Objective: To assess real-world effectiveness and safety of deutetrabenazine (DTBZ) for chorea associated with Huntington disease (HD).

Background: Real-world evidence on the impact of DTBZ for treatment of HD-related chorea is limited and may provide value to clinicians and patients.

Method: This non-interventional, retrospective chart review study from an HD-specialty clinical practice at the University of Alabama at Birmingham (UAB) had patients with a diagnosis of chorea associated with HD, DTBZ initiation from April 2017 through December 2021, ≥2 clinical encounters at UAB, and ≥3 months of chorea-related care records post-DTBZ initiation. The last Unified HD Rating Scale–Total Maximal Chorea (TMC) score within 3 months prior to DTBZ initiation and the first after reaching the last stable dose during follow-up were analyzed.

Results: The mean (SD) age of the 80 patients in the study was 52.1 (12.6) years, and 45 (56.3%) were female. The mean (SD) last stable dose of DTBZ was 39.2 (21.3) mg/day (n=73). TMC score analysis focused on 3 groups of patients with both pre- and post-DTBZ initiation TMC scores who reached a stable dose (n=50): those with no prior tetrabenazine (TBZ) or DTBZ use (n=30), those with prior TBZ use with a gap before DTBZ initiation (n=8), and those with an “overnight” switch from TBZ to DTBZ (n=9). Mean (SD) pre-DTBZ initiation TMC scores were 11.1 (5.4), 18.3 (5.0), and 9.9 (3.3), respectively. Mean (SD) TMC score changes for these groups were −3.7 (4.5), −7.8 (2.8), and −2.1 (4.2). Decreases in TMC scores were observed for 24 (80.0%), 8 (100%), and 7 (77.8%) patients. Overall, 27 (33.8%) patients had ≥1 adverse event recorded between DTBZ initiation and the first visit with a TMC score after reaching their last DTBZ stable dose or DTBZ discontinuation for those without a stable dose, including sedation/somnolence/fatigue (n=14, 17.5%), diarrhea (n=3, 3.8%), falls/balance issues (n=3, 3.8%), akathisia (n=2, 2.5%), anxiety (n=2, 2.5%), depression (n=1, 1.3%), and parkinsonism (n=1, 1.3%).

Conclusion: Improvements in TMC scores in all groups in this real-world study demonstrate DTBZ effectiveness in patients with HD-related chorea, which supports previous clinical trial data. In this study, the greatest improvement was seen in patients with a gap between TBZ and DTBZ use. In addition, the observed safety profile supports the known safety profile of DTBZ in this population.

« Back to 2023 International Congress

MDS Abstracts - https://www.mdsabstracts.org/abstract/real-world-effectiveness-and-safety-of-deutetrabenazine-in-patients-with-chorea-associated-with-huntington-disease/