OBJECTIVE:To explore congruence between child self-reported and caregiver-proxy-reported health-related quality of life (HRQOL) over time in juvenile idiopathic arthritis (JIA) and childhood-onset systemic lupus erythematosus (cSLE), and to identify factors associated with the level of congruence. METHODS:Data were from an observational, longitudinal cohort study conducted to validate the Patient-Reported Outcomes Measurement Information System (PROMIS) measures. HRQOL was assessed at baseline, 6, and 12 months. Four hundred fifty-one children (8-17 years) diagnosed with JIA or cSLE and their caregivers completed the PROMIS Pediatric and Parent Proxy measures, respectively. A 1-way random-effects model was used to estimate the intraclass correlation coefficient (ICC) for congruence between child and caregiver reports, and multivariable mixed-effect models were used to identify associated demographic and clinical factors. RESULTS:The study cohort (87.1% JIA) had a mean age of 13.8 years and were 71.4% female. Across all HRQOL domains, child self-reported and caregiver-proxy-reported mobility, physical activity, fatigue, pain interference, depressive symptoms, and psychological stress had moderate associations (ICC 0.50-0.68), whereas child self-reported and caregiver-proxy-reported family relationships and anxiety were weakly associated (ICC 0.34-0.42). Older children had higher congruence with their caregivers on symptom domains (0.25 to 0.75 points) than younger children; female children had higher congruence with their caregivers on psychological symptoms (-2.20 to -1.98 points) than male children. CONCLUSION:Caregivers provide complementary information on the physical aspects of HRQOL, with a tendency to estimate worse symptoms and decreased functioning. Child self-report remains the gold standard for understanding HRQOL in pediatric populations with rheumatic diseases.

BACKGROUND:Pediatric skin-limited discoid lupus erythematosus (DLE-only) is rare, with limited data on risk factors for progression to systemic lupus erythematosus (SLE). OBJECTIVE:To assess incidence, risk factors, and phenotype of pediatric DLE-only progression to SLE. METHODS:In this 17-site retrospective cohort of pediatric DLE, the primary outcome was time to SLE diagnosis (American College of Rheumatology classification criterion ≥4). Kaplan-Meier estimates for 1-, 2-, and 5-year progression to SLE were generated. Cox proportional hazards modeling identified baseline predictors of progression to SLE. RESULTS:The 1-year progression rate from DLE-only to SLE was 14.4% (95% CI, 9.6-18.9). Progression to SLE was most strongly associated with baseline antinuclear antibody positivity (hazard ratio, 3.71) and older age (hazard ratio, 1.11/y). Antiphospholipid antibodies and cytopenias also predicted progression to SLE in multivariable analysis. The SLE phenotype was relatively mild, with most patients meeting mucocutaneous and laboratory criteria (22/236, 9%) and a few patients in whom other end-organ diseases developed (7/236, 3%). LIMITATIONS:Retrospective design, missing data. CONCLUSION:Antinuclear antibody-positive patients with DLE-only warrant close monitoring for progression to SLE, especially within the first year. Severe end-organ disease in patients with DLE who progress to SLE is uncommon. Future studies should test whether early recognition and intervention in DLE-only slows progression to SLE.

Background/Objectives: Despite the growing popularity of the topic of wellness in society, children with chronic illnesses are rarely introduced to the concept. Wellness may be an unexpected and complex topic for a medical visit, especially for those living with chronic medical conditions. Our goal is to intentionally design an individualized referral process to wellness services for children with chronic illnesses. Methods: Human-Centered Design (HCD) methods were utilized to understand patient, caregiver and provider needs and challenges when making wellness service referrals. Stakeholders participated in workshops and interviews, which informed the design of a referral prototype. The referral prototype was evaluated through simulations and was pilot-tested in a new Center. Results: Optimal referrals to wellness services are best delivered through conversations that are compassionate, relational, respectful and motivating. We developed operational, contextual and experiential Design Requirements that informed a personalized “Wellness Conversation” to create an experience that was distinct from a medical visit. The conversation follows a four-step framework: trust and rapport building, assessment of current state of wellness, prioritization of wellness areas, and establishment of goals in wellness planning. Conclusions: HCD allowed us to produce a referral prototype with high perceived acceptability, feasibility and fidelity. These findings indicate that approaching referrals to wellness services as a conversation may help create a more positive, supportive, and hope-inspiring experience for children and families.

OBJECTIVE:Social determinants of health (SDOH) contribute to juvenile idiopathic arthritis (JIA) disparities, but most studies have assessed SDOH independently rather than cumulatively across individual, family, and neighborhood levels. Using a socioecological framework, we investigated the relationship among cumulative social disadvantage, neighborhood disadvantage, and JIA disease activity. METHODS:We conducted a retrospective cohort study using the Childhood Arthritis and Rheumatology Research Alliance (CARRA) Registry (2015-2022). Individual- and family-level SDOH were combined into a cumulative social disadvantage score (range: 0-3), with one point each for public or no insurance, family income less than $50,000 per year, and guardian education of high school level or less. Neighborhood disadvantage was measured using Area Deprivation Index (quartiles). The primary outcome was active disease by clinical Juvenile Arthritis Disease Activity-10 (cJADAS-10). Mixed effects models were generated to assess associations adjusted for demographic and clinical covariates, and causal mediation analysis evaluated whether cumulative social disadvantage mediated the relationship between neighborhood disadvantage and disease activity. RESULTS:Among 9,609 children with JIA with a median follow-up time of 2.1 (interquartile range 0.3-5.8) years, 39.1% had a cumulative social disadvantage score greater than 0 with higher scores correlating with greater neighborhood disadvantage. In adjusted analysis, cumulative social disadvantage was associated with higher odds of active disease (adjusted odds ratio 2.10, 95% confidence interval [CI] 1.73-2.53 for a score of 3 vs 0). A total of 87% (95% CI 38-100) of the effect of neighborhood disadvantage was mediated through cumulative social disadvantage. CONCLUSION:Cumulative social disadvantage is strongly associated with active JIA and mediates much of the effect of neighborhood disadvantage. Interventions addressing multilevel SDOH will be essential to reducing JIA health disparities.

OBJECTIVE:Race and household income impact outcomes in patients with rheumatic conditions; however, their role in pediatric antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis (AAV) remains poorly understood. We aimed to evaluate whether race and ethnicity and household income are associated with severe AAV disease and renal outcomes among pediatric patients in the United States. METHODS:We used the 2016 and 2019 Kids' Inpatient Database (KID) to identify pediatric AAV hospitalizations. Severe AAV disease was defined by International Classification of Diseases, Tenth Revision codes indicating organ failure, complications, or the use of life-sustaining procedures. We evaluated associations among race and ethnicity and household income quartile with severe disease, end-stage renal disease (ESRD), and renal transplantation among those with ESRD using univariate and multivariable logistic regression. RESULTS:Among the 673 unweighted AAV hospitalizations, 59% involved at least one severe disease feature. Hispanic race or ethnicity was associated with higher odds of severe disease (adjusted odds ratio [aOR] 1.61; 95% confidence interval [CI] 1.01-2.56) and ESRD (aOR 1.98; 95% CI 1.04-3.77). Among patients with ESRD, those in the lowest-income quartile had significantly lower odds of renal transplantation compared with those in the highest quartile (aOR 0.18; 95% CI 0.04-0.71). CONCLUSION:In this national sample of hospitalized pediatric patients with AAV, Hispanic race or ethnicity was a statistically significant predictor of severe AAV disease and ESRD, and household income was inversely associated with renal transplantation among those with ESRD. These findings suggest that race and household income may contribute to health inequities in pediatric AAV.

OBJECTIVE:Children and adolescents living with juvenile idiopathic arthritis (JIA) and childhood-onset systemic lupus erythematosus (cSLE) frequently experience mental health comorbidities. This study evaluated sex differences in symptoms of depression, anxiety, and psychological stress in JIA and cSLE. METHODS:This multicenter, prospective cohort study recruited children and adolescents from the Childhood Arthritis and Rheumatology Research Alliance (CARRA) Registry. Disease activity and Patient-Reported Outcomes Measurement Information System (PROMIS) pediatric self-report measures of Depressive Symptoms and Anxiety were collected at 3 timepoints over 12 months, and Psychological Stress was collected at baseline. Differences by sex were tested using chi-square and Wilcoxon rank-sum tests. Linear mixed effect models (LMMs) were created for each PROMIS measure to evaluate differences by sex. The prespecified α was 0.05. RESULTS:Among 393 children/adolescents with JIA and 58 children/adolescents with cSLE, Depressive Symptoms, Anxiety, and Psychological Stress scores were higher (indicating poorer mental health symptoms) for girls than boys. At baseline, approximately 1 in 3 girls with JIA and 1 in 2 girls with cSLE had moderate-to-severe Depressive Symptoms and Psychological Stress, compared to approximately 1 in 6 boys with JIA or cSLE. LMMs showed significantly higher scores (indicating poorer symptoms) for girls than boys, generally exceeding the minimally important difference threshold. CONCLUSION:Girls self-reported worse symptoms of depression, anxiety, and psychological stress compared to boys. Significant sex differences persisted after adjusting for rheumatic disease activity, time, and other pertinent variables. Mental health screening, management, and interventions may need to be tailored by sex.

OBJECTIVES:Childhood-onset systemic lupus erythematosus (cSLE), representing 15%-20% of individuals with SLE, has been difficult to study globally due to differences between registries. This initiative, supported by Childhood Arthritis Rheumatology Research Alliance (CARRA) and Paediatric Rheumatology European Society (PReS), aims to create Core and Expanded cSLE Datasets to standardise and enhance research worldwide. METHODS:21 international cSLE experts and 4 patients participated in a Delphi process (questionnaires, 2 topic-specific focus groups and 3 virtual consensus meetings) to create 2 standardised cSLE datasets. The Core cSLE Dataset was designed to include data essential to meaningful clinical research across many settings. The Expanded cSLE Dataset was designed for centres able to consistently collect data to address broader research questions. Final data items for the Core and Expanded datasets were determined by consensus defined as >80% agreement) using an adapted nominal group technique and voting. RESULTS:The resulting Core cSLE Dataset contains 46 items, including demographics, clinical features, laboratory results, medications and significant adverse events. The Expanded cSLE Dataset adds 26 additional items and includes patient-reported outcomes. Consensus was also achieved regarding the frequency and time points for data collection: baseline, quarterly follow-up visits, annually and flare visits. CONCLUSION:Standardised Core and Expanded cSLE Datasets for registry-based international cSLE research were defined through the consensus of global experts and patient/caregiver representatives, endorsed by CARRA and PReS. These datasets incorporate disease-specific and patient-specific features, optimised for diverse settings to facilitate international collaborative research for children and adolescents with SLE worldwide.

Objective Using data from participants with paediatric SLE (pSLE) in the Childhood Arthritis and Rheumatology Research Alliance Registry, we aimed to: (1) describe 2-year disease activity trajectories, measured by the SLE Disease Activity Index 2000 (SLEDAI 2K); (2) identify characteristics associated with each trajectory and (3) assess achievement of lupus low disease activity state (LLDAS) and associated baseline characteristics.Methods Participants were diagnosed with pSLE within 12 months of baseline visit. Baseline sociodemographic, clinical and treatment characteristics were included in latent trajectory analyses. Associations between patient characteristics with trajectory groups and LLDAS were analysed with multinomial generalised logistic regression modelling.Results 1002 patients were screened; 553 were included for SLEDAI 2K and 269 for LLDAS analyses. SLEDAI 2K trajectories included (T1) low and stable, (T2) high and decreasing, (T3) intermediate and stable. In multinomial generalised logistic regression, baseline SLEDAI 2K score and insurance type were significantly associated with trajectories. 51% (136/269) of patients achieved LLDAS at least once in 24 months as compared with 17% (47/269) at first assessment. LLDAS attainment at both time points was predicted by lower pain interference scores; LLDAS attainment over 24 months was also associated with baseline American College of Rheumatology classification criteria, rituximab use at baseline and highest completed level of parent/guardian education.Conclusions Disease activity trajectories in a pSLE cohort were predicted by baseline SLEDAI 2K and insurance. Only half of the patients achieved LLDAS during the 2-year study period, which was predicted by baseline characteristics including pain interference. The relationship between disease activity and socioeconomic factors and pain warrants further investigation to identify modifiable factors to reduce pSLE disease activity.

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

OBJECTIVE:Despite treatment advances, pain remains a serious problem for many children with juvenile idiopathic arthritis (JIA). To better understand pain in children with JIA and identify potentially modifiable factors, this study evaluated Patient-Reported Outcomes Measurement Information System (PROMIS) Pediatric Pain Interference (PI) and its relationships with other pain measures and demographic, clinical, psychosocial, and functional variables. METHODS:This cross-sectional, observational, multicenter study used descriptive statistics and a mix of bivariate and multivariable analyses to describe PI and characterize relationships with other measures and variables. RESULTS:Among 355 children with JIA, 27% reported moderate or severe PI and 13.3% reported daily pain. PI correlated with other pain measures. Increasing age, decreasing disease duration, and increasing number of active joints, as well as presence of active disease, steroid treatment, and biologic treatment, were associated with greater PI. All PROMIS psychosocial and functional measures were associated with PI in the expected direction except for PROMIS Pediatric Physical Activity, which showed no association. In multivariable analyses, only PROMIS Fatigue, PROMIS Mobility, and the exploratory interaction of PROMIS Anxiety and disease-modifying antirheumatic drug treatment were significant. CONCLUSION:Moderate and severe PI was prevalent in this sample of children with JIA. PI increased with age and indicators of disease activity, but was more strongly associated with increasing fatigue and decreasing mobility. Findings support the use of PI as a short, easily administered multidimensional pain measure as part of routine clinical care. Fatigue, mobility, and disease activity should be assessed further when PI is high.

Background/Objective: We sought to understand healthcare utilization and barriers to care among youth with chronic illness who interact frequently with the healthcare system. Methods: This was a retrospective analysis of healthcare utilization for youth ≤25 years of age with chronic illness during one calendar year (1 January 2021–31 December 2021) in a single urban academic healthcare system. Inclusion criteria were (1) having at least one healthcare encounter in the calendar year of 2021 and (2) having at least six healthcare encounters over the preceding 3-year period or having a qualifying chronic illness. Demographic and clinical characteristics were collected along with self-reported and derived social determinants of health. Univariable and multivariable regression models were created to identify predictors of missed clinic visits, telehealth use, and activated patient portal accounts. Results: The cohort (N = 14,245) was demographically, clinically, and socioeconomically diverse. The youth had frequent clinic visits (median 9, IQR 4–18), multiple subspecialty care referrals (median 4, 1–8), were prescribed multiple medications (median 6, 3–10), and a high proportion received emergency department (18%) or inpatient treatment (15%). Race and public insurance were significant predictors of missed clinic visits and telehealth use. Primary language was a significant predictor of patient portal activation. Conclusions: Youth with chronic illness who are high users of the healthcare system face a high burden of clinic, emergency room, and hospital visits, referrals, and medications. Systematic efforts to lower the healthcare burden and improve care access should address existing racial and socioeconomic disparities affecting this patient population, who are likely to need frequent healthcare over their lifetime.

Background Differential glucocorticoid exposure and related toxicity may exacerbate racial and ethnic disparities in lupus-related organ damage and mortality. Pediatric-onset systemic lupus erythematosus (pSLE) confers an even greater lifelong burden of cumulative medication exposure than adult-onset disease. Access to care and other social determinants of health may drive differential medication use. Therefore, we sought to determine how race and social determinants of health associate with cumulative glucocorticoid exposure over time in children with SLE. We hypothesized that minoritized race and living in more disadvantaged neighborhoods would be associated with greater average oral glucocorticoid exposure among children with pSLE in the Childhood Arthritis and Rheumatology Research Alliance (CARRA) Registry. Methods This was a retrospective cohort study of children with pSLE enrolled in the CARRA Registry between March 2017-December 2021 with a baseline enrollment visit and ≥1 follow up Registry visits as well as a valid U.S. zip code. The primary exposures were self-identified race and/or ethnicity and national area deprivation index (ADI) linked to census tract. Time-averaged mean prednisone dose (mg/day) was used as the primary measure of cumulative glucocorticoid exposure, calculated using oral prednisone- equivalent milligram doses at each Registry visit. As secondary outcomes, we also evaluated occurrence of any prednisone restarts or dose increases between Registry visits during the study period and disease activity scores (SLEDAI-2K) over time. Associations between the primary exposures and time-averaged mean glucocorticoid dose were examined using univariate and multivariable linear regression models, adjusted for covariates (insurance status, age at enrollment, sex, major organ involvement, medication use at enrollment, disease duration at enrollment, and any secondary rheumatologic disease). We used logistic regression to analyze prednisone restart or dose increase. Linear mixed effects models were used to model disease activity over time with a subject-level random intercept. Results A racially diverse cohort of 540 children with pSLE self-identified as 11% Asian, 27% Black, 23% Latino/a, 5% Other race, 25% White, and 9% selected more than one race and/or ethnicity. The median age at diagnosis was 14.2 years (IQR 3.2–19.0), and 46% of participants were publicly insured. Black participants were more likely to be publicly insured, live in areas with higher social deprivation (ADI), and have renal disease (table 1). In univariate analyses, Black race and living in areas in the highest ADI quartile were significantly associated with higher time-averaged mean prednisone dose (unadjustedβ=3.24, 95% CI: [0.88, 5.60] and β=4.68, 95% CI: [2.30, 7.06], respectively). ADI remained significantly associated with time-averaged mean prednisone when adjusted for race and disease duration at enrollment (adjusted β=3.21, 95% CI: [0.68, 5.75]). However, further adjustment for either insurance status or renal involvement attenuated this estimate, and neither race nor the highest quartile of ADI were significantly associated with time-averaged mean prednisone in the fully adjusted model (table 2). Black race was also associated with higher odds of prednisone dose increases or restarts in unadjusted analysis (OR=1.8, 95% CI: [1.1, 2.9], p=0.022) and approached but did not reach statistical significance at the 0.05 level in the fully adjusted model (OR= 1.7, 95% CI: [0.99, 3.1], p=0.055). Black race was independently associated with higher adjusted disease activity compared to White race (adjusted β=0.94, 95% CI: [0.11, 1.78]). Compared to privately insured children, uninsured children also had significantly higher disease activity, albeit the number of uninsured children was small (table 3). Conclusions Insurance status and renal disease were significant predictors of greater cumulative mean prednisone dose among children with pSLE in the CARRA Registry and attenuated the associations between Black race and neighborhood-level disadvantage with prednisone dose. In contrast, Black race was independently associated with higher disease activity over time, irrespective of renal disease, neighborhood-level disadvantage, or insurance status, suggesting that there may be additional unmeasured social determinants driving this association. Furthermore, given that Black participants were much more likely to be publicly insured and live in areas of higher neighborhood disadvantage, structural confounding due to the unequal segregation of Black participants into areas of high neighborhood disadvantage must be considered. Further work to understand the complex relationships between area-level segregation and individual social determinants of health or how they mediate racial disparities is needed to identify points of intervention to improve outcomes of pSLE.

BACKGROUND/OBJECTIVES:Integrated care models, like combined rheumatology/dermatology clinics (RDCs), facilitate efficient coordination between specialists and provide comprehensive care. Given the limited literature on pediatric RDC logistics, outcomes, benefits and, challenges, we comprehensively characterized our patient cohort at the UCSF Benioff Children's Hospital RDC and surveyed pediatric dermatologists participating in RDCs. METHODS:We retrospectively reviewed 71 patients new to the UCSF Pediatric RDC between September 2017 and September 2023. A survey was distributed in 2024 to 17 dermatologists in North America, each representing a unique pediatric RDC. RESULTS:69% of patients (49/71) were female. Seventeen (24%) presented without a known diagnosis; the first RDC visit established a diagnosis for 7 of them (41%). Of patients with a previously established diagnosis, initial RDC evaluation confirmed it in 52 (96%) and revised it for 2 (4%). The most encountered diagnoses were linear morphea (33%), lupus (23%), and psoriasis (13%). New systemic therapy was prescribed for 23% of patients, and additional work-up was recommended via skin biopsy (8%) and imaging (28%). Survey results revealed all pediatric RDCs include trainees, but only 59% (10/17) receive administrative support. All agreed that RDCs are valuable for patient care and most (15/17, 88%) felt that the RDC was a valuable use of their time. CONCLUSIONS:Pediatric RDCs are valuable for consensus diagnosis, streamlined evaluation, and management of complex patients. Though clinical and administrative support for RDCs is generally poor, RDCs are valuable to patients, a good use of time for clinicians, and offer educational opportunities for team members.