Validation of a Novel Ultrasound Score to Improve the Assessment of Joint Inflammation in Children with Juvenile Arthritis - PROJECT SUMMARY/ABSTRACT
Candidate: Patricia Vega-Fernandez, MD MSc RhMSUS is a pediatric rheumatologist whose overarching career
goal is to improve the outcomes of children affected by autoimmune rheumatologic diseases through a career
as an independent clinician scientist focused on biology-based imaging and clinical research to support the
development of personalized treatment approaches. Dr. Vega-Fernandez, an Assistant Professor of Pediatrics
at Cincinnati Children’s Hospital Medical Center (CCHMC), has conducted multiple studies focused on
addressing the feasibility and reliability of musculoskeletal ultrasound (MSUS) in Juvenile Idiopathic Arthritis
(JIA). The proposed K23 career development plan will focus on identified gaps in Dr. Vega-Fernandez’s
knowledge and training that are essential to advance her career towards an independent investigator: 1) Critical
appraisal of the biological signatures in JIA; 2) Performance of US-guided synovial biopsy in children with JIA;
3) Validation of instruments and longitudinal studies; 4) Strengthened personal, academic, and professional skills
to become an effective independently funded clinical-scientist.
Mentors/Environment: Dr. Vega-Fernandez and her mentors, Hermine Brunner MD MSc MBA, Sherry
Thornton PhD, Jonathan Dillman MD MSc, and Jareen Meinzen-Derr PhD MPH, have assembled a strong team
of advisors and collaborators from inside and outside of CCHMC to guide the proposed training and research
activities. The career development plan utilizes intellectual resources at CCHMC and the University of Cincinnati.
Research: JIA, the most common chronic rheumatic disease in childhood, is characterized by joint inflammation
(arthritis). Diagnosis of arthritis in children is based on the presence of joint swelling and limited range of motion
and/or tenderness on palpation. Given the young age of children at diagnosis, the report of joint pain or limitation
of motion can often times be inaccurate. In addition, clinical assessment of active arthritis in the developing
skeleton is subjective and has low inter-rater reliability. MSUS can objectively inform presence and severity of
joint inflammation. There is a knowledge gap on the clinical significance of MSUS findings in children. The central
hypothesis of this study is that a 10-joint focused MSUS score can serve as a diagnostic imaging tool to provide
an accurate assessment of both clinical and biologic activity of inflammation over time in children with JIA. Guided
by strong preliminary data, this hypothesis will be tested with three specific aims: 1) Determine the convergent
validity of the MSUS-10 score across the spectrum of JIA disease activity; 2) Determine the ability of the MSUS-
10 score to capture clinically relevant change of JIA; 3) Evaluate the relationship of the MSUS-10 score with
biologic markers of JIA inflammation. The proposed experiments are significant because the MSUS-10
examination and score may support safer and more appropriate choices for the treatment of arthritis, which in
turn will improve the outcomes of children with JIA. This project is innovative as this novel approach of using
MSUS scoring may lead to a paradigm shift in the treatment and care that JIA patients receive.