A targeted approach to cytokine blockade in juvenile arthritis
Tiphanie Vogel, MD, PhD
BACKGROUND: Juvenile idiopathic arthritis (JIA) is one of the most common chronic diseases of childhood, affecting 1:1000 children. Left untreated, JIA leads to severe disability and even death.
GAP: There has been an expansion of treatment options for JIA over the last decade due to the development of therapeutics that block immune system cytokine signaling. However, no data exist to direct targeted medication selection among the available options for an individual patient.
HYPOTHESIS: We hypothesize that there are subsets of JIA patients with characteristic and detectable patterns of immune activation and differentiation. The goal of this proposal is to identify patients with unique immune profiles that may respond better to a specific cytokine blockade.
METHODS: Peripheral blood will be collected serially from JIA patients and healthy pediatric controls. Levels of cytokine activation and frequency of T cell subsets will be determined using flow cytometry. Clinical data, including type of and response to medication, will be collected on enrolled patients and compared to their immune profiles.
IMPACT: Individual JIA patients could benefit from a targeted treatment approach based on unique immune profiles allowing for purposeful selection of the best treatment at the time of diagnosis. Further, assessment of specific immune activation status in treatment-naïve patients followed by serial assessment after treatment may allow for identification of factors predicting response to therapy or long-term prognosis in patients with JIA.
Dr. Vogel has clinical interest and expertise in all aspects of pediatric and adult autoimmune and immune dysregulatory conditions. She has a special clinical interest in helping patients with childhood-onset rheumatologic conditions prepare for and make the transition into either her own or another adult rheumatology clinic. Dr. Vogel’s laboratory focuses on several key missions: advancement of the understanding of cytokine signals in health and in autoimmune disease, investigation of unique biomarkers and therapeutic targets in pediatric autoimmunity, and identification of novel genetic causes of autoimmune and immune dysregulatory disorders.