Abstract Details

Title Person-Centered Care and Trial Design for Progressive Supranuclear Palsy: A Collaborative Project of AFTD and CurePSP

Topic Aging, Dementia, and Behavioral Neurology

Presentation(s) P8 - Poster Session 8 (8:00 AM-9:00 AM)

Poster/Presentation
Number 3-020

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.

Authors/Disclosures
Shana Dodge (Association for Frontotemporal Degeneration) PRESENTER	Shana Dodge has received personal compensation for serving as an employee of The Association for Frontotemporal Degeneration (AFTD).
Jessica Shurer, MSW	Ms. Shurer has received personal compensation for serving as an employee of CurePSP.
Farwa Ali, MD (Mayo Clinic)	Dr. Ali has nothing to disclose.
David Irwin, MD (University of Pennsylvania)	The institution of Dr. Irwin has received research support from NIH. The institution of Dr. Irwin has received research support from Prevail. The institution of Dr. Irwin has received research support from Passage Bio. The institution of Dr. Irwin has received research support from Alector. The institution of Dr. Irwin has received research support from Transposon. The institution of Dr. Irwin has received research support from Denali. The institution of Dr. Irwin has received research support from Cervo Med.
Sarah Kremen, MD (Cedars-Sinai Medical Center, Department of Neurology)	Dr. Kremen has received publishing royalties from a publication relating to health care.
Chi-Ying (Roy) Lin, MD, FAAN (Baylor College of Medicine)	Dr. Lin has received research support from Texas Alzheimer's Research and Care Consortium (TARCC). Dr. Lin has received research support from CurePSP. Dr. Lin has received research support from Mike Hogg Fund. Dr. Lin has received research support from The Michael J. Fox Foundation Parkinson's Progression Markers Initiative (PPMI). Dr. Lin has a non-compensated relationship as a Secretary with Broadway for Ataxia Foundation (a 501 (c) (3) non-profit organization) that is relevant to AAN interests or activities.
Julio C. Rojas-Martinez, MD, PhD (UCSF)	Dr. Rojas-Martinez has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Ferrer International, S.A.. The institution of Dr. Rojas-Martinez has received research support from NIH/NIA. The institution of Dr. Rojas-Martinez has received research support from Eli Lilly. The institution of Dr. Rojas-Martinez has received research support from Eisai. The institution of Dr. Rojas-Martinez has received research support from Amylyx.
Lawren VandeVrede, MD, PhD (UCSF)	Dr. VandeVrede has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biogen. Dr. VandeVrede has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Roche. Dr. VandeVrede has received personal compensation in the range of $500-$4,999 for serving as a Consultant for CND Life Sciences. Dr. VandeVrede has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Siemens. Dr. VandeVrede has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Roche. Dr. VandeVrede has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Peerview CME. Dr. VandeVrede has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Haymarket. Dr. VandeVrede has received personal compensation in the range of $500-$4,999 for serving as an Expert Witness for Peter Lacques, LLC. Dr. VandeVrede has received personal compensation in the range of $5,000-$9,999 for serving as an Expert Witness for Cunningham Bounds. The institution of Dr. VandeVrede has received research support from Alzheimer's Association. The institution of Dr. VandeVrede has received research support from NIH.
Cathy Wang, MSW	Ms. Wang has nothing to disclose.
Anne Marie A. Wills, MD (MGH)	Dr. Wills has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Accordant, a CVS/Caremark company. Dr. Wills has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sanofi/Genzyme. The institution of Dr. Wills has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Ono Pharmaceuticals. The institution of Dr. Wills has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Amylyx. Dr. Wills has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Parkinson's Foundation. The institution of Dr. Wills has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Amylyx Pharmaceuticals. The institution of Dr. Wills has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Ono Pharmaceuticals. The institution of Dr. Wills has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Roche/Genentech. The institution of Dr. Wills has received research support from Parkinson's Foundation. The institution of Dr. Wills has received research support from Biogen. The institution of Dr. Wills has received research support from Roche/Genentech. The institution of Dr. Wills has received research support from BioSensics.
Kristophe Diaz, PhD	Dr. Diaz has nothing to disclose.
Penny Dacks, PhD (AFTD)	Penny Dacks has received personal compensation for serving as an employee of The Association for Frontotemporal Degeneration.

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