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ObjectiveProspective comparative effectiveness research (CER) in chronic nonbacterial osteomyelitis (CNO) is lacking. Our objectives were to (1) determine the use and safety of each consensus treatment plan (CTP) regimen for CNO, (2) assess the feasibility of using the Chronic Nonbacterial Osteomyelitis International Registry (CHOIR) data for CER, and (3) develop and validate a CNO clinical disease activity score (CDAS) using CHOIR.MethodsConsenting children or young adults with CNO were enrolled into CHOIR. Demographic, clinical, and imaging data were prospectively collected. The CNO CDAS was developed through a Delphi survey and nominal group technique. External validation surveys were administered to CHOIR participants.ResultsOne hundred forty (78.2%) CHOIR participants enrolled between August 2018 and September 2020 received at least 1 CTP regimen. Baseline characteristics from different CTP groups were well matched. Patient pain, patient global assessment, and clinical CNO lesion count were key variables included in the CNO CDAS. The CDAS showed a strong correlation with patient/parent report of difficulty using a limb, back, or jaw and patient/parent report of disease severity, but a weak correlation with patient/parent report of fatigue, sadness, and worry. The change in CDAS was significant in patients reporting disease worsening or improvement (P< 0.001). The CDAS significantly decreased after initiating second-line treatments from median 12.0 (IQR 8.0-15.5) to 5.0 (IQR 3.0-12.0; P= 0.002). Although second-line treatments were well tolerated, psoriasis was the most common adverse event.ConclusionThe CNO CDAS was developed and validated for disease monitoring and assessment of treatment effectiveness. CHOIR provided a comprehensive framework for future CER.

Objective.To measure the associations between self-reported race and ethnicity and disease outcomes, including joint damage, pain, and functional ability, in children with juvenile idiopathic arthritis (JIA).Methods.A cross-sectional analysis of children with JIA enrolled in the Childhood Arthritis and Rheumatology Research Alliance (CARRA) Registry between May 2010 and March 2012. Mann-Whitney U test and chi-square testing were used to compare patient characteristics between race (white, African American, or Asian) and ethnicity (Non-Hispanic and Non-Latino; Hispanic or Latino) categories. Logistic regression was used to measure the associations between each race or ethnicity category and the outcome of interest.Results.Race category was available for 4292 of 4682 children (93% white, 5% African American, Asian 3%). Ethnicity data were available for 4644 (11% Hispanic or Latino). African American children with polyarticular-course JIA had an elevated OR for joint damage on radiographic imaging compared to white children (OR 1.9, 95% CI 1.0–3.1; p = 0.04). Hispanic/Latino children had increased odds of having disability scores > 75th percentile (OR 1.5, 95% CI 1.1–2.1; p < 0.01) compared to non-Hispanic/Latino children; however, these odds were no longer significant when the cohort was limited to children with polyarticular-course JIA. Asian children had decreased odds of higher pain and functional disability compared to white children (p < 0.05).Conclusion.Race and ethnicity were variably associated with joint damage, pain, and functional ability. Understanding outcome variation between different race and ethnicity groups may help to optimize care for children with JIA.

OBJECTIVE: To (1) estimate the health-related quality of life (HRQOL) of children with childhood-onset systemic lupus erythematosus (cSLE) and compare it to that of normative cohorts; (2) assess the relationship of HRQOL with cSLE disease activity and damage; and (3) determine the effects of changes of disease activity on HRQOL. METHODS: Patients with cSLE (n = 98) followed every 3 months completed HRQOL measures, the Pediatric Quality of Life Inventory Generic Core scale (PedsQL-GC), the Rheumatology Module (PedsQL-RM), and the Child Health Questionnaire (CHQ). The British Isles Lupus Activity Group Index (BILAG) was used to measure organ-system-specific disease activity. Physicians rated the course of cSLE between visits. RESULTS: At baseline, mean (standard deviation, SD) score [parent report] of the PedsQL-GC and the PedsQL-RM was 75 (17) and 79 (14), respectively; the mean (SD) of the CHQ physical summary score (CHQ-PHS) was 49 (7) and that of the CHQ psychological summary score was 42 (12). Higher BILAG scores, especially in the general, musculoskeletal, neurological, and vascular, but not the mucocutaneous, renal, cardiovascular, or hematological BILAG domains, were associated with a significantly lower HRQOL. Patients with damage had lower HRQOL than those without damage. All HRQOL measures included were at most modestly responsive to clinically important changes with cSLE. CONCLUSION: HRQOL with cSLE is significantly lower than that reported in healthy populations. Organ-specific involvement with cSLE has a differential effect on HRQOL. Higher disease activity and damage are associated with significantly lower HRQOL as measured by the PedsQL-RM and the CHQ-PHS, and worsening of cSLE leads to a further decline.