Longitudinal Treatment Patterns for Chorea in Patients with Huntington Disease: Data from Enroll-HD (S36.009)

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 under treated 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.

Disclosure: Dr. Furr-Stimming has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Wave Pharmaceuticals. Dr. Furr-Stimming has received personal compensation in the range of $500-$4,999 for serving as a Consultant 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 Amneal. 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 Sunovion. Dr. Furr-Stimming has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Sunovion. Dr. Furr-Stimming has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Teva Pharmaceuticals. The institution of Dr. Furr-Stimming has received research support from Cures within Reach. The institution of Dr. Furr-Stimming has received research support from Huntington's Disease Society of America. 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 Vaccinex. The institution of Dr. Furr-Stimming has received research support from Huntington Study Group/Neurocrine. Dr. Claassen has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Lundbeck. Dr. Claassen has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Teva Neuroscience. Dr. Claassen has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Spark . Dr. Claassen 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 Neuroscience. 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 Wave Life Sciences. 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 Griffin Family Foundation. The institution of Dr. Claassen has received research support from Neurocrine. The institution of Dr. Claassen has received research support from Vaccinex. The institution of Dr. Claassen has received research support from AbbVie. The institution of Dr. Claassen has received research support from CHDI. The institution of Dr. Claassen has received research support from Genentech/ Roche. Ginny Sen has received personal compensation for serving as an employee of Neurocrine Biosciences, Inc. Mallory Farrar has received personal compensation for serving as an employee of Neurocrine Biosciences, Inc. Ericha Franey has received personal compensation for serving as an employee of Neurocrine Biosciences, Inc.. Ericha Franey has received stock or an ownership interest from Ericha Franey. Chuck Yonan has received personal compensation for serving as an employee of Neurocrine Biosciences, Inc. Dr. Haubenberger has received personal compensation for serving as an employee of Neurocrine Biosciences, Inc. Dr. Haubenberger has received personal compensation for serving as an employee of Neurocrine Biosciences. Dr. Haubenberger has stock in Neurocrine Biosciences.