A Learning Health Systems Approach to Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis in Children

Anti-neutrophil cytoplasmic antibody-associated vasculitis (AAV) is an understudied, chronic inflammatory disease in children for which only half of children reach remission by 6-months and approximately 7% do not survive past young adulthood. Due to its rarity, there are no large observational studies or randomized control trials to guide management. Additionally, there is a known discordance between patient and physician global assessments among patients with rheumatic disease, with physician scores most influenced by objective measures (e.g., laboratory or imaging results) and patient scores most influenced by perceived quality of life. The optimal way to incorporate both patient and provider values into a standard of care for children with AAV is unknown, yet critical to improving outcomes in this patient population. A learning health systems (LHS) model is the ideal mechanism to transform the care of children with AAV from non-evidence based intuitive care to personalized care that is informed by both patients and providers. The overall objective of this study is to build the foundation for a LHS for children with AAV through both clinical outcome validation and patient engagement.