Abstract Details

Title Psychometric Validation of Huntington’s Disease Everyday Functioning (Hi-DEF) Scale –Study Design and Sample Characteristics

Topic Movement Disorders

Presentation(s) P3 - Poster Session 3 (5:30 PM-6:30 PM)

Poster/Presentation
Number 5-005

Background

Cognitive impairment is a key component of early Huntington’s Disease (HD), particularly in the executive functioning domain, which encompasses multi-tasking, organizing thoughts, planning, and decision making. The Huntington’s Disease Everyday Functioning (Hi-DEF) Scale was developed to capture the impact of executive function changes on tasks of everyday living.

Objective To describe the Hi-DEF Scale validation study and characteristics of the study sample.

Design/Methods A non-interventional, cross-sectional validation study was conducted at 9 HD centers of excellence across the US. HTT gene-positive (CAG repeats ≥36) HD patients, 25-65 years old, with TFC 8-13 were included. Centers enrolled eligible patients and supported completion of scales via an online research platform (CANTAB Connect). Patients self-completed the Hi-DEF scale, a computer-based battery of cognitive assessments (Emotional Recognition Task, One Touch Stocking of Cambridge, Paired Associated Learning, Spatial Working Memory, and Spatial Span), HD PRO Triad (PRO assessing cognitive, emotional/behavioral, and motor symptoms in HD) and demographic questionnaire. Clinical history, including HD genetic testing and other relevant medical problems, was collected. The analysis plan includes construct and known groups validity evaluation of the Hi-DEF scale, as well as scoring interpretation by anchoring cognitive assessment test scores on the Hi-DEF scale. The study was approved by a central IRB.

Results A total of 67 responses were collected to date. Patients were on average 48.92 (±SD 11.92) years old, 52% female, 90% non-Hispanic white, 7.32 (±SD 6.91) years since genetic testing, TFC scores 8-13, and HTT CAG repeats ranging from 39-52. Psychometric validation of the Hi-DEF Scale is currently underway. Scoring, study findings and instrument will be disseminated for use in broader HD community and research.

Conclusions Validation of the Hi-DEF Scale has the potential to facilitate assessment of functional changes associated with cognitive sequelae of HD, with an emphasis on higher-order executive abilities, integral to working, driving, and social function.

Authors/Disclosures
PRESENTER	No disclosure on file
	No disclosure on file
	No disclosure on file
Ruta Sawant, PhD	Ms. Sawant has received personal compensation for serving as an employee of Sage Therapeutics. Ms. Sawant has received stock or an ownership interest from Sage Therapeutics.
	No disclosure on file
	No disclosure on file
	No disclosure on file
	No disclosure on file
Jee Bang, MD (Johns Hopkins University)	Dr. Bang has received personal compensation in the range of $0-$499 for serving as a Consultant for GLG. Dr. Bang has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Adept Field Solutions.
Danny Bega, MD (Northwestern University)	Dr. Bega has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Teva Pharmaceuticals. Dr. Bega has received personal compensation in the range of $500-$4,999 for serving as a Consultant for GE Healthcare. Dr. Bega has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Acadia Pharmaceuticals. Dr. Bega has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alexion Pharmaceuticals. Dr. Bega has received personal compensation in the range of $500-$4,999 for serving as a Consultant for WebMD. Dr. Bega has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Teva Pharmaceuticals. Dr. Bega has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Acorda Therapeutics. Dr. Bega has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Neurocrine Biosciences. Dr. Bega has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Kyowa Kirin. Dr. Bega has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Supernus Pharmaceuticals. Dr. Bega has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Abbvie. Dr. Bega has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Sunovion. Dr. Bega has received personal compensation in the range of $500-$4,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for ACTN / ANA. The institution of Dr. Bega has received research support from Huntington Disease Society of America. The institution of Dr. Bega has received research support from Parkinson Foundation.
	No disclosure on file
Rajeev Kumar, MD, FRCPC (Rocky Mountain Movement Disorders Center)	The institution of Dr. Kumar has received personal compensation in the range of $50,000-$99,999 for serving as a Consultant for Supernus. The institution of Dr. Kumar has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Abbvie. The institution of Dr. Kumar has received personal compensation in the range of $100,000-$499,999 for serving as a Consultant for Teva Pharmaceuticals. The institution of Dr. Kumar has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Annexon. The institution of Dr. Kumar has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Roche. The institution of Dr. Kumar has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Acorda Therapeutics. The institution of Dr. Kumar has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Cerevel Therapeutics. Dr. Kumar has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Impel Pharma. Dr. Kumar has received personal compensation in the range of $50,000-$99,999 for serving on a Speakers Bureau for Supernus. The institution of Dr. Kumar has received personal compensation in the range of $100,000-$499,999 for serving on a Speakers Bureau for Teva. The institution of Dr. Kumar has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Acorda Therapeutics. The institution of Dr. Kumar has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Kyowa Kirin. Dr. Kumar has received personal compensation in the range of $1,000,000+ for serving as an officer or member of the Board of Directors for Research Catalyst, LLC. Dr. Kumar has received personal compensation in the range of $100,000-$499,999 for serving as an officer or member of the Board of Directors for CenExel RMCR. Dr. Kumar has stock in Research Catalyst, LLC. Dr. Kumar has stock in CenExel. The institution of Dr. Kumar has received research support from Supernus. The institution of Dr. Kumar has received research support from Sage Therapeutics. The institution of Dr. Kumar has received research support from Prilenia Therapeutics. The institution of Dr. Kumar has received research support from Roche. The institution of Dr. Kumar has received research support from Triplet Therapeutics. The institution of Dr. Kumar has received research support from CHDI Foundation. The institution of Dr. Kumar has received research support from Neurocrine Biosciences. The institution of Dr. Kumar has received research support from Biovie. The institution of Dr. Kumar has received research support from Neuroderm. The institution of Dr. Kumar has received research support from Sanofi. The institution of Dr. Kumar has received research support from Addex Pharma. The institution of Dr. Kumar has received research support from Integrative Research Laboratories. The institution of Dr. Kumar has received research support from Takeda Pharmaceuticals. The institution of Dr. Kumar has received research support from Neuraly. The institution of Dr. Kumar has received research support from Abbvie. The institution of Dr. Kumar has received research support from Cerevel Therapeutics. The institution of Dr. Kumar has received research support from Transposon Therapeutics. The institution of Dr. Kumar has received research support from Lundbeck. The institution of Dr. Kumar has received research support from Biohaven. The institution of Dr. Kumar has received research support from Revance Therapeutics. The institution of Dr. Kumar has received research support from Impax Laboratories. The institution of Dr. Kumar has received research support from Pharma Two B. The institution of Dr. Kumar has received research support from Enterin. The institution of Dr. Kumar has received research support from CND Life Sciences. The institution of Dr. Kumar has received research support from Neuron23. The institution of Dr. Kumar has received research support from Annexon Biosciences. The institution of Dr. Kumar has received research support from Annovis. The institution of Dr. Kumar has received research support from PTC Therapeutics. The institution of Dr. Kumar has received research support from Uniqure. The institution of Dr. Kumar has received research support from Alexza. The institution of Dr. Kumar has received research support from SparkNeuro. The institution of Dr. Kumar has received research support from Praxis. The institution of Dr. Kumar has received research support from Scion Neurostim. The institution of Dr. Kumar has received research support from Cognition Therapeutics. The institution of Dr. Kumar has received research support from Eli Lilly. Dr. Kumar has received personal compensation in the range of $100,000-$499,999 for serving as a Medical Director with CenExel RMCR. Dr. Kumar has received personal compensation in the range of $1,000,000+ for serving as a Managing Member with Research Catalyst, LLC.
Henry P. Moore, MD (University of Miami - Miller School of Medicine)	Dr. Moore has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Abbvie. Dr. Moore has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Ipsen Pharma. The institution of Dr. Moore has received research support from Sage Therapeutics. The institution of Dr. Moore has received research support from Bukwang Pharmaceutical. The institution of Dr. Moore has received research support from Neurocrine. The institution of Dr. Moore has received research support from CDHI Foundation. The institution of Dr. Moore has received research support from MODUS Outcomes LLC.
Susan L. Perlman, MD (UCLA)	Dr. Perlman has nothing to disclose.
Luis A. Sierra, Jr. (Beth Israel Deaconess Medical Center)	Mr. Sierra has nothing to disclose.
Stefan Cano, MD (Modus Outcomes)	Stefan Cano, MD has received personal compensation in the range of $100,000-$499,999 for serving as an officer or member of the Board of Directors for Modus Outcomes. Stefan Cano, MD has received stock or an ownership interest from Modus Outcomes. Stefan Cano, MD has received intellectual property interests from a discovery or technology relating to health care.
Aaron Koenig	Aaron Koenig has received personal compensation for serving as an employee of Sage Therapeutics.