Discordant Perception of Disease Activity Between Providers and Adolescents with Systemic Lupus Erythematosus: A Cohort Study.

Discordance in perception of disease activity between adolescent patients with lupus and their providers may influence disease outcomes. We found that patients endorsed higher perceptions of disease activity than providers. Discordance was present at all levels of disease activity, particularly in patients with high activity, nephritis, and/or taking corticosteroids or mycophenolate mofetil.

Transitioning from pediatric to adult-oriented healthcare in rheumatology: the pediatric side of the coin.

OBJECTIVES: The transition of adolescents and young adults (AYAs) from pediatric to adult-oriented healthcare may be affected by many factors, including the personal and cultural settings. We aimed to analyze the transition readiness and the factors affecting the transition success in rheumatology. METHODS: Patients older than 12 years were included in this prospective study. All filled out the Transition Readiness Assessment Questionnaire (TRAQ) 5.0. AYAs were phone-interviewed after their transfer to adult-oriented healthcare. Drug adherence was evaluated with 4-item Morisky Medication Adherence Scale (MMAS-4). AYAs rated their transitional care experience with visual analogue scale (VAS 0-10; 0, the worst; 10, the best). RESULTS: A total of 504 TRAQs were filled out by 406 patients (F/M = 1.5). The total TRAQ score was positively correlated with age and higher in the forms filled out by girls than boys (4.2 vs 4.0, respectively; p= 0.005). The transition was successful for 78 (83.9%) out of 93 patients transferred to adult-oriented healthcare. The VAS for the transition process was lower and the post-transfer MMAS-4 score was worse (8 vs 9, p= 0.030 and 3 vs 4, p= 0.020; respectively) in patients whose transition was not successful when compared with the successfully-transitioned ones. The best-performing TRAQ cut-off value was >4.0 for predicting transfer readiness in rheumatology. CONCLUSION: A TRAQ score of > 4 could be used while deciding about the transfer readiness of AYAs in rheumatology. Improving the AYAs' experience of the transition process and closely monitoring medication adherence during transition are essential for a successful transition.

Normative data on lower extremity entheseal tendon thicknesses in healthy children: an ultrasound study correlating age, sex, anthropometry.

OBJECTIVES: This study aims to establish normative data on lower extremity entheseal tendon thicknesses in healthy children and examine correlations with age, gender, and anthropometric measures using musculoskeletal ultrasound. The secondary objective of the study is to investigate the power Doppler properties of entheseal tendons. METHODS: A total of 192 healthy children, aged 5-18 years, participated in this cross-sectional study. Participants underwent detailed physical and ultrasonographic examinations. Entheseal tendon thickness measurements were taken from five specific regions: distal quadriceps tendon (DQT), proximal patellar ligament (PPL), distal patellar ligament (DPL), Achilles tendon (AT), and plantar fascia (PF). Correlations between thicknesses and age, weight, height, and body mass index (BMI) were analyzed. Intra-tendinous vascularity was evaluated using power Doppler. Interobserver and intraobserver agreements were assessed using intraclass correlation coefficients (ICC). RESULTS: Normative data on lower extremity entheseal tendon thicknesses according to age, weight, height, and BMI have been established. Significant positive correlations were found between thicknesses and age, weight, height, and BMI. Weight was identified as the most influential factor, particularly for the DPL and AT. Right side tendons (AT and PF) are statistically thicker. Minimal Doppler activity was detected in 10.6% of the entheseal DQTs in the group of children aged 5-9 years. The study achieved high to excellent interobserver and intraobserver agreement. CONCLUSION: This study examined the ultrasonographic characteristics of lower extremity entheseal tendons in healthy children using B-mode and power Doppler, provided normative data on their thicknesses, and demonstrated significant correlations between thicknesses and age, sex, and anthropometry.

Genetic changes from type I interferons and JAK inhibitors: clues to drivers of juvenile dermatomyositis.

OBJECTIVE: To better understand the pathogenesis of juvenile dermatomyositis (JDM), we examined the effect of the cytokines type I interferons (IFN I) and JAK inhibitor drugs (JAKi) on gene expression in bioengineered pediatric skeletal muscle. METHODS: Myoblasts from three healthy pediatric donors were used to create three-dimensional skeletal muscle units termed myobundles. Myobundles were treated with IFN I, either IFNα or IFNß. A subset of IFNß-exposed myobundles was treated with JAKi tofacitinib or baricitinib. RNA sequencing analysis was performed on all myobundles. RESULTS: Seventy-six myobundles were analysed. Principal component analysis showed donor-specific clusters of gene expression across IFNα and IFNß-exposed myobundles in a dose-dependent manner. Both cytokines upregulated interferon response and proinflammatory genes; however, IFNß led to more significant upregulation. Key downregulated pathways involved oxidative phosphorylation, fatty acid metabolism and myogenesis genes. Addition of tofacitinib or baricitinib moderated the gene expression induced by IFNß, with partial reversal of upregulated inflammatory and downregulated myogenesis pathways. Baricitinib altered genetic profiles more than tofacitinib. CONCLUSION: IFNß leads to more pro-inflammatory gene upregulation than IFNα, correlating to greater decrease in contractile protein gene expression and reduced contractile force. JAK inhibitors, baricitinib more so than tofacitinib, partially reverse IFN I-induced genetic changes. Increased IFN I exposure in healthy bioengineered skeletal muscle leads to IFN-inducible gene expression, inflammatory pathway enrichment, and myogenesis gene downregulation, consistent with what is observed in JDM.

Childhood-Onset Systemic Lupus Erythematosus (cSLE): An International Perspective.

PURPOSE OF REVIEW: Childhood-onset systemic lupus erythematosus (cSLE) is a severe and potentially life-threatening chronic autoimmune disease. cSLE is more aggressive and has poorer outcomes than adult-onset disease. The global burden of cSLE is poorly understood, with most publications on cSLE originating from high-resourced settings. The reports from less resourced settings indicate high morbidity and mortality in these populations. RECENT FINDINGS: In this article, we review the disparities in global access to rheumatology care and research for patients with cSLE. We highlight recent cSLE advances from all regions of the globe. We describe current obstacles to cSLE clinical care and research in all settings. Finally, we propose a path forward for high quality, equitable and accessible care to individuals with cSLE everywhere. Individuals with cSLE are at risk for morbidity and death, yet patients worldwide face challenges to adequate access to care and research. Sustained, collaborative efforts are needed to create pathways to improve care and outcomes for these patients.

Evaluation of pediatric rheumatologists' approach to rituximab use: a questionnaire study.

Rituximab (RTX) is a chimeric monoclonal antibody that targets the CD20 antigen on B cells and is used in various autoimmune disorders. In this study, we aimed to measure the awareness of pediatric rheumatologists about the use of RTX through a survey. Between February and March 2023, a 42-question survey was sent via email to pediatric rheumatology specialists in Turkey. The participants were questioned for which diagnoses and system involvement they preferred to use RTX, which routine tests they performed, vaccination policy, and adverse events that occurred during or after infusion. Forty-one pediatric rheumatologists answered the survey. They prescribed RTX most frequently for systemic lupus erythematosus (87.8%) and ANCA-associated vasculitis (9.8%). Prior to the administration of RTX, 95% of clinicians checked renal and liver function tests, as well as immunoglobulin levels. The most frequently tested hepatitis markers before treatment were HBsAg and anti-HBs antibody (97.6%), while 85.4% of rheumatologists checked for anti-HCV. Clinicians (31.4%) reported that they postpone RTX infusion 2 weeks following an inactivated vaccine. Sixty-one percent of rheumatologists reported starting RTX treatment 1 month after live vaccines, while 26.8% waited 6 months. The most frequent adverse events were an allergic reaction during RTX infusion (65.9%), hypogammaglobulinemia (46.3%), and rash (36.6%). In the event of hypogammaglobulinemia after RTX treatment, physicians reported that they frequently (58.5%) continued RTX after intravenous immunoglobulin administration. CONCLUSIONS: RTX has become a common treatment option in pediatric rheumatology in recent years. Treatment management may vary between clinician such as vaccination and routine tests. WHAT IS KNOWN: • During the course of rituximab therapy, clinicians should be attentive to specific considerations in pre-treatment, during administration, and in post-treatment patient monitoring. WHAT IS NEW: • There are differences in practice among clinicians in the management of RTX therapy. These practice disparities have the potential to impact the optimal course of treatment. • This study highlights that standardized guidelines are needed for RTX treatment in pediatric rheumatology, particularly for vaccination policies and routine tests.

The emerging paradigm in pediatric rheumatology: harnessing the power of artificial intelligence.

Artificial intelligence algorithms, with roots extending into the past but experiencing a resurgence and evolution in recent years due to their superiority over traditional methods and contributions to human capabilities, have begun to make their presence felt in the field of pediatric rheumatology. In the ever-evolving realm of pediatric rheumatology, there have been incremental advancements supported by artificial intelligence in understanding and stratifying diseases, developing biomarkers, refining visual analyses, and facilitating individualized treatment approaches. However, like in many other domains, these strides have yet to gain clinical applicability and validation, and ethical issues remain unresolved. Furthermore, mastering different and novel terminologies appears challenging for clinicians. This review aims to provide a comprehensive overview of the current literature, categorizing algorithms and their applications, thus offering a fresh perspective on the nascent relationship between pediatric rheumatology and artificial intelligence, highlighting both its advancements and constraints.

Implementation and assessment of a structured curriculum for a 4-week pediatric rheumatology rotation for pediatric residents.

BACKGROUND: General pediatricians often initially address children's musculoskeletal (MSK) issues and play a crucial role in triaging and managing patients' rheumatologic conditions. This study assessed the effectiveness of a structured curriculum in enhancing pediatric residents' knowledge, MSK examination skills, and confidence during a 4-week pediatric rheumatology rotation. METHODS: Pediatric residents in their either second or third year who participated in the 4-week rheumatology rotation once across three academic years (July 2020-June 2023) were enrolled. Residents' knowledge, MSK examination skills, and confidence were assessed at pre- and post-rotation by using 25 multiple-choice questions, the Thai pediatric Gait Arms Legs Spine examination, and a questionnaire, respectively. The curriculum comprised instruction on MSK examinations, interactive lectures, case-based discussion, topic reviews, MSK radiology conference, clinical experience in rheumatology clinic and consultations, with self-guided learning with educational resources. RESULTS: Fifty-eight pediatric residents (48 females, 10 males) with a mean age of 28.9 ± 0.8 years participated. Significant improvements were noted postrotation. Knowledge scores rose from 63.0 ± 12.2 to 79.7 ± 9.1 (mean difference 16.7 ± 10.3, p < 0.001). Similarly, MSK examination scores increased from 67.5 ± 14.4 to 93.6 ± 8.7 (mean difference 26.1 ± 14.6, p < 0.001). Residents also reported a marked increase in confidence across all evaluated areas, including history taking, MSK examination, arthrocentesis, and diagnosing and treating rheumatologic conditions (p < 0.001). CONCLUSIONS: The 4-week structured curriculum in the pediatric rheumatology rotation significantly enhanced pediatric residents' knowledge, MSK examination skills, and confidence. These findings support the integration of pediatric rheumatology rotations into pediatric residency training programs.

[Advances in the diagnostics and treatment of juvenile dermatomyositis]. / Fortschritte in der Diagnostik und Therapie der juvenilen Dermatomyositis.

Juvenile dermatomyositis (JDM) is by far the most frequent inflammatory myopathy in childhood and adolescence. It is clinically characterized by inflammatory changes of the skin and muscles but as a multisystemic disease can also affect the skeletal system, the gastrointestinal tract, lungs and heart. Intrinsic (multigenetic risk) and extrinsic factors (triggers) are involved in the pathogenesis resulting in endothelial damage, involvement of fascies, activation of the interferon system and autoimmune reactions including formation of myositis-specific autoantibodies (MSA). In contrast to dermatomyositis in adults, in children and adolescents there are no associations with malignant diseases. The variable expression, the rarity of the disease and the risk of long-term damage and complications necessitate pediatric rheumatological experience in the diagnostics and treatment. Recently, new approaches in drug treatment have substantially improved the outcome and prognosis but a multidisciplinary treatment (including physicians, physiotherapists, psychologists, social workers) is mandatory, especially in the first phases of the disease. Particularly important is a professionally correct treatment of the functional sequelae, which are a particular focus of this article.

Survey of diversity awareness and environment among members of the Pediatric Rheumatology Association of Japan.

BACKGROUND: Diversity management has gained traction in Japan. The Pediatric Rheumatology Association of Japan (PRAJ) has an Advisory Committee for Diversity Promotion with a broader focus on promoting diversity. The objectives of this study were to better understand the problems faced by PRAJ members regarding the work environment, childcare and nursing care, and work-life balance. METHODS: A web-based questionnaire was administered to members of the PRAJ and 79 responses were evaluated. RESULTS: Of the respondents, 73% were male and 27% were female. A total of 14% worked for more than 12 h on weekdays, and 22% worked for more than 60 h per week and 38% had fewer than 4 days off per month. Regarding childcare, 54% of the respondents were raising preschool children and 83% had taken parental leave for less than 1 year. A total of 17% of participants had family members in need of care. For both childcare and caregiving, the burden was greater for women. Only 18% of the respondents reported a well-balanced work-life balance, and the most common reasons for a lack of balance were not having enough time, heavy workload, and heavy housework load. CONCLUSIONS: The working hours of the respondents were long, and female members had a greater burden of childcare and caregiving, which was considered a barrier to the career development of women. In the future, there will be a need to promote a sense of equality in diverse human resources, develop support for family life, and shorten working hours.

Validating within-limb calibrated algorithm using a smartphone attached infrared thermal camera for detection of arthritis in children.

OBJECTIVES: To determine the impact of physical activity on temperature after within-limb calibration (TAWiC) measures and their reproducibility. To determine if thermal imaging from a smartphone attached thermal camera is comparable to thermal imaging using a handheld thermal camera for detection of arthritis in children. METHODS: Children without symptoms were enrolled to the "asymptomatic exercise cohort", and received infrared imaging, using a standard handheld camera, after initial resting period, after activity, and after second resting period. Children seen in the rheumatology clinic with knee pain were enrolled into the "symptomatic knee pain cohort" and received imaging with both the smartphone-attached and handheld cameras before a routine clinical exam. TAWiC was defined as the temperature differences between joint and ipsilateral mid-tibia as the main readout for arthritis detection. RESULTS: The asymptomatic exercise cohort demonstrated notable changes in absolute and TAWiC temperatures collected by thermal imaging after physical activity, and temperatures did not consistently return to pre-activity levels after a second period of rest. The 95th TAWiC from anterior view were, resting one -0.1 C (0.5), activity -0.7 C (0.5), resting two -0.2 C (0.6) (resting 1 vs resting 2, p-value = 0.13). In the symptomatic knee pain cohort, the smartphone attached and handheld thermal cameras performed similarly in regards to detection of joint inflammation and evaluation of joint temperature using the TAWiC algorithm, with high sensitivity of 80% (55.2-100.0%) and specificity of 84.2% (76.0-92.4%) in the anterior knee view when compared with the gold standard joint exam performed by a pediatric rheumatologist. The mean 95th TAWiC temperature difference between the two cameras was -0.1 C (-0.1 to 0.0) (p = 0.0004). CONCLUSIONS: This study showed continued validity of the TAWiC algorithm across two distinct thermal camera platforms and demonstrates promise for improved accessibility and utility of this technology for arthritis detection.

Parent Views on Telemedicine in Pediatric Rheumatology: A Survey Study.

Objectives: The rapid expansion in the use of telemedicine after the COVID-19 pandemic has led many patients with chronic diseases to seek alternative ways for follow-ups. This study aimed to investigate the demands and opinions of parents of children with rheumatic diseases toward telemedicine and to examine the factors affecting telemedicine preference. Methods: A single-center, cross-sectional, Web-based survey study was conducted. Sociodemographic data, characteristics of the disease, access to the clinic, internet use, and views on telemedicine were assessed. Factors effecting telemedicine preference were evaluated by multivariate analysis. Results: A total of 245 parents have completed the survey. The diagnoses of patients were recurrent fever syndromes (55.1%), juvenile idiopathic arthritis (31.0%), systemic connective tissue diseases (8.2%), and vasculitis (5.7%). The majority of patients came to the clinic by public transport (n = 190, 77.6%). Sixty-eight (27.8%) patients missed at least one outpatient appointment in the last year. Majority (n = 172, 70.2%) of parents stated that they would prefer telemedicine visits if it becomes available. Multivariate analysis revealed that the most related factors to telemedicine preference were higher education level (odds ratio [OR]: 6.69, confidence interval [95% CI]: 2.21-20.25, p = 0.001), missing an appointment (OR: 3.04, 95% CI: 1.41-6.56, p = 0.004), and travel time longer than 1 h (OR: 2.13, 95% CI: 1.13-3.86, p = 0.012). Conclusion: Telemedicine visits are in demand in pediatric rheumatology and should be considered an alternative method to ensure continuity of patient follow-up. A personal approach should be followed when selecting patients for telemedicine.

A Diagnostic Prediction Model for Separating Juvenile Idiopathic Arthritis and Chronic Musculoskeletal Pain Syndrome.

OBJECTIVE: To develop and validate a diagnostic prediction model that can distinguish between juvenile idiopathic arthritis (JIA) and chronic musculoskeletal pain syndrome (CMPS) based on patient-reported outcomes. STUDY DESIGN: This retrospective cohort study evaluated whether the Juvenile Arthritis Multidimensional Assessment Report (JAMAR) performs well in distinguishing JIA from CMPS. We analyzed JAMARs completed by 287 patients at their first visit to the pediatric rheumatology department of Wilhelmina Children's Hospital in Utrecht, The Netherlands. Relevant JAMAR items for predicting a diagnosis of JIA were selected in a penalized multivariable model suitable for clinical application. The model was subsequently validated with new data from the same center. RESULTS: A total of 196 JAMARs (97 JIA, 99 CMPS) were collected in the model development data, and 91 JAMARs (48 JIA, 43 CMPS) were collected in the validation data. Variables in the prediction model that were strongest associated with a diagnosis of JIA instead of CMPS were asymmetric pain/swelling in the shoulder (OR, 2.34), difficulty with self-care (OR, 2.41), skin rash (OR, 2.07), and asymmetric/pain swelling in the knee (OR, 2.29). Calibration and discrimination (area under the receiver operating characteristic curve, 0.83; 95% CI, 0.74-0.92) of the model in the validation data were good. CONCLUSIONS: Several items from the JAMAR questionnaire can potentially distinguish JIA from CMPS in patients with corresponding symptoms. We present an easy-to-use, adjusted, and validated model to separate these 2 diagnoses early at presentation based on patient-reported outcomes to facilitate proper referral and treatment.

Who Would Have Predicted Multisystem Inflammatory Syndrome in Children?

PURPOSE OF REVIEW: Multisystem inflammatory disease in children (MIS-C) is a novel post-infectious phenomenon following coronavirus disease-19 (COVID-19). Herein, we present an in-depth review of the latest MIS-C literature related to clinical findings, pathophysiology, imaging and laboratory studies, treatment algorithms, and disease outcomes. RECENT FINDINGS: With its non-specific presentation of fever, gastrointestinal symptoms, cardiovascular injury and shock, systemic inflammation, and Kawasaki disease (KD)-like features, MIS-C can be a diagnostic challenge, overlapping with KD and active COVID-19 infection. However, common laboratory features, imaging findings, and historical clues can lead to accurate diagnosis and allow for appropriate treatment with a variety of immunomodulatory therapies, including intravenous immunoglobulin (IVIG). Aggressive treatment of MIS-C leads to good outcomes. Longitudinal studies continue to illuminate long-term cardiac sequelae and recovery. MIS-C presents with fever, KD features, gastrointestinal symptoms, cardiac inflammation, and shock. Early recognition and prompt institution of IVIG and glucocorticoids provide for rapid improvement.

Cross-sectional characteristics of pediatric-onset discoid lupus erythematosus: Results of a multicenter, retrospective cohort study.

BACKGROUND: The incidence of systemic lupus in children with discoid lupus is unknown. OBJECTIVE: This study assessed the baseline characteristics of patients with pediatric discoid lupus erythematosus (pDLE). METHODS: Medical 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 <18 years. Baseline data were collected at the first documented visit. Outcomes included diagnosis of systemic lupus erythematosus (SLE) at the baseline visit using the 1997 American College of Rheumatology (primary) and the 2012 Systemic Lupus International Collaborating Clinics (secondary) criteria. RESULTS: Of the >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 8.9 years; P < .001), with shorter time from discoid lupus erythematosus onset to diagnosis, compared with patients with pDLE-only (median, 2 vs 7 months; P < .001). Patients with pDLE + SLE were more likely to be female (P = .004), with generalized discoid lupus erythematosus and clinically aggressive disease, including end-organ involvement, positive serologies, and higher- titer levels of antinuclear antibodies (P < .001). LIMITATIONS: Retrospective study. CONCLUSION: A diagnosis of discoid lupus erythematosus in adolescence should prompt thorough screening for SLE.

Assessing the Association of Kawasaki Disease Symptoms and Echocardiographic Findings.

Kawasaki disease is an acute systemic vasculitis which can cause cardiac involvement among other symptoms. In this study we aimed to assess the relationship between the echocardiographic findings of Kawasaki disease with the clinical and paraclinical findings of the patients. In this cross-sectional study, the symptoms of 307 Kawasaki patients were registered and the association of the symptoms with paraclinical findings and echocardiographic studies was assessed. 190 (61.9%) of the patients were male and 117 (38.1%) were female. 193 patients (62.9%) did not have any abnormalities in their echocardiography, while others showed coronary artery aneurysms, perivascular brightness, coronary artery dilatation, and trivial Mitral Regurgitation in their echocardiography. A significant inverse relationship was seen with echocardiographic findings and age. Thrombocytosis, conjunctivitis, and oral and/or pharyngeal erythema and/or strawberry tongue were associated with higher rates of echocardiographic abnormalities. Echocardiographic abnormalities are associated with younger age, higher platelets, and the existence of conjunctivitis and oral and/or pharyngeal erythema and/or strawberry tongue.

[Validation of the total morbidity score and investigation of the efficacy of methotrexate in localized scleroderma]. / Validierung des "Total Morbidity Scores" und Untersuchung der Wirksamkeit von Methotrexat bei der lokalisierten Sklerodermie.

BACKGROUND: Localized scleroderma is an autoimmune disease belonging to the group of collagenoses, which can manifest cutaneously and extracutaneously. The extracutaneous manifestations may have significant morbidity but are not considered in previous scoring systems. For this reason, another scoring system, the total morbidity score (TMS) was developed, which also takes into account the extracutaneous symptoms. METHOD: In the retrospective monocentric study at the Hamburg Center for Pediatric and Adolescent Rheumatology, the TMS was applied to patients from 2004-2019 suffering from localized scleroderma who had at least one control presentation. In addition, data were analyzed according to the previously established localized scleroderma cutaneous assessment tool (LoSCAT) scoring systems to ensure better comparability to the TMS. Furthermore, the score values were considered and compared during the course of treatment with methotrexate (MTX). RESULTS: Due to a lack of control presentations, data from 51 of the 95 patients with a confirmed diagnosis could be included in the retrospective evaluation. The treatment of these patients was considered over a period of 2 years, from the initial presentation over at least 3 further control presentations. The TMS total score remained largely constant. There was a weak correlation between the TMS total score and the localized scleroderma skin damage index (mLoSDI), which indicates the degree of damage. In addition, insignificant changes in the TMS total score were shown over time with MTX treatment (T1/T4: -0.007). DISCUSSION: The evaluation showed that the TMS total score is mainly fed by the extracutaneous manifestations, demonstrating the inaccuracy of previous scores. Another advantage of the TMS is that different scores are assigned depending on whether the feature is new, persistent, improving, or even worsening. The TMS is more time consuming to collect but enables a more accurate assessment of disease activity.

Childhood Sjögren syndrome: features of an international cohort and application of the 2016 ACR/EULAR classification criteria.

OBJECTIVE: Sjögren syndrome in children is a poorly understood autoimmune disease. We aimed to describe the clinical and diagnostic features of children diagnosed with Sjögren syndrome and explore how the 2016 ACR/EULAR classification criteria apply to this population. METHODS: An international workgroup retrospectively collected cases of Sjögren syndrome diagnosed under 18 years of age from 23 centres across eight nations. We analysed patterns of symptoms, diagnostic workup, and applied the 2016 ACR/EULAR classification criteria. RESULTS: We identified 300 children with Sjögren syndrome. The majority of patients n = 232 (77%) did not meet 2016 ACR/EULAR classification criteria, but n = 110 (37%) did not have sufficient testing done to even possibly achieve the score necessary to meet criteria. Even among those children with all criteria items tested, only 36% met criteria. The most common non-sicca symptoms were arthralgia [n = 161 (54%)] and parotitis [n = 140 (47%)] with parotitis inversely correlating with age. CONCLUSION: Sjögren syndrome in children can present at any age. Recurrent or persistent parotitis and arthralgias are common symptoms that should prompt clinicians to consider the possibility of Sjögren syndrome. The majority of children diagnosed with Sjögren syndromes did not meet 2016 ACR/EULAR classification criteria. Comprehensive diagnostic testing from the 2016 ACR/EULAR criteria are not universally performed. This may lead to under-recognition and emphasizes a need for further research including creation of paediatric-specific classification criteria.

Preliminary evidence on abatacept safety and efficacy in refractory juvenile localized scleroderma.

OBJECTIVE: To evaluate the safety and efficacy of abatacept treatment for refractory juvenile localized scleroderma (jLS) in a retrospective study. METHODS: A multicentre cohort study was performed to evaluate jLS subjects treated with abatacept with follow-up for 12 months to maximum of 24 months. Assessments at 6-month intervals included skin activity measures and physician global assessment of activity (PGA-A). Descriptive statistical analysis was performed. RESULTS: Eighteen subjects were studied with median age of 13.4 years, the majority had linear scleroderma subtype, and musculoskeletal involvement. All had previously failed MTX and/or mycophenolate mofetil treatment and glucocorticoids. Abatacept was added to the subject's maintenance DMARD treatment; 13 also received glucocorticoids at start of abatacept. No serious adverse events occurred. Skin activity and PGA-A scores declined in nearly all by 6 months and continued to improve from 6 to 12 months. At 12 months, 15 (83%) subjects were considered responders, two (11%) treatment failures, and one dropped out for adverse event. Response was sustained for 11 (61%) subjects to 18 months and eight (44%) to 24 months. Overall, four (22%) subjects were treatment failures and three (16.7%) discontinued abatacept for adverse event. Active musculoskeletal problems improved in most affected subjects. Ten subjects were able to discontinue initial glucocorticoid and six concomitant DMARD treatment. CONCLUSION: Abatacept was found to be safe and effective for jLS subjects refractory to standard of care treatment. Subjects experienced improvement in both skin and musculoskeletal activity. Prospective studies should be performed to more fully evaluate abatacept's efficacy.

Principles of pediatric lupus nephritis in a prospective contemporary multi-center cohort.

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.