Juvenile Arthritis Research Articles

Sonographic and Disease Activity Findings Related With Medication Change in JIA: A Historical Cohort Study.

Musculoskeletal ultrasound (MSUS) is increasingly used to evaluate pediatric inflammatory arthritis. This study aimed to explore the relationship between MSUS findings with medication modifications in patients with juvenile idiopathic arthritis (JIA) and clinical disease activity measurements (clinical Juvenile Arthritis Disease Activity Score [cJADAS-10], active joint count [AJC], patient/parent global assessment [PPGA], and physician global assessment [PGA]). Data from patients with JIA who underwent a 12-joint (bilateral second and third metacarpophalangeal, wrist, elbow, knee, and ankle) MSUS examination during a 57-month period were collected. Patients were categorized into 2 groups: a medication change group and a control group (patients without medication change). A pediatric-specific MSUS scoring system was used to assess MSUS findings. The association between clinical and MSUS findings was examined for the study groups. A total of 38 patients, 23 in the medication change group and 15 in the control group were included. The medication change group had higher AJC, PGA, and cJADAS-10. These patients also had a statistically significant presence of abnormal knee MSUS findings. For other joints, the frequency of abnormal MSUS findings was slightly higher in patients with a medication change, but the difference was not statistically significant. No strong correlation was observed between MSUS findings and clinical disease activity measurements. Abnormal MSUS findings were not observed to be higher in patients with a change in medication except for the involvement of the knee joint. Further longitudinal studies are needed to understand the role of MSUS in the medical decision-making process in JIA.

What are Juvenile-onset systemic sclerosis providers thoughts, experiences, and reasons for autologous stem cell transplant? Result of a multinational survey.

Juvenile-onset systemic sclerosis (jSSc) is a rare and life-threatening disease with no formal studies evaluating the indications for, access to, or benefits of autologous stem cell transplantation (ASCT). As a first step toward understanding pediatric jSSc specialist thoughts and experiences with ASCT, we conducted a multinational survey. An electronic survey was developed and distributed in November 2023 to members of the Pediatric Rheumatology European Society (PRES) and/or Childhood Arthritis and Rheumatology Research Alliance (CARRA) pediatric scleroderma workgroups. Twenty-nine (69%) jSSc specialists completed the survey. All participants have considered or would consider ASCT referral for a jSSc patient. Nearly all respondents indicated disease-modifying anti-rheumatic drugs (DMARDs) should be trialed prior to ASCT referral, with most indicating two to four DMARDs. The most common reasons selected for referral were rapidly progressive disease (despite DMARD) (90%), followed by severe disease status (83%), and significant impact on quality of life (83%). All respondents selected pulmonary disease as an indication for referral, followed by cardiac (93%), gastrointestinal (72%), and skin disease (66%). While pulmonary and cardiac involvement were considered individually sufficient for referral for ASCT, only a minority considered musculoskeletal involvement (28%) sufficient on its own. This survey is the first explore thoughts and experience with ASCT for jSSc. Results indicate pediatric rheumatologists were aware of and would consider ASCT for their patients. Our results indicate there is likely some variability in clinical practice regarding who is referred for ASCT, and further research is needed to guide development of evidence-based clinical care guidelines.

Commentary on "breathing breakthrough: Muscle training improves lung function in juvenile arthritis".

Commentary on "breathing breakthrough: Muscle training improves lung function in juvenile arthritis".

Клинико-эпидемиологическая характеристика ювенильного идиопатического артрита у детей в Республике Саха (Якутия), РФ

Due to the ethnic and geographical features, the incidence of rheumatic diseases in the Republic of Sakha (Yakutia), Russia, differs from it in other Russia regions. The purposes of this research were to describe the epidemiological, demographic, clinical and laboratory characteristics of pediatric patients with juvenile idiopathic arthritis (JIA) residing on the territory of the Republic of Sakha (Yakutia), Russia (RSY), to describe the therapeutical options and to evaluate the main outcomes. Materials and methods used: the continuous single-center cohort retrospective study included data from medical records of all patients (225) with diagnosed JIA who had undergone examination and treatment in the cardiorheumatological department of the Pediatric Center with the Sakha (Yakutia) Republican Hospital No. 1 - M.E. Nikolaev National Medicine Center (Yakutsk, Republic of Sakha (Yakutia), Russia) in 2016-2023. Results: the incidence of JIA in the RSY regional pediatric population aged 0 to 17 y/o was increasing noticeably, from 5.3 per 100,000 in 2016 to 15.5 per 100,000 in 2023, i.e. almost tripled (2.9) or by 192.9%. The 2023 RSY JIA prevalence was 84.5 and was higher in children living in rural areas - 102.8 per 100,000 children, than in cities - 73.6 per 100,000 children. By ethnicity, the JIA prevalence was higher in the Yakut children - 110.1 per 100,000, than in children of the Russian nationality - 69.4 per 100,000. Among the Yakut children living in the cities, the JIA prevalence was higher (139.4 per 100,000) than in those living in rural areas (89.8 per 100,000). The JIA prevalence among the Yakut boys and girls was identical whilst girls had predominated amongst the Russian children. The distribution of patients by JIA categories was as follows: juvenile arthritis with systemic onset counted for 3.5%, oligoarthritis - 33.8%, RF-positive polyarthritis - 0.9%, RF-negative polyarthritis - 14.7%, enthesitis-associated arthritis (EAA) - 44%, psoriatic arthritis - 3.1%. The frequency of uveitis was 10.2%, HLA-B27 carriage was 39.6%, seropositivity for ANF was 66.7%. The prevalence of EAA was 4.4 times higher in the Yakut children, and the systemic form was 2.9 times lower than that in Russians (p<0.001). 40.4% of children with JIA received therapy with genetically engineered biological drugs, with remission achieved in this group in 45.4% of cases. Conclusion: JIA in RSY has its own epidemiological, clinical and laboratory features associated with the prevalence of the HLA-B27 amongst the Yakut pediatric population. The Yakut children are more likely to suffer from JIA, the disease prevails among boys, the most common JIA form was EEA whilst uveitis and psoriasis being the rare extra-articular JIA manifestations.

Evaluating anchor variables and variation in meaningful score differences for PROMIS® Pediatric measures in children and adolescents living with a rheumatic disease.

Meaningful score differences (MSDs), as defined by recent FDA guidance, can improve the interpretation of outcome measure scores and score changes. Well-accepted methods for estimating MSDs typically rely on external anchor variables, but the applications of these methods are limited in children and adolescents with rheumatic diseases. This project explored multiple candidate anchors for the PROMIS® Pediatric measures of Physical Activity, Fatigue, Pain Interference, and Mobility for children with juvenile idiopathic arthritis (JIA) or systemic lupus erythematosus (SLE). Longitudinal data were extracted from the Childhood Arthritis and Rheumatology Research Alliance (CARRA) Registry. Candidate anchors included patient-reported domain-specific global impressions of change (GIC) along with other parent- and clinician-reported variables. Prior to MSD estimation, the quality of the anchors was assessed using a priori criteria (correlation ≥0.30, n≥10, <10% missing). Anchors meeting criteria were used to calculate MSDs. Among 289 children with JIA and 47 with SLE, the GIC did not meet criteria inhalf of the scenarios. Other candidate anchors performed slightly better. The calculated MSDs varied by external anchor across measures, diagnoses, and direction of change (better vs worse). Many of the candidate external anchoring variables did not meet pre-specified criteria for calculating MSDs. Even for those that did, the choice of anchoring variable had a strong impact on the estimated MSD value and were different from other published values. As in adults, establishing pediatric MSDs requires selection of high-quality anchors, as changes in the variables used as anchors can impact MSD values and any subsequent score interpretations.

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.

Implementation study of the CARRA Uveitis Consensus Treatment Plans: feasibility for clinical practice and applicability for research

BackgroundChronic anterior uveitis (CAU) carries a significant risk for eye complications and vision loss. The Childhood Arthritis and Rheumatology Research Alliance (CARRA) introduced consensus treatment plans (CTPs) to standardize treatment for CAU and facilitate future comparative effectiveness studies. Two CTPs were developed to address: 1) initiation of methotrexate (MTX) in patients with CAU naïve to steroid-sparing therapy, and 2) initiation of a TNF inhibitor (TNFi) in patients with severe uveitis or uveitis refractory to MTX. We evaluated implementation of the uveitis CTPs using existing CARRA Registry infrastructure and assessed feasibility of the CTPs for comparative effectiveness research.MethodsThis prospective observational cohort study was conducted at nine pilot sites between February 2020 and August 2022. Patients with JIA-associated CAU (JIA-U) were treated according to either the MTX or TNFi CTP. Uveitis activity and medication use were recorded at 0, 3, and 6 months. We assessed patient enrollment rates, CTP arm selection, uveitis control, and quality of data collection. We also evaluated CTP arm selection in a retrospective cohort of similar JIA-U patients enrolled in the CARRA Registry during the same study period.ResultsSeventeen patients were included in the pilot cohort. Eight were treated with the MTX CTP (4 oral MTX, 4 subcutaneous MTX), and 9 with the TNFi CTP (9 received standard-dose adalimumab, none selected high-dose adalimumab or infliximab). Uveitis was controlled in 13 of 17 patients by 6 months. Query of the CARRA-wide Registry identified 42 patients with JIA-U who were treated according to the MTX or TNFi CTPs. Among these, 26 were treated with MTX (8 oral, 18 subcutaneous) and 16 with TNFi (12 standard dose adalimumab, 2 high dose adalimumab, and 2 infliximab).ConclusionBoth the MTX and TNFi uveitis CTPs can practically be implemented in clinical settings and are currently being utilized across Registry sites. However, in patients starting TNFi therapy, all pilot study participants and most patients across the CARRA Registry were treated with a standard dose of adalimumab. This consensus on the treatment approach underscores its broad acceptance but also limits the applicability of the uveitis TNFi CTP for comparative effectiveness research.

Racial Disparities and Achievement of the Low Lupus Disease Activity State (LLDAS): A CARRA Registry Study.

Differential disease control may contribute to racial disparities in outcomes of childhood-onset systemic lupus erythematosus (cSLE). We evaluated associations of race and individual- or neighborhood-level social determinants of health (SDoH) with achievement of low lupus disease activity state (LLDAS), a clinically relevant treatment target. In this cSLE cohort study using the Childhood Arthritis and Rheumatology Research Alliance (CARRA) Registry, the primary exposure was self-reported race and/or ethnicity and collected SDoH included insurance status and area deprivation index (ADI). Outcomes included LLDAS, disease activity, and time-averaged prednisone exposure. Associations between race and/or ethnicity, SDoH, and disease activity were estimated with multivariable regression models, adjusting for disease-related and demographic factors. Among 540 children with cSLE, 27% identified as Black, 25% White, 23% Latino/a, 11% Asian, 9% more than one race, and 5% Other. More Black children (41%) lived in neighborhoods of highest ADI compared to White children (16%). Black race was associated with lower LLDAS achievement (adjusted OR 0.56, 95% CI: 0.38-0.82) and higher disease activity (adjusted β: 0.94, 95% CI: 0.11, 1.78). Highest ADI was not associated with lower LLDAS achievement upon adjustment for renal disease and insurance. However, renal disease was found to be a significant mediator (p=0.04) of the association between ADI and prednisone exposure. Children with cSLE identifying as Black are less likely to achieve LLDAS and have higher disease activity. Living in areas of higher ADI may relate to renal disease and subsequent prednisone exposure. Strategies to address root causes will be important to design interventions mitigating cSLE racial disparities.

Disease Damage in Juvenile Idiopathic Arthritis.

To estimate the prevalence and predictors of disease-related damage in children with juvenile idiopathic arthritis (JIA) in a resource-limited setting. A single-centre study was conducted from January 2021 - December 2022. Children (≤ 18 y) diagnosed with JIA as per International League of Associations for Rheumatology (ILAR) criteria, with a disease duration of more than one year, were enrolled for this study. The articular and extra-articular damage was assessed using the juvenile arthritis damage index (JADI) and modified JADI scores. Disease activity and disabilities were evaluated using the clinical juvenile arthritis disease activity score (cJADAS) and Childhood Health Assessment Questionnaire (CHAQ). One hundred and five children [44% (n = 42) boys] with JIA were enrolled in the study. The mean (SD) age of children at enrolment was 158 (46.2) mo. The median (IQR) disease duration was 48 (36-72) mo. Articular damage (JADI score ≥ 1) was present in 48.6%, and extra-articular damage (JADI-E ≥ 1) was observed in 21.9% of children. Half of the children (n = 22) with enthesitis-related arthritis (ERA) had joint damage (modified JADI score ≥ 1). Four children had ocular damage due to uveitis. Among the factors associated with articular damage, the odds of articular damage were high in those with positive rheumatoid factor (RF) and/ or anti-cyclic citrullinated peptides (CCPs) [OR: 4.4, 95% CI (1.00-19.60)]. 48.6% of children with JIA had articular damage, while 21.9% of the children had extra-articular damage. Children with RF and/ or anti-CCP positivity are associated with higher odds of joint damage.

Biosimilars Versus Originators in Children With Juvenile Idiopathic Arthritis: A Real-World Experience

We aimed to evaluate the efficacy, safety, and immunogenicity profile of Etanercept (ETA) and Adalimumab (ADA) biosimilars (BIOs) compared to their originators in children with juvenile idiopathic arthritis (JIA). Eighty-one JIA children treated with ETA or ADA originators or BIOs were examined at baseline (T0) and after 3- (T1), 6- (T2), 12- (T3), and 24-(T4) months after starting treatment. Lower Juvenile Arthritis Disease Activity Score 10 (JADAS-10) scores were reported at T1, T2, T3, and T4 in JIA children treated with BIOs than originators (all p < 0.05). At T1 and T3, anti-drugs antibodies levels were lower in children receiving BIOs than originators (p = 0.04 and p = 0.0007, respectively), even after adjustments (both p < 0.05). Relapses were lower for BIOs compared to originators (p < 0.001). Safety profile was comparable between the groups (p > 0.05). A better overall profile of BIOs than originators was demonstrated in JIA children, but larger confirmatory studies are needed.

Profile of anemia in children with juvenile idiopathic arthritis

Background: Anemia is one of the commonest extra-articular manifestations or complications associated with juvenile idiopathic arthritis (JIA) and is generally seen to corelate with disease-duration and disease-activity. Aim of the study was to find out the prevalence of anemia, its etiological spectrum and severity in children suffering from JIA, to determine its correlation with disease-activity and disease-duration and assess the change in the status of anemia after initiating cause- specific treatment in addition to disease-specific treatment in a follow-up period of 3 months. Methods: This prospective longitudinal observational study was conducted in a tertiary care teaching hospital from July to December 2022. Patients of JIA were recruited through purposive sampling stratified by disease activity based on juvenile arthritis disease activity score (JADAS) 27 score and anemia categorized by World Health Organization (WHO) criteria. Results: A total of 64 children were included in the final analysis. Mean age of patients was 8.19 years (SD 2.34 years), male: female ratio being 1.2:1. 78.12% were anemic, with 59.37% being moderately anemic. Overall, anemia-of-chronic-disease (ACD) was the most (48%) prevalent type of anemia, followed by iron-deficiency-anemia (IDA) at 28%, followed by a combination of both IDA and ACD (12%). Remaining 12% was attributed to other causes of anemia. Mean-hemoglobin at 1st visit was found to decrease with increase in disease duration. Significant moderate linear negative correlation was found (r=-0.54) (p&lt;0.001) between mean-hemoglobin-at-1st-visit and mean-disease-activity-at-1st-visit. Significant rise in hemoglobin level after initiation of cause-specific treatment with disease-specific treatment was also observed in this study. Conclusions: Anemia associated with pediatric rheumatological conditions has significant negative impact on the disease process. Prompt diagnosis of anemia, denoting its type and severity, and thereby treating anemia alongside disease-specific treatment, significantly improves the patient outcome.

Benefits of a Juvenile Arthritis Support Program (JASP-1) for children recently diagnosed with Juvenile Idiopathic Arthritis and their parents

BackgroundMedical treatment for children with Juvenile Idiopathic Arthritis (JIA) has improved radically since the development of biological disease-modifying antirheumatic drugs. However, children suffer from pain and anxiety, and parents often experience loneliness and lack of support. Some parents reported that information provided at the time their child was diagnosed could be difficult to assimilate. Therefore, the aim of this study was to develop a Juvenile Arthritis Support Program (JASP-1) for children recently diagnosed with JIA and their parents. Moreover, the aim was to explore patients´ and parents´ experiences with JASP-1 and its potential impact on patients´ physical health.MethodsJASP-1 included seven patient- and family-centered clinical visit from time of diagnose and one year ahead. Data were collected from a study-specific questionnaire answered by children and their parents after participation in JASP-1 and from the pediatric rheumatology register. The study-specific questionnaire explored participants´ experience with the care they received during their first year with JIA. Registry and questionnaire data from the intervention (JASP-1) group was compared to a control group.ResultsThe analysis revealed that children and parents who completed JASP-1 were more satisfied with the care they had received during their first year with JIA than the control group. The results also showed that children who completed JASP-1 were assessed as having better overall health after 12 months, than children in the control group (JASP-1 = mean 4.33, 95% Confidence Interval (CI) 4.17 − 4.46), (Control = mean 3.68, 95% CI 3.29 − 4.06), (p = 0.002). Moreover, children in the JASP-1 group had less disease impact on daily life (JASP-1 = mean 0.15, 95% CI 0.07 − 0.24) (Control = mean 0.40, 95% CI 0.13 − 0.67), (p = 0.017) and less active joints than the control group (JASP-1 = mean 0.62, 95% CI 0.35 − 1.58), (Control = mean 0.87, 95% CI 0.18 − 1.56), (p = 0.054).ConclusionA support program like JASP-1 could be an effective way of not only supporting children newly diagnosed with JIA and their parents psychologically but may also increase children’s overall physical health and improve quality of care within pediatric rheumatology.Trial registrationRetrospectively registered in ClinicalTrials.gov, the 13th of February with ID NCT06284616.

Childhood Arthritis and Rheumatology Research Alliance Biologic Disease-Modifying Antirheumatic Drug Consensus Treatment Plans for Refractory Moderately Severe Juvenile Dermatomyositis.

The objective was to develop consensus treatment plans (CTPs) for patients with refractory moderately severe juvenile dermatomyositis (JDM) treated with biologic disease-modifying antirheumatic drugs (bDMARDs). The Biologics Workgroup of the Childhood Arthritis and Rheumatology Research Alliance JDM Research Committee used case-based surveys, consensus framework, and nominal group technique to produce bDMARD CTPs for patients with refractory moderately severe JDM. Four bDMARD CTPs were proposed: tumor necrosis factor α (TNFα) inhibitor (adalimumab or infliximab), abatacept, rituximab, and tocilizumab. Each CTP has different options for dosing and/or route. Among 76 respondents, consensus was achieved for the proposed CTPs (93% [67 of 72]) as well as for patient characteristics, assessments, outcome measures, and follow-up. By weighted average, respondents indicated that they would most likely administer rituximab, followed by abatacept, TNFα inhibitor, and tocilizumab. CTPs for the administration of bDMARDs in refractory moderately severe JDM were developed using consensus methodology. The implementation of the bDMARD CTPs will lay the groundwork for registry-based prospective comparative effectiveness studies.

Validation of the EQ-5D-Y-5L parent-proxy version among children with juvenile idiopathic arthritis

ObjectivesJuvenile idiopathic arthritis (JIA) is the most common type of arthritis among children. It can cause joint pain and permanent physical damage, which affects mobility and daily activities. The EQ-5D-Y-3L self-report version has been validated in JIA, but the validity of EQ-5D-Y-5L remains unknown. We examined the psychometric properties of the EQ-5D-Y-5L parent-proxy version among children with JIA.MethodsWe used data from the Understanding Childhood Arthritis Network Canadian-Dutch collaboration study cohort, including patients with new-onset JIA, and those starting or stopping biologics. Clinical data and the parent-proxy version of the childhood health assessment questionnaire (CHAQ) and EQ-5D-Y-5L were collected. We evaluated the ceiling and floor effect; convergent and divergent validity using Spearman’s rank correlation; known-group validity using one-way ANOVA (Analysis of Variance) and effect size; and informativity using Shannon’s evenness index.Results467 patient visits representing 407 patients were analyzed. The EQ-5D-Y-5L had no ceiling/floor effect. The EQ-5D-Y-5L showed good convergent (e.g., EQ-5D-Y-5L pain/discomfort dimension vs. CHAQ pain index (Spearman’s r = 0.74, 95% confidence interval (C.I.): 0.69–0.79)), divergent (e.g., EQ-5D-Y-5L pain/discomfort dimension vs. CHAQ eating dimension (Spearman’s r = 0.19, 95% C.I.: 0.09–0.29)) and known-group validity (e.g., mean EQ-5D-Y-5L level summary score for patients with inactive versus active disease status, 6.34 vs. 10.52 (p < 0.001, effect size = 1.20 (95% C.I.: 0.95–1.45)). Shannon’s evenness index ranged from 0.52 to 0.88, suggesting most dimensions had relatively even distributions.ConclusionsIn this patient sample, EQ-5D-Y-5L parent-proxy version exhibited construct validity and informativity, suggesting the EQ-5D-Y-5L can be used to measure the quality of life of children with JIA.

The epigenetic determinants for systemic juvenile idiopathic arthritis phenotyping and treatment response

BackgroundDetermining the role of epigenetics in systemic juvenile idiopathic arthritis (SJIA) provides an opportunity to explore previously unrecognized disease pathways and new therapeutic targets.AimWe aimed to identify the clinical significance of microRNAs (miRNA-26a, miRNA-223) in SJIA.Materials and methodsThis cross-sectional study was conducted on a group of children with SJIA attending to pediatric rheumatology clinic, at Mansoura University Children’s Hospital (MUCH) from December 2021 to November 2022. Patient demographics, and clinical, and laboratory data were collected with the measurement of microRNAs by quantitative real-time PCR. The Mann–Whitney, Kruskal–Wallis, and Spearman correlation tests were used for variable comparison and correlations, besides the receiver operating characteristic (ROC) curve for microRNAs disease activity and treatment non-response discrimination.ResultsForty patients were included in the study. On comparison of miRNA-26a, and miRNA-223 levels to the clinical, assessment measures, and laboratory features, miRNA-26a was statistically higher in cases with systemic manifestations versus those without. Similarly, it was higher in children who did not fulfill the Wallace criteria for inactive disease and the American College of Rheumatology (ACR) 70 criteria for treatment response. Meanwhile, miRNA-223 was not statistically different between cases regarding the studied parameters. The best cut-off value for systemic juvenile arthritis disease activity score-10 (sJADAS-10) and the ability of miRNA-26a, and miRNA-223 to discriminate disease activity and treatment non-response were determined by the (ROC) curve.ConclusionThe significant association of miRNA-26a with SJIA features points out that this molecule may be preferentially assessed in SJIA disease activity and treatment non-response discrimination.

Overview of the Treatment Goal, the Method of Evaluating Disease Activity/Physical Function, Activities of Daily Living, and Traditional Care for Systemic or Articular Juvenile Idiopathic Arthritis in Japan.

Juvenile idiopathic arthritis (JIA) is a chronic arthritis of unknown cause that develops in patients younger than 16 years of age and persists for at least 6 weeks. It is an important cause of short- and long-term physical and mental impairments in children. The goal of treatment for JIA is remission. A T2T (treatment-to-target) has been proposed and practiced as a means of achieving remission. The method of evaluating the disease activity of JIA depends on the disease type. For systemic JIA, disease activity is determined by comprehensively considering joint findings, systemic inflammatory findings, changes in inflammatory and synovitis markers, imaging findings, and other factors. For articular JIA other than systemic JIA, the Juvenile Arthritis Disease Activity Score (JADAS-27) is used to evaluate disease activity. The CHAQ (Childhood Health Assessment Questionnaire) and the Japanese version of the modified Rankin Scale (mRS) are mainly used to assess the physical function and ADL. The CHAQ is a global standard assessment method with the advantage that it can be transitioned to the HAQ used in adults, making it useful for international comparisons. The mRS is used to classify the severity of JIA as a chronic disease, and is an indispensable evaluation method in the specific disease procedure in Japan. It is necessary to have pediatric-specific knowledge of growth and development and routine childhood immunizations and to consider transition support tailored to the patient's situation. Ultimately, the goal is to foster the patient's independence and to provide an uninterrupted follow-up in the adult care department. Continuous follow-up will be provided during the schooling (and later, employment) period, and the relationship with the patient will be tailored to their developmental stage. It is also important to understand and communicate the importance of contraception and the drugs that cannot be used during pregnancy.

Real-life data of etanercept efficacy and safety in juvenile idiopathic arthritis: a 24-month retrospective study at a single center

ABSTRACT Objective The aim of this study was to assess the efficacy and safety of etanercept (ETA) use in juvenile idiopathic arthritis (JIA). Methods The 24-month data of patients with JIA on etanercept in a single center were evaluated retrospectively. Response to treatment was assessed according to 10-joint Juvenile Arthritis Disease Activity Score (JADAS10), and JIA-American College of Rheumatology (ACR) improvement criteria. Safety assessments were based on adverse event (AE) reports. Results The study included 152 patients with JIA. The mean age at diagnosis of JIA was 8.5 ± 4.4 years, and treatment with ETA started at a mean age of 11.1 ± 4.4 years. The mean duration of ETA use was 16 ± 11.1 months. The mean JADAS10 score at baseline was 18.5 ± 5.9. By the third month, it had reduced to 8.6 ± 6.6 and by the sixth month to 5.7 ± 6. By the twelfth month, the JADAS10 score was 4.9 ± 6.7, and by the twenty-fourth month, it had worsened to 7.3 ± 7.8. ACR50 response was achieved in 79.6% of patients at 3 months, 67.1% at 6 months, 79.3% at twelfth months, 70.7% at the twenty-fourth month. During ETA treatment, 10 patients required hospitalization for serious infections. Conclusion Etanercept is a safe and effective option for patients with JIA. However, variations in response between JIA subtypes highlight the need for individualized treatment strategies.

Intravenous Golimumab in Children With Polyarticular-Course Juvenile Idiopathic Arthritis: Long-Term Extension of an Open-Label Phase III Study.

To report pharmacokinetics (PK), immunogenicity, clinical effect, and safety of intravenous (IV) golimumab in children with active polyarticular-course juvenile idiopathic arthritis (pcJIA) who participated in A Study to Evaluate the Pharmacokinetics, Efficacy and Safety of Intravenous Golimumab in Pediatric Participants With Active Polyarticular Course Juvenile Idiopathic Arthritis Despite Methotrexate Therapy (GO-VIVA)'s open-label, long-term extension (LTE) through week 252. GO-VIVA participants who continued IV golimumab (80 mg/m2 every 8 weeks) after week 52 were included. PK and safety were assessed through week 244 (last dose) and week 252, respectively, and clinical response through week 116. Clinical outcomes included JIA-American College of Rheumatology (ACR) responses and clinical Juvenile Arthritis Disease Activity Score in 10 joints (cJADAS10). Binary outcomes used nonresponder imputation, and other descriptive analyses used observed data. Of 112/127 (88.2%) participants entering the LTE, 69 completed the week 252 visit. Median steady-state trough golimumab concentrations were generally maintained from week 52 through week 244 (range 0.3-0.6 μg/mL). Antigolimumab antibody rates were consistent through week 52 (39.2% [49/125]) and week 244 (44.8% [56/125]). Week 52 JIA-ACR 30/50/70/90 response rates (75.6% [96/127], 74% [94/127], 65.4% [83/127], and 48.8% [62/127], respectively) were generally maintained through week 116 (72.4% [92/127], 71.7% [91/127], 63.8% [81/127], and 50.4% [64/127], respectively), when the median cJADAS10 was 1.6 and 56.7% (72/127) of participants achieved cJADAS10 ≤ 5 (minimal disease activity). Rates (per 100 patient-years) of serious adverse events and serious infections through week 252 were 7.7 and 3.9, respectively. GO-VIVA LTE participants experienced adequate PK exposure and stable safety and immunogenicity. The majority of participants experienced no more than minimal residual disease activity. Data suggest IV golimumab treatment provided durable clinical response through week 116, with an acceptable risk-benefit profile.

COEXISTENCE OF GOLDENHAR SYNDROME WITH JUVENILE IDIOPATHIC ARTHRITIS: A CAUSAL LINK OR MERE COINCIDENCE?

Goldenhar syndrome, a rare congenital anomaly primarily affecting the face, external ear and vertebrae, and juvenile idiopathic arthritis (JIA), a chronic inflammatory disease in children under 16, represent distinct and uncommon clinical entities. We report the case of a five-and-a-half-year-old girl, with a history of treated congenital hip dislocation, admitted for persistent joint pain for over 6 months, affecting both knees, wrists and ankles with inflammatory signs without associated visceral manifestations. Clinical examination revealed a dysmorphic facies characteristic of Goldenhar syndrome, including facial asymmetry with mandibular hypoplasia, auricular anomalies such as a bilateral pre-tracheal appendix, an acrochordon on the right cheek, a pre-auricular sinus, microtia, and a closed right external auditory canal. A lumbar scoliosis was also identified on dorsolumbar spine imaging, with a CT scan of the rock that objectified an imperforation of the right external auditory canal and lack of visualization of the tympanic membrane, consolidating the diagnosis of Goldenhar syndrome.Biologically, the patient presented with inflammatory anemia with a moderate inflammatory syndrome, as well as hypergammaglobulinemia detected by protein electrophoresis. Tests for antinuclear antibodies and rheumatoid factor were negative (15 IU/mL). X-rays of the affected joints were taken, and the diagnosis of Idiopathic Juvenile Arthritis was retained after eliminating all differential diagnoses. This unusual coexistence raises complex diagnostic and therapeutic challenges, requiring a multidisciplinary approach and ongoing surveillance for comprehensive management.

Assessing disparities through missing race and ethnicity data: results from a juvenile arthritis registry.

Ensuring high-quality race and ethnicity data within the electronic health record (EHR) and across linked systems, such as patient registries, is necessary to achieving the goal of inclusion of racial and ethnic minorities in scientific research and detecting disparities associated with race and ethnicity. The project goal was to improve race and ethnicity data completion within the Pediatric Rheumatology Care Outcomes Improvement Network and assess impact of improved data completion on conclusions drawn from the registry. This is a mixed-methods quality improvement study that consisted of five parts, as follows: (1) Identifying baseline missing race and ethnicity data, (2) Surveying current collection and entry, (3) Completing data through audit and feedback cycles, (4) Assessing the impact on outcome measures, and (5) Conducting participant interviews and thematic analysis. Across six participating centers, 29% of the patients were missing data on race and 31% were missing data on ethnicity. Of patients missing data, most patients were missing both race and ethnicity. Rates of missingness varied by data entry method (electronic vs. manual). Recovered data had a higher percentage of patients with Other race or Hispanic/Latino ethnicity compared with patients with non-missing race and ethnicity data at baseline. Black patients had a significantly higher odds ratio of having a clinical juvenile arthritis disease activity score (cJADAS10) of ≥5 at first follow-up compared with White patients. There was no significant change in odds ratio of cJADAS10 ≥5 for race and ethnicity after data completion. Patients missing race and ethnicity were more likely to be missing cJADAS values, which may affect the ability to detect changes in odds ratio of cJADAS ≥5 after completion. About one-third of the patients in a pediatric rheumatology registry were missing race and ethnicity data. After three audit and feedback cycles, centers decreased missing data by 94%, primarily via data recovery from the EHR. In this sample, completion of missing data did not change the findings related to differential outcomes by race. Recovered data were not uniformly distributed compared with those with non-missing race and ethnicity data at baseline, suggesting that differences in outcomes after completing race and ethnicity data may be seen with larger sample sizes.