the Coronavirus disease 2019 pandemic, ambulatory pediatric rheumatology healthcare rapidly transformed to a mainly telehealth model. However, pediatric patient and caregiver satisfaction with broadly deployed telehealth programs remains largely unknown. This study aimed to evaluate patient/caregiver satisfaction with telehealth and identify the factors associated with satisfaction in a generalizable sample of pediatric rheumatology patients. Methods Patients with an initial telehealth video visit with a rheumatology provider between April and June 2020 were eligible. All patients/caregivers were sent a post-visit survey to assess a modified version of the Telehealth Usability Questionnaire (TUQ) and demographic and clinical characteristics. TUQ total and sub-scale (usefulness, ease of use, effectiveness, satisfaction) scores were calculated and classified as “positive” based on responses of “agree” or “strongly agree” on a 5-point Likert scale. Results were analyzed using standard descriptive statistics and Wilcoxon signed rank testing. The association between demographic and clinical characteristics with TUQ scores was assessed using univariate linear regression. Results 597 patients/caregivers met inclusion criteria, and the survey response rate was 42% (n = 248). Juvenile idiopathic arthritis was the most common diagnosis (33.5%). The majority of patients were diagnosed greater than 6 months previously (72.6%) and were prescribed chronic medications (59.7%). The median total TUQ score was 4 (IQR: 4–5) with positive responses in 81% of items. Of the subscales, usefulness scores were lowest (median: 4, p p Conclusions Our results suggest telehealth is a promising mode of healthcare delivery for pediatric rheumatic diseases but also identifies opportunities for improvement. Innovation and research are needed to design a telehealth system that delivers high quality and safe care that improves healthcare outcomes. Since telehealth is a rapidly emerging form of pediatric rheumatology care, improved engagement and training of patients, caregivers, and providers may help improve the patient experience in the future.

Patient engagement in pediatric rheumatology research can ensure protocol acceptability, project relevance, facilitate a successful study, and share results with the community. Research partners can collaborate along the entire continuum of research, contributing both lived and professional experience with roles as small as a single point-in-time consultation to as large as multi-year shared leadership. Patient and caregiver partners can be found in the myriad of places-the clinic, in advocacy organizations, on social media, and through networking with existing patient partners. Patient engagement begins with relationship building and requires effective training of both researcher and patient family.

The electronic health record (EHR) ecosystem is undergoing rapid evolution in response to new rules and regulations promulgated by the US HITECH Act (2009) and the 21st Century Cures Act (2016), which together promote and support enhanced information use, access, exchange, as well as vendor-agnostic application development. By leveraging emerging new standards and technology for EHR data interchange, for example, FHIR and SMART, pediatric rheumatology clinical care, research, and quality improvement communities will have the opportunity to streamline documentation workflows, integrate patient-reported outcomes into clinical care, reuse clinical data for research purposes, and embed implementation science approaches within the EHR.

A comprehensive review of reproductive health subtopics, including puberty, menarche, sexual orientation, gender identity, and gynecologic cancers as they pertain to patients with pediatric rheumatic diseases and those who care for them. Rheumatic disease medications and their effect on reproductive health across childhood and adolescence are also reviewed.

This article provides an in-depth review of telemedicine and its use in pediatric rheumatology. Historical barriers to the use of telemedicine in pediatric chronic care are described, and recent policy changes that have supported the use of telemedicine are discussed. Future directions and suggestions for the evaluation of telemedicine in pediatric rheumatology care are provided with a special focus on clinical outcomes, its use in research, patient acceptability, and health equity.

A comprehensive review of reproductive health subtopics, including sexual intercourse, romantic relationships, contraception, sexually transmitted infections, pregnancy, and infertility, as they pertain to patients with pediatric rheumatic diseases and those who care for them.

Advancements in eHealth offer unique opportunities for assisting in and augmenting aspects of evidence-based pain evaluation and management with children and adolescents. In this article, we present an overview of some of the opportunities and challenges in pain eHealth for pediatric rheumatologists to consider while caring for children and adolescents seen in their practice.

BackgroundThe incidence of systemic lupus in children with discoid lupus is unknown.ObjectiveThis study assessed the baseline characteristics of patients with pediatric discoid lupus erythematosus (pDLE).MethodsMedical records at 17 sites were reviewed for pediatric dermatology and rheumatology patients with discoid lupus erythematosus. The inclusion criteria were clinical and/or histopathologic diagnosis of discoid lupus erythematosus with an age at onset of 1500 charts reviewed, 438 patients met the inclusion criteria. The cohort was predominantly female (72%) and racially/ethnically diverse. A diagnosis of SLE at the baseline visit (pDLE+SLE) was rendered in 162 (37%) patients using the American College of Rheumatology and in 181 (41%) patients using the Systemic Lupus International Collaborating Clinics criteria. Patients with pDLE+SLE were older at the time of rash onset (median, 12.9 vs 8.9years; P&nbsp

Background Biotherapy result of spectacular advances in genetics and research in molecular and cellular biology, has considerably improved the management of rheumatic and autoinflammatory diseases in children. The Objective is to assess the efficacy and tolerance of biotherapy in pediatrics Methods A retro-prospective study was carried out in the Pediatric Department B at the pediatric rheumatology consultation, university hospital center. An operating sheet was drawn up including epidemiological, clinical, paraclinical and therapeutic data of the cases diagnosed as well as their evolution on biotherapy. Results 26 children received treatment with biotherapy. 73% presented with JIA (54% systemic, 19% polyarticular with positive RF, 19% severe uveitis, and 4% familial Mediterranean fever. 4% behçet disease The mean time to start biotherapy was 12 months [2–48]. There is a clear clinical and biological improvement for 93% of patients. Etanercept was effective in polyarticular-progressive JIA, Adalimumab in oligoarthritis with severe uveitis, Tocilizumab in systemic forms of JIA and Anakinra in familial Mediterranean fever resistant to colchicine and systemic arthritis. The adverse reactions noted were: one case of tuberculosis, hepatic cytolysis in 4 cases, and one case of psoriasis. Conclusion The introduction of biotherapy in pediatric has considerably improved the management of inflammatory rheumatic diseases and transformed the prognosis of these chronic diseases. The maintenance of the good response to treatment and the good tolerance of biotherapy must be evaluated prospectively over the long term.

The advent of biologic disease-modifying antirheumatic drugs targeting specific cytokines or cell-cell interactions has dramatically changed the outlook of patients with juvenile idiopathic arthritis. However, safety concerns remain around the use of therapeutic agents for children with juvenile idiopathic arthritis. Foremost among these are the risks of serious infections and malignancy. This article provides an overview of methodologies for pharmacosurveillance in juvenile idiopathic arthritis, including spontaneous reporting systems and the use of diverse data sources, such as electronic health records, administrative claims, and clinical registries. The risks of infections and malignancies are then briefly reviewed.

In order to improve the route to market for approved pediatric therapeutics, the current Pediatric Regulation in the EU and the Food and Drug Administration (FDA) Amendments Act (FDAAA) were both adopted in 2007..

Objective To assess anti–cytosolic 5′‐nucleotidase 1A (anti–cN‐1A) autoantibodies in children with juvenile dermatomyositis (DM) and healthy controls, using 3 different methods of antibody detection, as well as verification of the results in an independent cohort. Methods Anti–cN‐1A reactivity was assessed in 34 Dutch juvenile DM patients and 20 healthy juvenile controls using the following methods: a commercially available full‐length cN‐1A enzyme‐linked immunosorbent assay (ELISA), a synthetic peptide ELISA, and immunoblotting with a lysate from cN‐1A–expressing HEK 293 cells. Sera from juvenile DM patients with active disease and those with disease in remission were analyzed. An independent British cohort of 110 juvenile DM patients and 43 healthy juvenile controls was assessed using an in‐house full‐length cN‐1A ELISA. Results Anti–cN‐1A reactivity was not present in sera from juvenile DM patients or healthy controls when tested with the commercially available full‐length cN‐1A ELISA or by immunoblotting, in either active disease or disease in remission. Additionally, in the British juvenile DM cohort, anti–cN‐1A reactivity was not detected. Three Dutch juvenile DM patients had weakly positive results for 1 of 3 synthetic cN‐1A peptides measured by ELISA. Conclusion Juvenile DM patients and young healthy individuals did not show anti–cN‐1A reactivity as assessed by different antibody detection techniques.

Objective To assess the first-year features of patients with chronic nonbacterial osteomyelitis (CNO). Methods Patients with a diagnosis of CNO, disease duration of under 13 months, and first registration in the German National Pediatric Rheumatologic Database (NPRD) between 2009 and 2018 were included in this cross-sectional analysis. Results Of 774 documented patients, 62.8% were female, and all patients had a median age of 11 years. The most affected clinical sites were the tibia (29.7%), pelvis (28.0%), and femur (27.8%). HLA-B27 was positive in 48 of 314 analyzed patients (15.3%). In 406 patients, an X-ray was performed at the first visit; X-ray results showed osteosclerosis/−lysis in 34.0% and hyperostosis in 14.5% of the patients. MRI scans (focal and whole-body scans) were performed in 648 patients, and 81.5% showed a positive TIRM/STIR signal. A total of 84.7% of the patients were administered nonsteroidal anti-inflammatory drugs, 9.6% were administered oral glucocorticoids, 10.8% were administered disease-modifying anti-rheumatic drugs (DMARDs), and 6.1% were administered bisphosphonates. An evaluation of the patient’s questionnaire showed an overall well-being (NRS 0–10) of 2.0. The PedCNO disease “activity” score revealed a 70% improvement in variables in 43% of patients in the initial 1-year follow-up. Copresentation with diagnostic criteria of pediatric enthesitis-related arthritis was rare. Conclusion To our knowledge, the NPRD cohort seemed to be the largest cohort of children and adolescents suffering from CNO worldwide. Most patients were treated effectively with NSAIDs, and only a small group of patients was administered additional medication. The patient-defined measures of disease activity had a moderate impact on patients’ daily lives. Trial registration Not applicable.

To define the spectrum and phenotypic characteristics of systemic autoinflammatory diseases (SAIDs) other than familial Mediterranean fever (FMF) in Arab children and to delineate diagnostic evaluation. Data retrospectively collected on patients with clinical and/or genetically proven SAIDs other than FMF at 10 tertiary Arab pediatric rheumatology clinics from 1990 to 2018. The collected data comprised the clinical findings and diagnostic evaluation including genetic testing, the provided treatment and the accrual damage related to SAIDs. A total of 144 patients (93 female) with a median age at onset of 2.5 (range 0.1-12) years were enrolled. The initial diagnosis was inaccurate in 49.3%. Consanguinity rate among parents was 74.6%. The median time-to-diagnosis for all SAIDs was 2.5 (range 0.1-10) years. There were 104 patients (72.2%) with a confirmed diagnosis and 40 patients with suspected SAIDs. Seventy-two had monogenic and 66 patients with multifactorial SAIDs while six patients had undifferentiated SAIDs. The most frequent monogenic SAIDs were LACC1 mediated monogenic disorders (n = 23) followed by CAPS (12), TRAPS (12), HIDS (12), and Majeed's syndrome (6). The most frequent multifactorial SAIDs was CRMO (34), followed by PFAPA (18), and early onset sarcoidosis (EOS) (14). Genetic analysis was performed in 69 patients; 50 patients had genetically confirmed disease. Corticosteroid used for 93 patients while biologic agents for 96 patients. Overall, growth failure was the most frequent accrual damage (36%), followed by cognitive impairment (13%). There were three deaths because of infection. This study shows a heterogenous spectrum of SAIDs with a high number of genetically confirmed monogenic diseases; notably, LACC1 associated diseases. Hopefully, this work will be the first step for a prospective registry for SAIDs in Arab countries.

Objective: To develop a questionnaire that allows the early detection of patients at risk for poor adherence to medical and non-medical treatment in children and adolescents with chronic rheumatic diseases. Methods: The Pediatric Rheumatology Adherence Questionnaire (PRAQ) was applied in recently diagnosed patients within a period of one to four months after confirmation of the rheumatic disease. After six months, the patients’ adherence to the medical and non-medical treatment was assessed. An internal consistency analysis was conducted to eliminate redundant questions in the PRAQ. Results: A total of 33 patients were included in the pilot study. Six months after the PRAQ had been applied, poor global adherence was observed in seven (21.2%) patients and poor adherence to medical treatment in eight (24.2%) patients. No correlation was observed between the PRAQ scores and the percentages of adherence, as well as the stratification for each index, except for a tendency to a correlation between socioeconomic index and poor adherence to medical treatment (p=0.08). A new PRAQ questionnaire with 25 of the 46 original questions was generated as a result of the reliability analysis. Conclusions: The usefulness of this questionnaire in clinical practice should be still evaluated. Due to the importance of a tool for the early detection of rheumatic patients at risk of poor adherence to treatment, the new PRAQ questionnaire should be reviewed and applied in a larger study to better define its validity and reliability. RESUMO Objetivo: Desenvolver um questionário que permitisse a detecção precoce de pacientes em risco de má adesão ao tratamento medicamentoso e não medicamentoso para crianças e adolescentes com doenças reumáticas crônicas. Métodos: O Questionário de Adesão em Reumatologia Pediátrica (QARP) foi aplicado em pacientes recentemente diagnosticados, de um a quatro meses após a confirmação da doença reumática. Seis meses depois, foi avaliada a adesão do paciente ao tratamento medicamentoso e não medicamentoso. Foi realizada uma análise de consistência interna para eliminar questões redundantes no QARP. Resultados: Ao todo, 33 pacientes foram incluídos no estudo-piloto. Seis meses após a aplicação do QARP, observou-se baixa adesão global em sete pacientes (21,2%) e baixa adesão ao tratamento medicamentoso em oito (24,2%) pacientes. Não foi encontrada correlação entre os escores QARP e as porcentagens de adesão, bem como a estratificação para cada indicador, com exceção de uma tendência a uma correlação entre o indicador socioeconômico e a baixa adesão ao tratamento medicamentoso (p=0,08). Um novo questionário QARP com 25 das 46 perguntas originais foi gerado como resultado da análise de confiabilidade. Conclusões: A utilidade deste questionário na prática clínica ainda deve ser avaliada. Devido à importância de uma ferramenta para a detecção precoce de pacientes reumáticos em risco de má adesão ao tratamento, o novo questionário QARP deve ser revisado e aplicado em um estudo maior para que sua validade e confiabilidade sejam mais bem definidas.

Objective Evidence suggests that aberrant function of innate lymphoid cells (ILCs), whose functional and transcriptional profiles overlap with those of Th cell subsets, contributes to immune‐mediated pathologies. To date, analysis of juvenile idiopathic arthritis (JIA) immune pathology has concentrated on the contribution of CD4+ T cells; we have previously identified an expansion of Th17 cells within the synovial fluid (SF) of JIA patients. We undertook this study to extend this analysis to further investigate the role of ILCs and other interleukin‐17 (IL‐17)–producing T cell subsets in JIA. Methods ILCs and CD3+ T cell subsets were defined in peripheral blood mononuclear cells (PBMCs) from healthy adults, healthy children, and JIA patients and in SF mononuclear cells (SFMCs) from JIA patients using flow cytometry. Defined subsets in SFMCs were correlated with clinical measures including physician's global assessment of disease activity on a visual analog scale, number of joints with active disease, and erythrocyte sedimentation rate. Transcription factor and cytokine profiles of sorted ILCs were assessed by quantitative reverse transcriptase–polymerase chain reaction. Results Group 1 ILCs (ILC1s), NKp44− group 3 ILCs (natural cytotoxicity receptor–negative [NCR−] ILC3s), and NKp44+ ILC3s (NCR+ ILC3s) were enriched in JIA SFMCs compared to PBMCs, which corresponded to an increase in transcripts for TBX21, IFNG, and IL17A. Of the ILC subsets, the frequency of NCR− ILC3s in JIA SFMCs displayed the strongest positive association with clinical measures, which was mirrored by an expansion in IL‐17A+CD4+, IL‐17A+CD8+, and IL‐17A+ γδ T cells. Conclusion We demonstrate that the strength of the IL‐17A signature in JIA SFMCs is determined by multiple lymphoid cell types, including NCR− ILC3s and IL‐17A+CD4+, IL‐17A+CD8+, and IL‐17A+ γδ T cells. These observations may have important implications for the development of stratified therapeutics.

Objectives The purpose of this study is to evaluate the performances of recently proposed Pediatric Rheumatology International Trials Organization criteria versus current International League of Associations for Rheumatology criteria for systemic juvenile idiopathic arthritis (sJIA). Methods The study was performed at the Department of Pediatric Rheumatology in Istanbul Faculty of Medicine with a retrospective design, covering the date range 2010–2021. Patients diagnosed with sJIA, Kawasaki disease and common autoinflammatory diseases were included. Both the International League of Associations for Rheumatology and Pediatric Rheumatology International Trials Organization classification criteria were applied to each patient and cross-checked with expert rheumatologist diagnosis. Results Eighty-two patients with sJIA were compared against 189 (74 Kawasaki disease, 83 familial Mediterranean fever, 16 mevalonate kinase deficiency, 10 cryopyrin-associated periodic syndromes, and 6 tumour necrosis factor receptor-associated periodic syndrome) patients. The Pediatric Rheumatology International Trials Organization criteria demonstrated higher sensitivity (62.2% vs 80.5%, P =.003) but comparable specificity (90.5% vs 91%) as regards the International League of Associations for Rheumatology criteria. Conclusions The revised criteria appear to enhance the ability to provide early recognition and pertinent classification of sJIA. No superiority was observed in segregating sJIA from common autoinflammatory diseases and Kawasaki disease, namely in terms of specificity.

ObjectivesDue to their underlying disease as well as therapeutic immunosuppression, children and adolescents with rheumatic and musculoskeletal diseases (RMD) may be at higher risk for a severe course or worse outcome of COVID-19, and SARS-CoV2 infection may trigger a flare of the RMD. To address these issues, a specific SARS-CoV-2 questionnaire was implemented in the National Pediatric Rheumatology Database (NPRD) in Germany.MethodsDemographic, clinical and treatment data from juvenile patients with RMD as well as data about SARS-CoV-2 infection like test date and method, clinical characteristics, disease course, outcome and impact on the disease activity of the RMD documented on this questionnaire were analyzed.ResultsFrom April 17th, 2020, to February 14th, 2021, data were collected from 79 patients (53% female) with RMD with median age of 14 years, diagnosed with juvenile idiopathic arthritis (57%), autoinflammatory (23%) and connective tissue disease (8%). Sixty-one patients (77%) received disease-modifying antirheumatic drugs (DMARDs), 43% biologic DMARDs, and 9% systemic glucocorticoids. Sixty patients (76%) developed symptoms of COVID-19. Disease severity was mild and outcome was good in the majority of patients. Two patients were hospitalized, one of whom required intensive care and died of cardiorespiratory failure. In 84% of SARS-CoV-2-positive patients, no relevant increase in disease activity of the RMD was observed.ConclusionsIn our cohort, COVID-19 in juvenile patients with RMD under various medications was mild with good outcome in the majority of cases. SARS-CoV-2 infection does not appear to have a relevant impact on disease activity of the underlying condition.

International audience; Children with a diagnosis of Autoimmune Encephalitis (AE) frequently require multi-disciplinary care in order to mobilize the assessment and treatment necessary for recovery. Institutional and provider practice differences often influence the diagnostic workup and treatment pathways made available to patients. There are a variety of provider coalitions in pediatric rheumatology, internal medicine, and neurology that have been making meaningful progress toward the development of consensus in assessment and treatment approaches to patient care. However, child psychiatry is currently underrepresented in this work in spite of the high psychiatric symptom burden seen in some young patients. The need for consensus is often made visible only with inter-institutional dialogue regarding patient care trajectories. We aim to review key updates in the assessment and treatment of children and adolescents with autoimmune encephalitis during the acute phase, with or without catatonia, and to outline provider perspectives by comparing current treatment models in the United States, Canada, and Europe.

Background It is currently unknown whether patients with childhood-onset Takayasu disease (c-TA) are prone to physical inactivity and poor aerobic capacity. In this study, we assessed physical activity levels and cardiorespiratory fitness along with health-related quality of life (HRQL) and various traditional and non-traditional risk factors in patients with c-TA vs. healthy controls. Methods c-TA patients with non-active disease (n = 17) and age- and sex-matched healthy controls (n = 17) were enrolled in the study. We assessed physical activity levels, aerobic capacity, body composition, systemic inflammation, cardiometabolic markers, disease-related parameters, and HRQL. Results c-TA patients showed greater time spent in sedentary behavior (P = 0.010), and lower moderate-to-vigorous physical activity (P > 0.001) and lower step counts per day (P > 0.001). VO2peak (P P = 0.016) were significantly lower in patients with c-TA and they had worse HRQL in physical domain (P P ≤ 0.05). Conclusions c-TA patients exhibited reduced physical activity levels and aerobic capacity, worse cardiometabolic risk factors and HRQL parameter compared with healthy peers. Physical inactivity and aerobic deconditioning emerge as potentially novel risk factors for c-TA. The role of physical activity interventions in preventing poor outcomes and improving HRQL in c-TA remains to be explored.

Background Systemic juvenile idiopathic arthritis (sJIA) is a diagnosis of exclusion. The complex nature and clinical variety of the disease, as well as the vast clinical variation of disease presentation, may lead to difficulties in disease detection and subsequent delays in treatment. Aim To provide a consensus guidance on the management of newly diagnosed sJIA patients among pediatric rheumatologists in Arab countries. Methods This work was conducted in two phases. The first phase utilized an electronic survey sent through an email invitation to all pediatric rheumatologists in Arab countries. In the second phase, a Task Force of ten expert pediatric rheumatologists from Arab countries met through a series of virtual meetings. Results obtained in phase one were prioritized using a nominal group and Delphi-like techniques in phase two. Results Seven overarching principles and a set of recommendations were approved by the Task Force to form the final consensus. Conclusion This is the first consensus on a clinical approach for pediatric rheumatic diseases among Arab pediatric rheumatologists. It is presented as a guidance on the clinical approach to sJIA that requires further evidence, and future updates are anticipated.

Objectives The Committee for Support of Transition to Adult Medical Care, Pediatric Rheumatology Association of Japan, has developed a checklist for patients with pediatric rheumatologic diseases to evaluate readiness for transition to adult medical care. Methods Using checklists for general pediatric chronic diseases developed by researchers at the Ministry of Health, Labour and Welfare, committee members discussed points for modification or addition specific to pediatric rheumatic diseases and pediatric rheumatism clinical practice. Results Three patient-assessment checklists based on patient age and understanding level and a parent-assessment checklist were developed. The checklist for junior high school students and above included a ‘Health Education in Adolescence and Young Adulthood’ section with items related to sexual knowledge and concerns. Also, items on oral medications and subcutaneous injections management in the ‘Self-management of Daily Medical Care,’ domain and next medical visits management were added. The checklist for junior high school students included 30 items in seven domains and can be completed within 10 minutes. The checklist was given to 28 children with pediatric rheumatic diseases aged 10 years and older and their mothers. Conclusion The checklist was developed to share the challenges of independence during transition with patients and parents.

Providing a summary of the latest research on outcome measures in juvenile idiopathic arthritis, childhood -onset systemic lupus erythematosus, and juvenile dermatomyositis. A rational management of patients with pediatric rheumatic diseases requires the regular assessment of the level of disease activity and damage, as well as the evaluation of therapeutic response through validated and standardized outcome measures. Ideally, such tools should be simple, feasible, and easily applicable in routine care. Recently, there has been a great deal of effort to refine existing tools and devise novel outcome measures, aiming to address the various aspects of disease impact and to improve the reliability of research studies and clinical trials. The newest outcome tools in pediatric rheumatology have markedly enlarged the spectrum of health domains assessable in a standardized way, thus increasing the reliability of evaluation of clinical response and fostering future clinical trials.

Background There are few reports of COVID-19 in pediatric patients with rheumatic diseases. This study describes the clinical presentation and outcomes of COVID-19 in this population. Methods We analyzed a single-center case series of pediatric patients with rheumatic diseases and laboratory-confirmed COVID-19. Demographic, baseline and COVID-19 associated clinical features were compared between ambulatory and hospitalized patients using univariate analysis. Results Fifty-five cases were identified: 45 (81.8%) in the ambulatory group and 10 (18.2%) hospitalized. African American race (OR 7.78; 95% CI [1.46–55.38]; p = 0.006) and cardiovascular disease (OR 19.40; 95% CI 2.45–254.14; p = 0.001) predominated in hospitalized patients. Active rheumatic disease (OR 11.83; 95% CI 1.43–558.37; p = 0.01), medium/high-dose corticosteroid use (OR 14.12; 95% CI [2.31–106.04]; p = 0.001), mycophenolate use (OR 8.84; 95% CI [1.64–63.88]; p = 0.004), rituximab use (OR 19.40; 95% CI [2.45–254.14]; p = 0.001) and severe immunosuppression (OR 34.80; 95% CI [3.94–1704.26]; p = p = 0.006), dyspnea (OR 26.28; 95% CI [2.17–1459.25]; p = 0.003), chest pain (OR 13.20; 95% CI [1.53–175.79]; p = 0.007), and rash (OR 26.28; 95% CI [2.17–1459.25]; p = 0.003) were more commonly observed in hospitalized patients. Rheumatic disease flares were almost exclusive to hospitalized patients (OR 55.95; 95% CI [5.16–3023.74]; p Conclusions Medium/high-dose corticosteroid, mycophenolate and rituximab use, and severe immunosuppression were risk factors for hospitalization. Fever, dyspnea, chest pain, and rash were high-risk symptoms for hospitalization. Rheumatic disease activity and flare could contribute to the need for hospitalization.

ObjectiveSince 2010, the rheumatology community has developed guidelines and tools to improve healthcare transition. In this study, we aimed to compare current transition practices and beliefs among Childhood Arthritis and Rheumatology Research Alliance (CARRA) rheumatology providers with transition practices from a provider survey published in 2010.MethodsIn 2018, CARRA members completed a 25-item online survey about healthcare transition. Got Transition’s Current Assessment of Health Care Transition Activities was used to measure clinical transition processes on a scale of 1 (basic) to 4 (comprehensive). Bivariate analyses were used to compare 2010 and 2018 survey findings.ResultsOver half of CARRA members completed the survey (202/396), including pediatric rheumatologists, adult- and pediatric-trained rheumatologists, pediatric rheumatology fellows, and advanced practice providers. The most common target age to begin transition planning was 15–17 years (49%). Most providers transferred patients prior to age 21 years (75%). Few providers used the American College of Rheumatology transition tools (31%) or have a dedicated transition clinic (23%). Only 17% had a transition policy in place, and 63% did not consistently address healthcare transition with patients. When compared to the 2010 survey, improvement was noted in 3 of 12 transition barriers: availability of adult primary care providers, availability of adult rheumatologists, and pediatric staff transition knowledge and skills (P < 0.001 for each). Nevertheless, the mean current assessment score was < 2 for each measurement.ConclusionThis study demonstrates improvement in certain transition barriers and practices since 2010, although implementation of structured transition processes remains inconsistent.

BackgroundHigh-intensity glucocorticoid regimens are commonly used to induce and maintain remission in Juvenile Dermatomyositis but are associated with several adverse side-effects. Evidence-based treatment guidelines from North American and European pediatric rheumatology research societies both advocate induction with intravenous pulse steroids followed by high dose oral steroids (2 mg/kg/day), which are then tapered. This study reports the time to disease control with reduced glucocorticoid dosing.MethodsWe retrospectively reviewed the records at a single tertiary-care children’s hospital of patients diagnosed with Juvenile Dermatomyositis between 2000 and 2014 who had a minimum of 2 years of follow-up. The primary outcome measure was time to control of muscle and skin disease. Additional outcome measures included glucocorticoid dosing, effect of treatment on height, frequency of calcinosis, and complications from treatment.ResultsOf the 69 patients followed during the study period, 31 fulfilled inclusion criteria. Median length of follow-up was 4.58 years, (IQR 3–7.5). Myositis control was achieved in a median of 7.1 months (IQR 0.9–63.4). Cutaneous disease control was achieved in a median of 16.7 months (IQR 4.3–89.5). The median starting dose of glucocorticoids was 0.85 mg/kg/day, (IQR 0.5–1.74). The median duration of steroid treatment was 9.1 months, (IQR 4.7–17.4), while the median duration of any pharmacotherapy was 29.2 months (IQR 10.4 to 121.3). Sustained disease control off medications was achieved in 21/31 (68%) patients by the end of review. Persistent calcinosis was identified in only one patient (3%).ConclusionCurrent accepted treatment paradigms for Juvenile Dermatomyositis include oral glucocorticoids beginning at 2 mg/kg/day and reduced over a prolonged time period. However, our results suggest that treatment using reduced doses and duration with early use of steroid-sparing agents is comparably effective in achieving favorable outcomes in Juvenile Dermatomyositis.