The candidate is a junior investigator in pediatric rheumatology with advance training in clinical epidemiology and a research focus on pharmacoepidemiology and comparative effectiveness of chronic rheumatic disease, including enthesitis-related arthritis (ERA), which is a category of juvenile idiopathic arthritis (JIA). The candidate proposes a comprehensive interdisciplinary program that will provide her with the skills and experience for her development into an independent clinical investigator in pediatric rheumatology. Her career development will be guided by a comprehensive group of co-investigators with a long history of productive collaboration. The project will build upon the foundations of pharmacoepidemiology and clinical epidemiology research knowledge that she obtained during her MSCE training and thesis work and will prepare her to conduct intervention studies in children with all forms of JIA. JIA is the most common rheumatologic condition in childhood, affecting 1 to 4 per 1,000 children. ERA is a JIA category that accounts for 10-20% of JIA. Physicians treat ERA to control inflammation and to prevent morbidities such as chronic uveitis, growth disturbance, contracture, joint destruction, and functional limitation. A subset of children with ERA will progress to ankylosing spondylitis (AS), a condition characterized by spinal and back pain, stiffness, and eventual fusion of the vertebra. Observational studies suggest that less than 20% of children with ERA achieve remission within five years after diagnosis. Significant challenges to providing and studying ERA treatment today are limited by methods for assessing enthesitis, disease activity, and response to therapy. The proposed plan of research addresses these challenges and will (1) evaluate the accuracy of physical examination to detect enthesitis, (2) develop and validate a composite ERA disease activity index, and (3) compare the effectiveness and safety of alternative treatment strategies in incident cases of ERA. This research will provide the preliminary data, hypotheses, and outcome measures for future safety, efficacy, and cost- effectiveness studies of ERA treatment algorithms.

Public Health Relevance

New medications, including biologic agents, have revolutionized the treatment of enthesitis-related arthritis (ERA), a category of Juvenile Idiopathic Arthritis (JIA), and improved long-term expectations. Knowledge of the safety and effectiveness of these drugs is particularly important for children, for whom their use is often not licensed, and who may need to use them for much longer than their adult counterparts. This research will provide the preliminary data, hypotheses, and outcome measures for future intervention studies for children with all forms of JIA.

National Institute of Health (NIH)
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
Mentored Patient-Oriented Research Career Development Award (K23)
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Arthritis and Musculoskeletal and Skin Diseases Special Grants Review Committee (AMS)
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Witter, James
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Children's Hospital of Philadelphia
United States
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Weiss, Pamela F; Xiao, Rui; Brandon, Timothy G et al. (2018) Comparative Effectiveness of Tumor Necrosis Factor Agents and Disease-modifying Antirheumatic Therapy in Children with Enthesitis-related Arthritis: The First Year after Diagnosis. J Rheumatol 45:107-114
Gmuca, Sabrina; Yu, JiaDe; Weiss, Pamela F et al. (2018) Erythema Ab Igne in an Adolescent With Chronic Pain: An Alarming Cutaneous Eruption From Heat Exposure. Pediatr Emerg Care :
Gmuca, Sabrina; Xiao, Rui; Brandon, Timothy G et al. (2017) Multicenter inception cohort of enthesitis-related arthritis: variation in disease characteristics and treatment approaches. Arthritis Res Ther 19:84
Brandon, Timothy G; Becker, Brandon D; Bevans, Katherine B et al. (2017) Patient-Reported Outcomes Measurement Information System Tools for Collecting Patient-Reported Outcomes in Children With Juvenile Arthritis. Arthritis Care Res (Hoboken) 69:393-402
James, Karen E; Xiao, Rui; Merkel, Peter A et al. (2017) Variation in the Treatment of Children Hospitalized With Antineutrophil Cytoplasmic Antibody-Associated Vasculitis in the US. Arthritis Care Res (Hoboken) 69:1377-1383
Weiss, Pamela F; Colbert, Robert A (2016) Reply. Arthritis Care Res (Hoboken) 68:1213-4
James, Karen E; Xiao, Rui; Merkel, Peter A et al. (2016) Clinical course and outcomes of childhood-onset granulomatosis with polyangiitis. Clin Exp Rheumatol :
Ogdie, Alexis; Weiss, Pamela (2015) The Epidemiology of Psoriatic Arthritis. Rheum Dis Clin North Am 41:545-68
Gmuca, Sabrina; Weiss, Pamela F (2015) Evaluation and Treatment of Childhood Enthesitis-Related Arthritis. Curr Treatm Opt Rheumatol 1:350-364
Basiaga, Matthew L; Weiss, Pamela F; Behrens, Edward M (2015) BIRC4 Mutation: An Important Rare Cause of Uveitis. J Clin Rheumatol 21:444-7

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