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Article Abstract
Background: Dystonia may respond to VMAT2 inhibition.
Objectives: Providing pilot data on the safety, tolerability, and efficacy of deutetrabenazine in non dopa-responsive dystonia.
Methods: Deutetrabenazine was titrated by adults with isolated dystonia. Primary study endpoints included the proportion who maintained the individual, maximum tolerated dose for 6 weeks, and how many titrated to 48 mg/day. Secondary endpoints included rates of QTc prolongation/arrhythmias, suicidality, excessive daytime sleepiness, cognitive decline, and drug-induced parkinsonism. Exploratory endpoints for clinical efficacy were assessed.
Results: Among 15 participants, four (26.7%) withdrew early and six (40%) titrated to 48 mg/day. Common adverse events included fatigue and diarrhea. Secondary safety endpoints did not change significantly, but MDS-UPDRS III scores worsened by ≥3 points in seven participants (46.7%). PGI-C and the blinded CGI-C and GDS improved in three women with blepharospasm.
Conclusions: Most participants tolerated deutetrabenazine for 6 weeks, and those with blepharospasm may have benefitted from its use.
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| http://www.ncbi.nlm.nih.gov/pmc/articles/PMC11998685 | PMC |
| http://dx.doi.org/10.1002/mdc3.14327 | DOI Listing |
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