Objective: To evaluate real-world use and outcomes with deutetrabenazine for Huntington’s disease.

Background: Grade A evidence for Huntington’s disease chorea treatment includes tetrabenazine and deutetrabenazine. Use of the latter since US FDA approval has had limited description in the literature. We sought to compare our center’s experience with deutetrabenazine to its pivotal trial and the one post-approval study published to date.

Method: All patients prescribed deutetrabenazine from the University of Alabama at Birmingham Huntington’s Disease Society of America Center of Excellence were identified retrospectively (n=58) with data extracted from the medical record. The total maximal chorea (TMC) scores recorded prior to deutetrabenazine initiation and after dose titration were identified as well as maximal and current dosages.

Results: A preliminary analysis of data available through February 2020 resulted in a sample of 47% men of an average age of 52 years (SEM 1.8) at the time baseline TMC score was recorded.  The mean maximum titrated dose was 37 mg (2.9). Four patients (7%) stopped the medication, and the remaining patients had a mean final dose of 36 mg (3.1). The FDA-recommended maximum dosage (48 mg) was exceeded in 7 patients (12%; maximum of 96 mg in 3 patients) with no increase in adverse events. The mean baseline TMC was 12.2 (0.7), and the mean TMC stable dose was reached was 7.5 (0.6), with a significant mean treatment difference of -4.9 (0.7; p<0.0001). Deutetrabenazine generally was well tolerated with no serious adverse events recorded.

Conclusion: Our Center’s use of deutetrabenazine closely approximates the data from its pivotal trial for Huntington’s disease chorea for TMC improvement and safety. Though our average final dose is slightly lower than the pivotal trial’s, some patients required exceeding the maximum recommended daily dose for symptom control and perhaps were able to do so by the drug’s favorable tolerability profile.

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MDS Abstracts - https://www.mdsabstracts.org/abstract/deutetrabenazine-for-huntingtons-disease-chorea-a-single-centers-experience/