In the first multicenter prospective cohort of patients with juvenile SSc in North America, the disease burden was high: multiorgan manifestations were common, and functional disability was greater than that observed in patients with other childhood-onset rheumatic diseases. Gastrointestinal involvement had the greatest impact on quality of life.

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BackgroundSystemic sclerosis (SSc) is a rare multisystem autoimmune disease characterized by vasculopathy and organ fibrosis. From 2010–2013, the North American CARRA Legacy Registry enrolled and prospectively followed children with rheumatologic conditions including juvenile systemic sclerosis (jSSc).ObjectivesDescribe disease manifestations, physician metrics, and patient quality of life (QOL) at enrollment and follow up of jSSc subjects.MethodsDescriptive statistics were used for demographic and clinical features. We used McNemar's test for paired data and Fisher's exact for group comparisons.ResultsFor the 64 children with SSc in the database, the majority were female (84%) and Caucasian (78%); 86% identified as non-Hispanic. Median age of onset was 10.3 yr while age at first pediatric rheumatology (PRH) evaluation was 11.8 yr, with 23% having a ≥2 yr delay to PRH. Baseline visit occurred a median of 3.6 yrs after disease onset and data obtained 1–2 yr from enrollment was analyzed for follow up (n=25, median 1.4 yrs).For those with complete clinical data, 81% met ACR/EULAR 2013 adult criteria for SSc at baseline. Overlap with juvenile dermatomyositis was reported in 3 individuals, with mixed connective tissue disease in 1. Of those with documented testing for specific antibodies, 80% were ANA positive, 46% anti-Scl70 and 15% ACA. Three of 15 patients tested for anti-PM-Scl antibodies were positive.At baseline, the most prevalent organ manifestations were dermatologic (93%) and vascular (92%), with Raynaud phenomenon as the most common feature. Multiple organs were affected in 93%, and 38% had ≥4 organ systems involved. Disease manifestations remained stable at follow up with the exception of decreased frequency of arthritis (4 to 1) and increased joint contractures (7 to 11). At conclusion of the study, no interval development of renal, cardiac, or pulmonary manifestations or mortalities occurred.Frequency of disability, measured by ACR functional class, decreased from 44% at baseline to 25% at follow up. Median Physician Global Disease Activity scores improved from 3 (IQR 1–4) to 2 (1–3), with trending significance for those with <2 years of disease at enrollment (n=7, p=0.059). While there were no statistically significant differences in QOL measures over time, trends of improvement in pain scale (3.5 to 2) and global well-being scale (5 to 3) were appreciated in this early disease group.ConclusionsEven with inclusion of overlap patients, baseline clinical data from this cohort are similar to the jSSc cohort described by Martini et al in 2006 and most met current ACR/EULAR adult SSc criteria. Most organ manifestations were stable with time and for the duration of the study observation period, there was no interval development of major organ involvement or mortality. While limited by the cross sectional nature of enrollment, trends of improvement in disability and disease activity were appreciated after 1–2 years of follow up.ReferencesMartini G, et al. Systemic Sclerosis in Childhood. Arthritis Rheum 2006;54...

Introduction: The Children's Dermatology Life Quality Index has been used to measure quality of life in studies of pediatric localized scleroderma, which suggested only modest effects on quality of life. However, the Children's Dermatology Life Quality Index psychometric performance has not been examined in localized scleroderma and it was validated in populations lacking localized scleroderma's distinctive clinical features, possibly underestimating the quality of life impact. This study assessed psychometric properties of the Children's Dermatology Life Quality Index in a cohort of pediatric localized scleroderma patients. Methods: Existing Children's Dermatology Life Quality Index data from a large pediatric localized scleroderma cohort were analyzed. Children's Dermatology Life Quality Index score distributions were examined and internal consistency was evaluated with Cronbach's alpha for the entire Children's Dermatology Life Quality Index and after deletion of individual items. Construct validity was assessed by calculating Spearman's correlations between Children's Dermatology Life Quality Index scores and disease severity/impact measures. Dimensionality was examined using exploratory factor analysis with sequential item elimination. Results: Children's Dermatology Life Quality Index scores suggested modest adverse effects on quality of life. Internal consistency was adequate (Cronbach's alpha = 0.727) but increased after eliminating items regarding friendships, sleep, and treatment burdens. Children's Dermatology Life Quality Index scores were not associated with physician-scored disease severity measures but were moderately associated with patient/parent assessments of disease impact. Exploratory factor analysis yielded a three-factor solution encompassing functional limitations, psychosocial effects, and skin symptoms/treatment burden. Conclusion: The Children's Dermatology Life Quality Index may capture functional and psychosocial domains of quality of life in localized scleroderma, but likely underestimates the quality of life impact given that it includes some items with limited relevance in localized scleroderma, incompletely explores skin symptoms and treatment burdens, and demonstrates limited construct validity. Further study to optimize quality of life measurement in pediatric localized scleroderma is warranted.

Table of ContentsP1 Serologic evidence of gut-driven systemic inflammation in juvenile idiopathic arthritisLampros Fotis, Nur Shaikh, Kevin Baszis, Anthony French, Phillip TarrP2 Oral health and anti-citrullinated peptide antibodies (ACPA) in juvenile idiopathic arthritisSriharsha Grevich, Peggy Lee, Sarah Ringold, Brian Leroux, Hannah Leahey, Megan Yuasa, Jessica Foster, Jeremy Sokolove, Lauren Lahey, William Robinson, Joshua Newsom, Anne StevensP3 Novel autoantigens for endothelial cell antibodies in pediatric rheumatic diseases identified by proteomicsRie Karasawa, Mayumi Tamaki, Megumi Tanaka, Toshiko Sato, Kazuo Yudoh, James N. JarvisP4 Transcriptional profiling reveals monocyte signature associated with JIA patient poor response to methotrexateHalima Moncrieffe, Mark F. Bennett, Monica Tsoras, Lorie Luyrink, Huan Xu, Sampath Prahalad, Paula Morris, Jason Dare, Peter A. Nigrovic, Margalit Rosenkranz, Mara Becker, Kathleen M. O’Neil, Thomas Griffin, Daniel J. Lovell, Alexei A. Grom, Mario Medvedovic, Susan D. ThompsonP5 A multi-dimensional genomic map for polyarticular juvenile idiopathic arthritisLisha Zhu, Kaiyu Jiang, Laiping Wong, Michael J Buck, Yanmin Chen, Halima Moncrieffe, Laura Brungs, Tao Liu, Ting Wang, James N JarvisP6 Tocilizumab for treatment of children with refractory JIAKhaled Alsaeid, Jasim Alfailakawi, Hamid Alenezi, Hazim AlsaeedP7 Clinical characteristics of the initial patients enrolled in the Childhood Arthritis and Rheumatology Research Alliance (CARRA) RegistryTim Beukelman, Marc Natter, Norm Ilowite, Kelly Mieszkalski, Grendel Burrell, Brian Best, Helen Bristow, Shannon Carr, Anne Dennos, Rachel Kaufmann, Yukiko Kimura, Laura SchanbergP8 Comparative performance of small and large clinical centers in a comprehensive pediatric rheumatology disease registryPeter R BlierP9 Clinical characteristics of children with membranous lupus nephritis: The Childhood Arthritis and Rheumatology Research Alliance Legacy RegistryAlexis Boneparth, Scott E. Wenderfer, L. Nandini Moorthy, Suhas M. Radhakrishna, Anna Carmela P. Sagcal-Gironella, Emily von SchevenP10 Rituximab use in pediatric lupus anticoagulant hypoprothrombinemia syndrome - a two center experienceKader Cetin Gedik, Salma Siddique, Cassyanne L. Aguiar, Doruk ErkanP11 Predictors of complementary and alternative medicine use and response in children with musculoskeletal conditionsEzra Cohen, Yvonne Lee, Michelle Dossett, Darshan Mehta, Roger DavisP12 Comparison of pediatric rheumatology and nephrology survey results for the treatment of refractory proliferative lupus nephritis and renal flare in juvenile SLEMileka Gilbert, Beatrice Goilav, Esra Meidan, Joyce Hsu, Alexis Boneparth, Anabelle Chua, Stacy Ardoin, Scott E. Wenderfer, Emily Von Scheven, Natasha M. RuthP13 Transitioning lupus patients from pediatric to adult rheumatologyJoyce Hui-Yuen, Kader Cetin Gedik, Liza Bermudez, Ashlea Cook, Lisa Imundo, Amy Starr, Andrew Eichenfield, Anca AskanaseP14 The systemic juvenile idiopathic arthritis cohort of the Childhood Arthritis & Rheumatolo...