Abstract Details

Title Longitudinal Treatment Patterns for Chorea in Patients with Huntington Disease: Data from Enroll-HD

Topic Movement Disorders

Presentation(s) S36 - Movement Disorders: Clinical and Pathologic Characterization of Neurodegenerative Movement Disorders (2:36 PM-2:48 PM)

Poster/Presentation
Number 009

Objective

To determine longitudinal treatment patterns for chorea in individuals with Huntington disease (HD) using Enroll-HD Periodic Dataset 5.0.

Background

Enroll-HD is a worldwide, prospective, observational study of individuals with (or at-risk for) HD. Previous analyses of Enroll-HD data suggest that chorea may be undertreated despite the availability of FDA-approved medications such as the vesicular monoamine transporter 2 [VMAT2] inhibitors and off-label medications such as antipsychotics and benzodiazepines.

Design/Methods Data were collected from Jun-2012 to Oct-2020 for North American patients (≥18 years) with Unified Huntington’s Disease Rating Scale (UHDRS) Diagnostic Confidence Level 4 at each study visit. Presence of chorea was defined as UHDRS Total Maximal Chorea ≥2. Medications intended for chorea (as indicated in the Enroll-HD Database) were categorized as follows: VMAT2 alone, antipsychotics alone, medications other than VMAT2 inhibitors or antipsychotics (Other), and 2+ different medications from previous 3 categories (Combination).

Results Chorea was documented in 96.8% (2507/2590) of eligible patients and 96.5% (6678/6920) of visits. Medications for chorea were prescribed to 36.1% (906/2507) of patients with chorea, with first-line treatments as follows: VMAT2 inhibitors (49.9%), antipsychotics (27.5%), other medications (18.7%), and combination treatments (4.0%). Average treatment duration ranged from 28.8 (VMAT2) to 41.2 (antipsychotics) months. Of the 906 treated patients, 688 (75.9%) continued their first-line medication; 147 (16.2%) changed treatment, often to a combination therapy (e.g., VMAT2 inhibitor plus antipsychotic, with/without other medication [n=43]); and 71 (7.8%) discontinued treatment for >90 days (mean 1-2 years). 29.4% (64/218) of patients switched/discontinued their second-line therapy.

Conclusions

In this analysis of a natural history database, 36.1% of patients with chorea received medication intended for chorea. Patients generally continued their first-line treatment, but those who switched often received a combination therapy. Continued research is needed to better understand optimal treatment patterns for chorea in patients with HD.

Authors/Disclosures
Erin Furr-Stimming, MD, FAAN (University of Texas Health Science Center-Houston) PRESENTER	Dr. Furr-Stimming has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Michael J. Fox Foundation. Dr. Furr-Stimming has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Medscape. Dr. Furr-Stimming has received personal compensation in the range of $500-$4,999 for serving as a Consultant for MedPage. Dr. Furr-Stimming has received personal compensation in the range of $500-$4,999 for serving as a Consultant for PTC Therapeutics. Dr. Furr-Stimming has received personal compensation in the range of $500-$4,999 for serving as a Consultant for MedPage. Dr. Furr-Stimming has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Wave Life Sciences. Dr. Furr-Stimming has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Teva Pharmaceuticals. Dr. Furr-Stimming has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for PTC Therapeutics. Dr. Furr-Stimming has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Wave Life Sciences. Dr. Furr-Stimming has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Latus Bio. The institution of Dr. Furr-Stimming has received research support from Roche/Genetech. The institution of Dr. Furr-Stimming has received research support from Uniqure. The institution of Dr. Furr-Stimming has received research support from CHDI. The institution of Dr. Furr-Stimming has received research support from Huntington Study Group/Neurocrine Bioscienes. The institution of Dr. Furr-Stimming has received research support from NIH/University of Iowa. The institution of Dr. Furr-Stimming has received research support from Sage Therapeutics. The institution of Dr. Furr-Stimming has received research support from HDSA. Dr. Furr-Stimming has received publishing royalties from a publication relating to health care. Dr. Furr-Stimming has received publishing royalties from a publication relating to health care. Dr. Furr-Stimming has a non-compensated relationship as a Committee member with AAN UES Committee that is relevant to AAN interests or activities.
Daniel O. Claassen, MD, FAAN (Vanderbilt University Medical Center)	Dr. Claassen has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Spark . The institution of Dr. Claassen has received personal compensation in the range of $50,000-$99,999 for serving as a Consultant for Alterity. Dr. Claassen has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for University of Michigan. Dr. Claassen has received personal compensation in the range of $500-$4,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for HD Insights. The institution of Dr. Claassen has received research support from NIH. The institution of Dr. Claassen has received research support from CHDI. The institution of Dr. Claassen has received research support from HDSA. The institution of Dr. Claassen has received research support from Department of Defense. The institution of Dr. Claassen has received research support from CHDI. The institution of Dr. Claassen has received research support from Genentech/ Roche.
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Dietrich Haubenberger, MD, FAAN (Neurocrine Biosciences)	Dr. Haubenberger has received personal compensation for serving as an employee of Neurocrine Biosciences, Inc. Dr. Haubenberger has stock in Neurocrine Biosciences. Dr. Haubenberger has a non-compensated relationship as a Member of the Board with American Society for Experimental Neurotherapeutics that is relevant to AAN interests or activities.

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