Chorea Severity Change Over Time in Huntington Disease and by Huntington Disease Stage (P1-11.010)

Abstract

<h3>Objective:</h3> To describe chorea severity over time in individuals with Huntington disease (HD) stratified by Total Functional Capacity (TFC). <h3>Background:</h3> Chorea, the most common motor symptom in HD, negatively affects quality of life and TFC. Addressing chorea is crucial to reduce the burden of motor symptomatology in HD. Chorea has been reported to increase initially, plateau, and potentially decrease over time; however, longitudinal data assessing chorea severity by disease stage are limited. <h3>Design/Methods:</h3> Participants were adults from Enroll-HD, a global, longitudinal, observational registry for individuals with or at risk for HD (data cut 2013–31 October 2020). The manifest population was stratified by baseline TFC score as a proxy for HD stage (TFC 7–13/stage 1–2 [early], 3–6/stage 3 [middle], 0–2/stage 4–5 [late]). Chorea severity was assessed by Total Maximal Chorea (TMC) score at baseline and at annual visits. Patients with ≥4 years of follow-up were selected for estimating chorea progression over time. <h3>Results:</h3> The manifest population was grouped by HD stage: early, n=7441; middle, n=2330; late, n=1120. Baseline TMC scores (mean [SD]) increased as HD stage progressed (early, 8.0 [4.7]; middle, 10.4 [5.8]; late, 10.6 [6.9]). For participants with ≥4 years of follow-up (n=1271), TMC scores increased over time in early-stage HD (n=963; years 1–2, 8.5 [5.0]; years 2–3, 9.1 [5.3]; years 3–4, 9.5 [5.6]), but plateaued at high levels in middle- (n=222; 10.6 [5.3]; 10.6 [6.0]; 10.1 [5.7], respectively) and late-stage HD (n=86; 10.4 [6.6]; 9.8 [6.9]; 9.7 [6.2]). <h3>Conclusions:</h3> Chorea severity increased in early-stage HD, and plateaued in middle- and late-stage HD. Chorea severity did not decrease longitudinally in any stage; however, the limited sample size for the follow-up period should be considered. These results support the need for increased awareness about the potential for persistent chorea in late-stage HD. <b>Disclosure:</b> 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. 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. 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.