Real-World Experience With Deutetrabenazine for Huntington Disease Chorea.

Huntington disease (HD) is a hereditary neurodegenerative disorder with a hallmark feature of chorea. While no disease-modifying therapies currently exist for HD, symptomatic treatment of HD-associated chorea includes US Food and Drug Administration-approved vesicular monoamine transporter type 2 inhibitors-tetrabenazine and deutetrabenazine. Deutetrabenazine was more recently approved (2017), and while structurally similar to tetrabenazine, deutetrabenazine has a unique pharmacokinetic profile that allows for a longer half-life, reduced plasma fluctuations, and less frequent dosing. In pivotal trials, deutetrabenazine seemed to have an improved safety and tolerability profile over tetrabenazine but real-world data to confirm this are lacking. Here, we evaluate our real-world clinical experience with deutetrabenazine for HD-associated chorea. We performed a retrospective chart review of all patients with HD who initiated treatment with deutetrabenazine from January 2017 to May 2019 at the University of Alabama at Birmingham. Total maximal chorea scores, patient-reported subjective efficacy, dosing information, and subjective reports of adverse events (AEs) were abstracted for each patient. Our review included 58 patients with a mean length of treatment of 476.4 days. In the reviewed time period, the mean treatment difference in total maximal chorea scores was 4.4. The combined total rate of occurrence of any AEs was relatively low, at 32.8%, and the most commonly reported AEs were sedation (15.5%), insomnia (6.9%), and diarrhea (3.4%). Our real-world data support current literature indicating that deutetrabenazine is an effective and well-tolerated treatment for HD-associated chorea. Further studies repeating this on a larger scale, across a greater geography and practice pattern, are needed. © 2023, The American College of Clinical Pharmacology.

Citation

Kendall Curtis, Victor Sung. Real-World Experience With Deutetrabenazine for Huntington Disease Chorea. Journal of clinical pharmacology. 2024 Feb;64(2):178-181

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PMID: 37565322

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