Advancing knowledge and clinical development in frontotemporal dementia (FTD) using the American Academy of Neurology’s (AAN) Axon Registry® (S15.007)

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Objective: Evaluate feasibility of a registry-based approach to real-world research and trial recruitment for FTD. Background: FTD is a rare, disabling disease with no cure. Registries play an important role in understanding rare diseases and also have application to identifying clinical trial candidates. The Axon Registry includes electronic health record data (EHR) of more than 3.1 million patients from over 150 U.S. neurology practices. We assessed the utility of the Axon Registry to support FTD trial recruitment by characterizing FTD patients identified. Design/Methods: We performed a retrospective cohort analysis of patients between 25 and 85 years old with FTD (≥1 FTD ICD code and no other neurodegenerative disease ICD codes) between 09/16/2017 to 09/16/2021. Patients with <12 months of follow-up were excluded. Structured data was used to assess demographics, outpatient follow-up, and comorbidities. Results: Of 3,031 patients with ≥1 FTD ICD code, 698 met the eligibility criteria. Of excluded patients, 46.6% had other neurodegenerative disease ICD codes at some point. The eligible cohort had age of 60.9±9.2 (mean±standard deviation) years and was 50.0% female. Among patients with known race and ethnicity, 91.7% were White, 5.4% were Black, and 6.2% were Hispanic. A third (33.8%) of patients were seen at neurology practices that did not have experience with trials. Patients had on average 4.0 encounters within their first year in the Axon Registry with visit frequency decreasing to 2.5 encounters during the second year. Memory loss (47.7%), aphasia (17.9%), and psychiatric symptoms (anxiety, depression) (24.9%) were the most common concomitant diagnoses. Conclusions: EHR data in the Axon Registry can be used to surface patients with FTD. While minority patients are likely underrepresented, we identified 698 potential FTD clinical trial subjects, including 236 who were seen at non-trial experienced practices. Pairing the Axon Registry with provider outreach and education might expand access to clinical trials. Disclosure: Mrs. Oliver has received personal compensation for serving as an employee of Verana Health. Mrs. Oliver has stock in Verana Health. Mrs. Kane has received personal compensation for serving as an employee of The Association for Frontotemporal Degeneration. Dr. Dodge has received personal compensation for serving as an employee of The Association for Frontotemporal Degeneration. Mr. Doherty has received personal compensation for serving as an employee of Verana Health. An immediate family member of Mr. Doherty has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Delfi Diagnostics. Mr. Goldfeder has received personal compensation for serving as an employee of Verana Health. Mrs. Popescu has nothing to disclose. Ms. Li has received personal compensation for serving as an employee of Verana Health. Ms. Li has a non-compensated relationship as a Strategic Partnerships lead with Verana Health that is relevant to AAN interests or activities. Dr. Torres has received personal compensation for serving as an employee of Verana Health. Dr. Moss has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Twenty Twenty Therapeutics. Dr. Moss has received personal compensation in the range of $100,000-$499,999 for serving as a Consultant for Verana Health. Dr. Moss has received personal compensation in the range of $500-$4,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Medlink Inc. Dr. Moss has received personal compensation in the range of $10,000-$49,999 for serving as an Expert Witness for Legal Firms. The institution of Dr. Moss has received research support from NIH. The institution of Dr. Moss has received research support from Department of Defense. The institution of Dr. Moss has received research support from Research to Prevent Blindness. Dr. Moss has received personal compensation in the range of $0-$499 for serving as a grant review panel with NASA. Dr. Moss has received personal compensation in the range of $0-$499 for serving as a grant review panel with National Institutes of Health. Dr. Moss has a non-compensated relationship as a Board of Directors with North American Neuro-ophthalmology Society that is relevant to AAN interests or activities.

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