VMAT2 inhibitors may offer effective symptom relief for pediatric hyperkinetic movement disorders, according to a new retrospective study reviewing over a decade of clinical use. While these dopamine-depleting agents are approved for certain adult conditions, their off-label use in children has remained limited due to regulatory constraints. This large-scale analysis provides new clarity on prescribing patterns, treatment outcomes, and barriers to access in pediatric settings.
The study, conducted at a pediatric movement disorders clinic between 2011 and 2023, reviewed 359 prescriptions of VMAT2 inhibitors among 340 children, most of whom were male (65.3%) with an average age of 11.9 years. Tetrabenazine was the most frequently prescribed medication, accounting for 94% of prescriptions, followed by deutetrabenazine and valbenazine.
Tics were the most common clinical indication, representing nearly three-quarters of cases. Of the children who initiated therapy (75.8%), nearly two-thirds experienced clinical improvement, with an average Clinical Global Impression-Improvement (CGI-I) score of 1.8—suggesting that symptoms were “much” or “very much” improved. However, 11.5% did not experience meaningful symptom relief.
Side effects were commonly reported, with drowsiness being the most frequent. While 63.9% of patients noted adverse effects, only 10.1% discontinued treatment as a result. In some cases, transitioning from tetrabenazine to deutetrabenazine or valbenazine resolved tolerability issues.
Despite promising therapeutic outcomes, access to treatment emerged as a major hurdle. More than half of all prescriptions faced at least one insurance denial. Additionally, nearly one in four patients were unable to begin treatment due to these barriers, and close to 20% reported significant financial concerns.
These real-world findings suggest that while VMAT2 inhibitors are clinically beneficial for many pediatric patients with hyperkinetic movement disorders, access and affordability remain persistent challenges. The results underscore the need for expanded pediatric research, clearer regulatory pathways, and broader insurance coverage to improve therapeutic equity.
Reference:
Manohar S et al. Real-World Experiences with VMAT2 Inhibitors in Pediatric Hyperkinetic Movement Disorders. Tremor Other Hyperkinet Mov (N Y). 2025:15:26.
