Outcomes of Bilateral Globus Pallidus Internus Deep Brain Stimulation in GNAO1-Related Disorder: An International Multicenter Experience.
Bilateral pallidal deep brain stimulation was associated with fewer dyskinetic crises in GNAO1-related disorder
Outcomes of Bilateral Globus Pallidus Internus Deep Brain Stimulation in GNAO1-Related Disorder: An International Multicenter Experience.
Small series suggest benefit from bilateral globus pallidus internus deep brain stimulation (GPi-DBS), but optimal timing, patient selection, long-term outcomes, and genotype-specific response remain unclear.
GNAO1-related disorder is a severe childhood-onset hyperkinetic movement disorder punctuated by life-threatening dyskinetic crises.
Retrospective multicenter cohort study conducted through the DBSMatchMaker platform, including children and young adults with genetically confirmed GNAO1-related disorder treated with bilateral GPi-DBS across 17 centers.
Over half (52.2%) were implanted emergently during dyskinetic status.
The Burke Fahn Marsden Dystonia Rating Scale (Burke Fahn Marsden Dystonia Rating Scale) motor scores improved by 27.5% (p < 0.001, Cohen's d = 1.12); Clinical Global Impression was improved in 93.5%, with greater benefit for chorea than dystonia.
Pain, sleep, and medication burden improved; functional classification was largely unchanged.
Complications occurred in 19.6%, predominantly in emergent cases.
Bilateral GPi-DBS was associated with sustained prevention of dyskinetic crises and reduction of overall disease burden in GNAO1-related disorder, with limited impact on functional classification.
These findings support earlier elective neuromodulation before recurrent crises drive cumulative morbidity and identify GPi-DBS as a crisis-preventive, symptom-modifying intervention rather than a restorative one. ANN NEUROL 2026.