Comparative Effectiveness Research in Axial Spondyloarthritis

Axial spondyloarthritis (axSpA) comprises a heterogeneous group of infl ammatory spine conditions that affects 0.9 – 1.4 % of the adult population in the United States. The treatment options for symptom control in patients with axSpA have progressed dramatically in the past few decades, with the availability of tumor necrosis factor inhibitors, interleukin 17 inhibitors, and Janus kinase inhibitor, in addition to non-steroidal anti- infl ammatory drugs (NSAIDs). However, limited evidence is available to guide clinicians and patients to make an individualized treatment plan based on current group level effectiveness and safety data of therapeutic agents. Traditional head-to-head randomized clinical trials are limited by small number of study arms, high cost, and inability to measure individual treatment effects. N-of1 trials use multi-crossover to address patient-treatment interaction and to measure individual responses to different therapies. I have submitted an NIH K23 application with an overall goal to test the feasibility of N-of-1 trials of two NSAIDs in axSpA and to develop informatics tools to facilitate the planning and implementation of such trials in a larger scale. The proposed series of N-of-1 trials will provide a framework for comparative effectiveness of other therapeutic agents in patients with axSpA. Coupled with an EHRbased cohort identifi cation tool, this trial design will increase therapeutic precision in individual patients and promote patientcentered research and personalized medicine in axSpA.