Person-Centered Care and Trial Design for Progressive Supranuclear Palsy: A Collaborative Project of AFTD and CurePSP
Shana Dodge1, Jessica Shurer2, Farwa Ali3, David Irwin4, Sarah Kremen5, Chi-Ying (Roy) Lin6, Julio Rojas-Martinez7, Lawren VandeVrede7, Catherine Wang7, Anne Marie Wills8, Kristophe Diaz2, Penny Dacks1
1Association for Frontotemporal Degeneration, 2CurePSP, 3Mayo Clinic, 4University of Pennsylvania, 5Cedars-Sinai Medical Center, Department of Neurology, 6Baylor College of Medicine, 7UCSF, 8MGH
Objective:
To investigate how the lived experiences of people with progressive supranuclear palsy (PSP) can inform strategies for early and accurate diagnosis, effective interventions, and person-centered clinical trial designs.
Background:
PSP is a relatively rare adult-onset, progressive and irreversible neurodegenerative disease that impacts movement, cognition, behavior, language, and vision. Clinicians and researchers must identify potential participants for available clinical trials early in the disease and ensure experimental drugs or interventions address clinically meaningful domains.
Design/Methods:
The anonymized online FTD Insights Survey queried aspects of the lived experiences of PSP and other types of frontotemporal degeneration. Respondents included 27 persons with PSP (PwPSP) and 76 PSP care partners (CPs) (not matched dyads).
Results:
Respondents experienced a lag between age when symptoms first emerged (ages 50-69) and age at PSP diagnosis (between ages 60-79). More than half of PwPSP (56%) and CPs (62%) reported initial misdiagnoses and seeing more than 3 doctors to obtain an accurate PSP diagnosis.
Respondents cited language and motor dysfunction as being the most distressing symptoms. CPs also reported distress associated with changes in their loved one’s personality (e.g., acting inappropriately) and cognition (e.g., poor judgment).
Respondents noted a significant symptom impact on interpersonal relationships, activities of daily living within the home, and independence in the community. The majority of PwPSP (81%) and CPs (69%) expressed willingness to participate in a clinical trial for PSP and outlined what they would want most out of a PSP treatment.
Conclusions:
The FTD Insights Survey revealed a complicated diagnostic journey for individuals and families impacted by PSP, including complex symptoms and impairment in multiple cognitive domains and functionality. High-priority items for care and research include a decrease in time to diagnosis and better understanding of the effect of PSP symptoms and experiences for optimal clinical trial participation and design.
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