Objective: We aim to evaluate the effectiveness of VMAT2 inhibitors in the treatment of stereotypy.

Background: Stereotypies are continual, rhythmic, patterned movements that can occur  in typically-developing children, and are more common in those with cognitive deficits.  Particularly in the latter population, the movements can result in inadvertent and devastating self-injury, or interfere with the ability to be present in social settings.  In patients with typical cognition, behavioral retraining has been effective;, however, for patients with intellectual disability ,other avenues are explored.  VMAT2 inhibitors have been effective in the treatment of several movement disorders.  Outside of use for self-mutilating behaviors in Lesch Nyhan syndrome, the utility of VMAT2 inhibitors in stereotypy has been unclear.

Method: A retrospective review included patients under 18 years with stereotypy requiring treatment (due to either self-injurious behaviors or inhibiting socialization/therapy) who were evaluated at Texas Children’s pediatric movement disorders clinic from 2014-2019 and underwent a trial of tetrabenazine.

Results: Six patients met inclusion criteria.  All patients had severe or profound intellectual disability.  The ages of patients ranged from 4 to 15 years.  Three patients were treated for self-injurious motor stereotypy that placed them at risk for cervical spine injury or retinal detachment.  Of these patients, two had 50% reduction of the concerning movements.  One was later discontinued due to agitation.  Three patients had , near-constant vocal stereotypy preventing meaningful social interactions.  Of these patients, two demonstrated a notable decrease in the frequency of the vocalizations.  One was later discontinued in the setting of acute dystonic reactions (which recurred after switching to deutetrabenazine).

Conclusion: Of six patients with impairing stereotypy, four demonstrated meaningful benefit when treated with tetrabenazine .

« Back to MDS Virtual Congress 2020

MDS Abstracts - https://www.mdsabstracts.org/abstract/utility-of-vmat2-inhibitors-in-management-of-stereotypy-in-children-a-series-of-six-patients/