Baseline Characteristics and Treatment Patterns of a Global Huntington Disease Population Stratified by Chorea Severity (P2-11.011)

Abstract

<h3>Objective:</h3> To describe baseline characteristics and treatment patterns in Huntington Disease-associated chorea by chorea severity. <h3>Background:</h3> Though multiple treatment options exist for HD-associated chorea, there are little real-world data on treatment patterns, especially across disease trajectory and variation by severity of chorea. <h3>Design/Methods:</h3> Participants were adults from “Enroll-HD”, a global observational registry for patients with HD and their families (data cut 2013 – 31 October, 2020). Data on age, sex, Total Maximal Chorea (TMC) score, vesicular monoamine transporter 2 (VMAT2) inhibitors, and antipsychotic agents (APs; typical, atypical, and other [lithium]) were collected at the baseline visit. Participants were grouped by chorea severity (TMC score categories) recorded at baseline (0–7, 8–14, 15–21, 22–28). <h3>Results:</h3> There were 10,903 participants (TMC 0–7, n=5055; TMC 8–14, n=4374; TMC 15–21, n=1310; TMC 22–28, n=164). 51% were female (51.2%, 51.5%, 51.4%, and 53.1%, respectively) and the mean age at baseline was 53.0 years (50.8, 54.5, 55.9, 54.4, respectively). Overall, VMAT2i use at baseline was 14.9% and increased with chorea severity (10.8%, 16.4%, 23.9%, 29.3%, respectively). The proportion of patients using APs at baseline was 34.5% overall and increased with chorea severity (29.9%, 35.1%, 47.8%, 57.3%, respectively). Overall, 5.8% of patients used VMAT2 inhibitors combined with APs, and combined use increased with chorea severity (3.9%, 5.8%, 11.5%, 15.9%, respectively). <h3>Conclusions:</h3> Use of medications to treat chorea was low across the spectrum of chorea severities but increased incrementally in patients with greater chorea severity. Use of a combination of VMAT2 inhibitors and APs at baseline was low but increased as chorea severity increased. These data suggest that HD-associated chorea may be under treated globally. More data are needed to understand chorea treatment considerations and the utility of VMAT2 inhibitors and APs for treating HD-associated chorea and comorbidities. <b>Disclosure:</b> Dr. Sung has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Teva Neuroscience. Dr. Sung has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Genentech. 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 as a Consultant for Michael J. Fox Foundation. 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 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 Huntington Study Group/Neurocrine. 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. Dr. Furr-Stimming has received publishing royalties from a publication relating to health care. Dr. Reshef has received personal compensation for serving as an employee of Teva. Ms. Willock has received personal compensation for serving as an employee of HCD Economics. Rinat Ribalov has received personal compensation for serving as an employee of TEVA. Miss Brighton has nothing to disclose. Dr. Leo has received personal compensation for serving as an employee of Teva Pharmaceutical Industries.