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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.

To provide updated guidelines for pharmacologic management of juvenile idiopathic arthritis (JIA), focusing on treatment of oligoarthritis, temporomandibular joint (TMJ) arthritis, and systemic JIA with and without macrophage activation syndrome. Recommendations regarding tapering and discontinuing treatment in inactive systemic JIA are also provided. We developed clinically relevant Patient/Population, Intervention, Comparison, and Outcomes questions. After conducting a systematic literature review, the Grading of Recommendations Assessment, Development and Evaluation approach was used to rate the quality of evidence (high, moderate, low, or very low). A Voting Panel including clinicians and patients/caregivers achieved consensus on the direction (for or against) and strength (strong or conditional) of recommendations. Similar to those published in 2019, these JIA recommendations are based on clinical phenotypes of JIA, rather than a specific classification schema. This guideline provides recommendations for initial and subsequent treatment of JIA with oligoarthritis, TMJ arthritis, and systemic JIA as well as for tapering and discontinuing treatment in subjects with inactive systemic JIA. Other aspects of disease management, including factors that influence treatment choice and medication tapering, are discussed. Evidence for all recommendations was graded as low or very low in quality. For that reason, more than half of the recommendations are conditional. This clinical practice guideline complements the 2019 American College of Rheumatology JIA and uveitis guidelines, which addressed polyarthritis, sacroiliitis, enthesitis, and uveitis. It serves as a tool to support clinicians, patients, and caregivers in decision‐making. The recommendations take into consideration the severity of both articular and nonarticular manifestations as well as patient quality of life. Although evidence is generally low quality and many recommendations are conditional, the inclusion of caregivers and patients in the decision‐making process strengthens the relevance and applicability of the guideline. It is important to remember that these are recommendations. Clinical decisions, as always, should be made by the treating clinician and patient/caregiver.

To provide recommendations for the management of juvenile idiopathic arthritis (JIA) with a focus on nonpharmacologic therapies, medication monitoring, immunizations, and imaging, irrespective of JIA phenotype. We developed clinically relevant Patient/Population, Intervention, Comparison, and Outcomes questions. After conducting a systematic literature review, the Grading of Recommendations Assessment, Development and Evaluation approach was used to rate the quality of evidence (high, moderate, low, or very low). A Voting Panel including clinicians and patients/caregivers achieved consensus on the direction (for or against) and strength (strong or conditional) of recommendations. Recommendations in this guideline include the use of physical therapy and occupational therapy interventions; a healthy, well‐balanced, age‐appropriate diet; specific laboratory monitoring for medications; widespread use of immunizations; and shared decision‐making with patients/caregivers. Disease management for all patients with JIA is addressed with respect to nonpharmacologic therapies, medication monitoring, immunizations, and imaging. Evidence for all recommendations was graded as low or very low in quality. For that reason, more than half of the recommendations are conditional. This clinical practice guideline complements the 2019 American College of Rheumatology JIA and uveitis guidelines, which addressed polyarthritis, sacroiliitis, enthesitis, and uveitis, and a concurrent 2021 guideline on oligoarthritis, temporomandibular arthritis, and systemic JIA. It serves as a tool to support clinicians, patients, and caregivers in decision‐making. The recommendations take into consideration the severity of both articular and nonarticular manifestations as well as patient quality of life. Although evidence is generally low quality and many recommendations are conditional, the inclusion of caregivers and patients in the decision‐making process strengthens the relevance and applicability of the guideline. It is important to remember that these are recommendations. Clinical decisions, as always, should be made by the treating clinician and patient/caregiver.

To describe the character and composition of the 2015 pediatric rheumatology workforce in the US, evaluate current workforce trends, and project future supply and demand of the pediatric rheumatology workforce through 2030. The American College of Rheumatology created the workforce study group to study the rheumatology workforce. The workforce study group used primary and secondary data to create a representative workforce model. Pediatric rheumatology supply and demand was projected through 2030 using an integrated data‐driven framework to capture a more realistic clinical full‐time equivalent (FTE) and produce a better picture of access to care issues in pediatric rheumatology. The 2015 pediatric rheumatology workforce was estimated at 287 FTEs (300 providers), while the estimated excess demand was 95 (33%). The projected demand will continue to increase to almost 100% (n = 230) by 2030 if no changes occur in succession planning, new graduate entrants into the profession, and other factors associated with the workforce. This study projects that the pediatric rheumatology workforce gap will continue to worsen significantly from the 2015 baseline, and by 2030 the demand for pediatric rheumatologists will be twice the supply. Innovative strategies are needed to increase the workforce supply and to improve access to care.

To examine glucocorticoid‐sparing immunomodulatory medication use in youth with systemic lupus erythematosus (SLE) during their first year of care. We conducted a retrospective cohort study using administrative claims for 2000 to 2013 from Clinformatics DataMart for youth ages 10–24 years with an incident diagnosis of SLE (≥3 International Classification of Diseases, Ninth Revision codes for SLE [710.0], each >30 days apart). We determined the proportion of subjects filling a prescription for immunomodulatory medications within 12 months of the first SLE code (index date). We used multivariable regression to examine associations between demographic/disease factors and time to prescription fill in the first year, and also between prescription fill at any time after the index date. We identified 532 youth with an incident SLE diagnosis, of which 413 (78%) had a glucocorticoid‐sparing immunomodulatory prescription fill in the first year. Prescriptions for hydroxychloroquine and immunosuppressants were filled in the first year by 366 youth (69%) and by 182 (34%), respectively. Those with adult‐onset (versus childhood‐onset) disease were less likely to fill an immunomodulatory medication by 12 months. No other statistically significant associations were found, although there was increasing likelihood of immunomodulatory medication fills with each subsequent calendar year. Among youth with newly diagnosed SLE, hydroxychloroquine use is prevalent although not universal, and prescription immunosuppressant use is notably low during the first year of care. Further research is needed to identify factors contributing to suboptimal immunomodulatory medication use during the first year of care.

Youth with systemic lupus erythematosus (SLE) experience high rates of psychiatric comorbidities, which may affect medication adherence. We undertook this study to examine the association between psychiatric disorders and hydroxychloroquine adherence and to determine whether psychiatric treatment modifies this association. We identified incident hydroxychloroquine users among youth with SLE (ages 10–24 years) using de‐identified US commercial insurance claims in Optum Clinformatics Data Mart (2000–2016). Adherence was estimated using medication possession ratios (MPRs) over a 365‐day time period. Multivariable linear regression models were used to estimate the effect of having any psychiatric disorder on MPRs, as well as the independent effects of depression, anxiety, adjustment, and other psychiatric disorders. We tested for interactions between psychiatric diagnoses and treatment with psychotropic medications or psychotherapy. Among 873 subjects, 20% had a psychiatric diagnosis, most commonly depression. Only adjustment disorders were independently associated with decreased MPRs (β –0.12, P= 0.05). We observed significant crossover interactions, in which psychiatric disorders had opposite effects on adherence depending on the receipt of psychiatric treatment. Among youth with any psychiatric diagnosis, psychotropic medication use was associated with a 0.15 increase in the MPR compared with no psychotropic medication use (P= 0.02 for interaction). Among youth with depression or anxiety, psychotherapy was also associated with a higher MPR compared with no psychotherapy (P= 0.05 and P< 0.01 for interaction, respectively). The impact of psychiatric disorders on medication adherence differed by whether youth had received psychiatric treatment. Improving recognition and treatment of psychiatric conditions may increase medication adherence in youth with SLE.

To validate the preliminary criteria of global flare for childhood‐onset SLE(cSLE). Pediatricians experienced in cSLEcare (n = 268) rated unique patient profiles; results of standard cSLElaboratory testing and information about the cSLEflare descriptors were presented as follows: global assessment of patient well‐being, physician global assessment of disease activity (MD‐global), Disease Activity Index score, protein/creatinine ratio (PCR), and erythrocyte sedimentation rate (ESR). Using rater interpretation of the course of cSLE(baseline versus followup as the gold standard), performance (sensitivity, specificity, area under the receiver operating characteristic curve [AUC]) of the preliminary flare criteria was tested. An international consensus conference was held to rank the preliminary flare criteria as per the American College of Rheumatology recommendations and delineate threshold scores for minor, moderate, and major flares. The accuracy of the 2 highest‐ranked candidate criteria that consider absolute changes (∆) of the Systemic Lupus Erythematosus Disease Activity Index (SLEDAI) or British Isles Lupus Assessment Group (BILAG) (numeric scoring: A = 12, B = 8, C = 1, and D/E = 0), MD‐global, PCR, and ESRwere confirmed (both AUC>0.93). For the SLEDAI‐based criteria (0.5 × ∆SLEDAI+ 0.45 × ∆PCR+ 0.5 × ∆MD‐global + 0.02 × ∆ESR) flare scores ≥6.4/3.0/0.6 constituted major/moderate/minor flares, respectively. For the BILAG‐based algorithm (0.4 × ∆BILAG+ 0.65 × ∆PCR+ 0.5 × ∆MD‐global + 0.02 × ∆ESR) flare scores ≥7.4/3.7/2.2 delineated major/moderator/minor flares, respectively. These threshold values (SLEDAI,BILAG) were all >82% sensitive and specific for capturing flare severity. Provisional criteria for global flares in cSLEare available to identify patients who experienced a flare. These criteria also allow for discrimination of the severity of cSLEexacerbations.

To describe the character and composition of the 2015 USadult rheumatology workforce, evaluate workforce trends, and project supply and demand for clinical rheumatology care for 2015–2030. The 2015 Workforce Study of Rheumatology Specialists in the USused primary and secondary data sources to estimate the baseline adult rheumatology workforce and determine demographic and geographic factors relevant to workforce modeling. Supply and demand was projected through 2030, utilizing data‐driven estimations regarding the proportion and clinical full‐time equivalent (FTE) of academic versus nonacademic practitioners. The 2015 adult workforce (physicians, nurse practitioners, and physician assistants) was estimated to be 6,013 providers (5,415 clinical FTE). At baseline, the estimated demand exceeded the supply of clinical FTEby 700 (12.9%). By 2030, the supply of rheumatology clinical providers is projected to fall to 4,882 providers, or 4,051 clinical FTE(a 25.2% decrease in supply from 2015 baseline levels). Demand in 2030 is projected to exceed supply by 4,133 clinical FTE(102%). The adult rheumatology workforce projections reflect a major demographic and geographic shift that will significantly impact the supply of the future workforce by 2030. These shifts include baby‐boomer retirements, a millennial predominance, and an increase of female and part‐time providers, in parallel with an increased demand for adult rheumatology care due to the growing and aging USpopulation. Regional and innovative strategies will be necessary to manage access to care and reduce barriers to care for rheumatology patients.

Noninvasive estimation of the degree of inflammation seen on kidney biopsy with lupus nephritis (LN) remains difficult. The objective of this study was to develop a Renal Activity Index for Lupus (RAIL) that, based solely on laboratory measures, accurately reflects histologic LN activity. We assayed traditional LN laboratory tests and 16 urine biomarkers (UBMs) in children (n = 47) at the time of kidney biopsy. Histologic LN activity was measured by the National Institutes of Health activity index (NIH‐AI) and the tubulointerstitial activity index (TIAI). High LN‐activity status (versus moderate/low) was defined as NIH‐AI scores >10 (versus ≤10) or TIAI scores >5 (versus ≤5). RAIL algorithms that predicted LN‐activity status for both NIH‐AI and TIAI were derived by stepwise multivariate logistic regression, considering traditional biomarkers and UBMs as candidate components. The accuracy of the RAIL for discriminating by LN‐activity status was determined. The differential excretion of 6 UBMs (neutrophil gelatinase–associated lipocalin, monocyte chemotactic protein 1, ceruloplasmin, adiponectin, hemopexin, and kidney injury molecule 1) standardized by urine creatinine was considered in the RAIL. These UBMs predicted LN‐activity (NIH‐AI) status with >92% accuracy and LN‐activity (TIAI) status with >80% accuracy. RAIL accuracy was minimally influenced by concomitant LN damage. Accuracies between 71% and 85% were achieved without standardization of the UBMs. The strength of these UBMs to reflect LN‐activity status was confirmed by principal component and linear discriminant analyses. The RAIL is a robust and highly accurate noninvasive measure of LN activity. The measurement properties of the RAIL, which reflect the degree of inflammatory changes as seen on kidney biopsy, will require independent validation.

The small size of many pediatric rheumatology programs translates into limited mentoring options for early career physicians. To address this problem, the American College of Rheumatology (ACR) and the Childhood Arthritis and Rheumatology Research Alliance (CARRA) developed a subspecialty‐wide interinstitutional mentoring program, the ACR/CARRA Mentoring Interest Group (AMIGO). We sought to assess the impact of this program on mentoring within pediatric rheumatology. In a longitudinal 3‐year study, participant ratings from the AMIGO pilot program were compared with those after the program was opened to general enrollment. Access to mentoring as a function of career stage was assessed by surveys of the US and Canadian pediatric rheumatologists in 2011 and 2014, before and after implementation of AMIGO. Participants in the pilot phase (19 dyads) and the general implementation phase (112 dyads) reported comparable success in establishing mentor contact, suitability of mentor‐mentee pairing, and benefit with respect to career development, scholarship, and work‐life balance. Community surveys showed that AMIGO participation as mentee was high among fellows (86%) and modest among junior faculty (31%). Implementation correlated with significant gains in breadth of mentorship and in overall satisfaction with mentoring for fellows but not junior faculty. AMIGO is a career mentoring program that serves most fellows and many junior faculty in pediatric rheumatology across the US and Canada. Program evaluation data confirm that a subspecialty‐wide interinstitutional mentoring program is feasible and can translate into concrete improvement in mentoring, measurable at the level of the whole professional community.

To assess the quality of medical care in childhood‐onset systemic lupus erythematosus (SLE) at tertiary pediatric rheumatology centers as measured by observance of SLE quality indicators (SLE‐QIs). International consensus has been achieved for childhood‐onset SLE‐QIs capturing medical care provision in 9 domains: diagnostic testing, education of cardiovascular (CV) risk and lifestyles, lupus nephritis (LN), medication management, bone health, ophthalmologic surveillance, transition, pregnancy, and vaccination. Using medical record information, the level of performance of these childhood‐onset SLE‐QIs was assessed in childhood‐onset SLE populations treated at 4 tertiary pediatric rheumatology centers in the US, 2 in Brazil, and 1 center in India. A total of 483 childhood‐onset SLE patients were assessed. Care for the 310 US patients differed markedly for childhood‐onset SLE‐QIs addressing LN, bone health, vaccinations, education on CV risk, and transition planning. Performance of safety blood testing for medications was high at all centers. Despite often similar performance on the childhood‐onset SLE‐QI, access to kidney biopsies was lower in Brazil than in the US. Irrespective of the country of practice, larger centers tended to meet the childhood‐onset SLE‐QIs more often than smaller centers. The childhood‐onset SLE‐QIs, evidence‐based minimum standards of medical care, are not consistently met in the US or some other countries outside the US. This has the potential to contribute to suboptimal childhood‐onset SLE outcomes.

To validate clinical indices of lupus nephritis activity and damage when used in children against the criterion standard of kidney biopsy findings. In 83 children requiring kidney biopsy, the Systemic Lupus Erythematosus Disease Activity Index renal domain (SLEDAI‐R), British Isles Lupus Assessment Group index renal domain (BILAG‐R), Systemic Lupus International Collaborating Clinics (SLICC) renal activity score (SLICC‐RAS), and SLICC Damage Index renal domain (SDI‐R) were measured. Fixed effects and logistic models were calculated to predict International Society of Nephrology/Renal Pathology Society (ISN/RPS) class; low‐to‐moderate versus high lupus nephritis activity (National Institutes of Health [NIH] activity index [AI]) score: ≤10 versus >10; tubulointerstitial activity index (TIAI) score: ≤5 versus >5; or the absence versus presence of lupus nephritis chronicity (NIH chronicity index) score: 0 versus ≥1. There were 10, 50, and 23 patients with ISN/RPS class I/II, III/IV, and V, respectively. Scores of the clinical indices did not differentiate among patients by ISN/RPS class. The SLEDAI‐R and SLICC‐RAS but not the BILAG‐R differed with lupus nephritis activity status defined by NIH‐AI scores, while only the SLEDAI‐R scores differed between lupus nephritis activity status based on TIAI scores. The sensitivity and specificity of the SDI‐R to capture lupus nephritis chronicity was 23.5% and 91.7%, respectively. Despite being designed to measure lupus nephritis activity, SLICC‐RAS and SLEDAI‐R scores significantly differed with lupus nephritis chronicity status. Current clinical indices of lupus nephritis fail to discriminate ISN/RPS class in children. Despite its shortcomings, the SLEDAI‐R appears best for measuring lupus nephritis activity in a clinical setting. The SDI‐R is a poor correlate of lupus nephritis chronicity.

To develop and initially validate a global cognitive performance score (CPS) for the Pediatric Automated Neuropsychological Assessment Metrics (PedANAM) to serve as a screening tool of cognition in childhood lupus. Patients (n = 166) completed the 9 subtests of the PedANAM battery, each of which provides 3 principal performance parameters (accuracy, mean reaction time for correct responses, and throughput). Cognitive ability was measured by formal neurocognitive testing or estimated by the Pediatric Perceived Cognitive Function Questionnaire‐43 to determine the presence or absence of neurocognitive dysfunction (NCD). A subset of the data was used to develop 4 candidate PedANAM‐CPS indices with supervised or unsupervised statistical approaches: PedANAM‐CPSUWA, i.e., unweighted averages of the accuracy scores of all PedANAM subtests; PedANAM‐CPSPCA, i.e., accuracy scores of all PedANAM subtests weighted through principal components analysis; PedANAM‐CPSlogit, i.e., algorithm derived from logistic models to estimate NCD status based on the accuracy scores of all of the PedANAM subtests; and PedANAM‐CPSmultiscore, i.e., algorithm derived from logistic models to estimate NCD status based on select PedANAM performance parameters. PedANAM‐CPS candidates were validated using the remaining data. PedANAM‐CPS indices were moderately correlated with each other (|r| > 0.65). All of the PedANAM‐CPS indices discriminated children by NCD status across data sets (P< 0.036). The PedANAM‐CPSmultiscorehad the highest area under the receiver operating characteristic curve (AUC) across all data sets for identifying NCD status (AUC >0.74), followed by the PedANAM‐CPSlogit, the PedANAM‐CPSPCA, and the PedANAM‐CPSUWA, respectively. Based on preliminary validation and considering ease of use, the PedANAM‐CPSmultiscoreand the PedANAM‐CPSPCAappear to be best suited as global measures of PedANAM performance.

To determine the direct cost of care of children with childhood‐onset systemic lupus erythematosus (cSLE), and to determine the direct cost per quality‐adjusted life year (QALY) with cSLE.Administrative databases from 2 large tertiary pediatric rheumatology centers in the United States were reviewed for all patients with cSLE (n = 119) diagnosed and regularly treated in these centers between January 2001 and April 2004. Health‐related quality of life estimates for patients with cSLE (n = 297) reported in the literature were used to calculate QALYs based on global health ratings of the Child Health Questionnaire (range 0–100).Information on 3,184 patient‐months of followup was included in the analysis. During a mean ± SD followup of 27 ± 11.8 months, the direct cost of care for the cohort amounted to $3,965,048, excluding outpatient medications. Irrespective of patient sex, the mean ± SD cost of cSLE per month was $1,245 ± $2,352, or ∼$14,944 per year. Inpatient and day hospital care accounted for 28% of the cost, laboratory testing accounted for 21%, inpatient/day‐patient medication costs accounted for 13%, and dialysis accounted for 11%. Visits to the rheumatology clinic only contributed 9% to the direct cost of care. When including an estimated outpatient medication cost of $1,190, the direct cost of cSLE per QALY was $30,908.Children diagnosed with cSLE were found to have a considerable direct cost of care. The treatment of cSLE appears to be far more costly than that of adult SLE and juvenile idiopathic arthritis reported in the literature.

To examine the strength of the association between different measures of health‐related quality of life (HRQOL), disability, pain, and well‐being in children with chronic arthritis. To evaluate whether HRQOL scores vary as a function of disability status beyond chance. To assess the quality of the parent proxy report for HRQOL as compared with disability, pain, and well‐being.Measures of HRQOL (visual analog scale [VAS] of health, Pediatric Quality of Life Inventory [PedsQL], Juvenile Arthritis Quality of Life Questionnaire (JAQQ), and modified standard gamble technique [SG]), disability (Childhood Health Assessment Questionnaire), VAS of pain, and VAS of well‐being (VAS‐well) were completed by the parents (n = 119) and patients ≥8 years (SG: ≥12 years).HRQOL was highest when measured by the SG, whose utilities were no more than weakly correlated with any of the other outcomes. The values of all other HRQOL measures were at least moderately correlated with each other and with the VAS‐well. Irrespective of the measure used, disability was associated with significantly decreased HRQOL. There was fair to good agreement and moderate consistency of the HRQOL ratings (SG: fair consistency) between patients and parents with marked differences between health domains.HRQOL measured by the PedsQL, JAQQ, and VAS are moderately to highly correlated with each other in children with chronic arthritis. The children's HRQOL significantly decreases with increasing disability. Despite more pronounced differences for some health domains, parents are moderate to good proxy reporters of HRQOL, disability, and well‐being of children with chronic arthritis.

To assess the reliability and validity of the Disease Activity Score (DAS), an instrument used to evaluate children with juvenile dermatomyositis (JDM). Psychometric study of internal consistency, reliability, rater agreement, and the relationship with measures of muscle strength and disability was conducted. The DAS ratings are internally consistent (reliability = 0.89) and describe a wide range of disease activity. The pediatric rheumatologists in this study agree on the presence of most of the disease indicators. Their disagreements tend to cancel each other, resulting in highly correlated (r = 0.79) overall measures across raters. Estimates of muscle weakness using the DAS and ratings of muscle strength obtained independently from therapists are highly related (r = −0.77), but estimates of disease activity and disability are weakly related (r = 0.20). The DAS exhibits evidence of good reliability and validity. The combination of skin and muscle strength assessments makes this easily administered instrument a useful addition in the evaluation of children with JDM.

To perform a cost-identification and cost-effectiveness analysis comparing oral corticosteroids (OCS) with high-dose intermittent intravenous corticosteroid (IVCS) regimens in the treatment of juvenile dermatomyositis (JDM). Children previously diagnosed and treated for JDM (without myositis-specific or myositis-associated autoantibodies) at a single medical center by a single provider were identified. Two treatment protocols were compared: OCS and IVCS. Data on initial disease severity, time to remission, resource use, and costs generated were collected from patient records. Incremental cost-effectiveness ratios (ICE) were constructed. Patients treated with IVCS achieved median remission 2 years earlier at median increased cost of $13,736. The ICE ratio comparing IVCS to OCS is $6,868 per year of disease avoided. This study suggests that, although IVCS treatments are costly, they are cost-effective.