Advancing knowledge and clinical development in frontotemporal dementia (FTD) using the American Academy of Neurology’s (AAN) Axon Registry®
Delaney Oliver1, Esther Kane2, Shana Dodge2, Roisheen Doherty1, Gabe Goldfeder 1, Oana Popescu 1, Sonya Li1, Aracelis Torres1, Heather Moss3
1Verana Health, 2The Association for Frontotemporal Degeneration, 3Stanford University

Evaluate feasibility of a registry-based approach to real-world research and trial recruitment for FTD.

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.
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.
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.
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.