Abstract

<h3>Objective:</h3> To describe the effectiveness and safety of vesicular monoamine transporter 2 inhibitor deutetrabenazine (DTBZ) combined with antipsychotic agents (APs) in patients with Huntington disease (HD)-related chorea in a real-world setting. <h3>Background:</h3> DTBZ effectiveness and safety for HD-related chorea have been well documented in clinical trials; however, patients receiving APs were not included. Safety and effectiveness data for DTBZ+AP are needed to inform real-world practice. <h3>Design/Methods:</h3> Data were collected from charts of patients with HD-associated chorea who received DTBZ+AP at Vanderbilt University Medical Center from April 3, 2017, until data collection (May–November 2021). Effectiveness was assessed by Total Maximal Chorea (TMC) scores over time. Safety was assessed via adverse drug reactions (ADRs) based on a predefined list of known DTBZ ADRs. <h3>Results:</h3> Among 57 patients treated with DTBZ+AP, 13 started APs first and had TMC scores available within 1 month before and any time after DTBZ addition, and 8 had TMC scores available within 1 month before AP initiation. After initiating APs and before adding DTBZ, TMC scores improved for 2/8 (25%) patients and worsened for 6 (75%). After adding DTBZ, TMC scores further improved for 8/13 (62%) patients, did not change for 1 (8%), and worsened for 4 (31%). In patients with history of tetrabenazine use, TMC scores improved after adding DTBZ for 4/8 (50%), versus 4/5 (80%) patients without history. Among all 57 patients treated with DTBZ+AP, 25 (44%) patients had ≥1 new/continuing ADR, with agitation, akathisia (both n=7; 12%), and somnolence (n=4; 7%) being most frequent; 2 (4%) patients discontinued DTBZ because of ADRs/tolerability issues while receiving combination treatment. <h3>Conclusions:</h3> In this retrospective chart review, adding DTBZ to APs improved motor signs and had a favorable benefit-risk profile. However, prospective, controlled trials investigating the efficacy and safety of DTBZ+AP in patients with HD-related chorea are needed. <b>Disclosure:</b> 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 . 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 $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 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. The institution of Dr. Claassen has received research support from Prilenia. The institution of Dr. Claassen has received research support from Neurocrine/ HSG. Dr. Romdhani has nothing to disclose. Rajeev Ayyagari, PhD has received personal compensation for serving as an employee of Analysis Group, Inc. . Debbie Goldschmidt has received personal compensation for serving as an employee of Analysis Group. Ms. Moroz has nothing to disclose. Ms. Hernandez has nothing to disclose. Nayla Chaijale has received personal compensation for serving as an employee of Teva Pharmaceuticals. Dr. Leo has received personal compensation for serving as an employee of Teva Pharmaceutical Industries.

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