Thalamic deep brain stimulation for acquired dystonia in children and young adults: a phase 1 clinical trial.

摘要: OBJECTIVE The aim of this study was to evaluate the feasibility and preliminary efficacy safety combined bilateral ventralis oralis posterior/ventralis intermedius (Vop/Vim) deep brain stimulation (DBS) for treatment acquired dystonia in children young adults. Pallidal DBS is efficacious severe, medication-refractory isolated dystonia, providing 50%-60% long-term improvement. Unfortunately, pallidal response rates are modest unpredictable, with frequent nonresponders. Acquired most commonly caused by cerebral palsy, more common than pediatric populations recalcitrant standard treatments. Given limitations there a need explore alternative targets. Preliminary evidence has suggested that thalamic may be dystonia. METHODS Four participants, 3 perinatal injuries 1 postencephalitic symptomatic underwent Vop/Vim bimonthly evaluations 12 months. primary outcome change Burke-Fahn-Marsden Dystonia Rating Scale (BFMDRS) Barry-Albright (BADS) scores between baseline 12-month assessments. Video documentation used blinded ratings. Secondary outcomes included evaluation spasticity (Modified Ashworth score), quality life (Pediatric Quality Life Inventory [PedsQL] modified Unified Parkinson's Disease Part II [UPDRS-II] scores), neuropsychological Adverse events were monitored safety. RESULTS All participants tolerated procedure well, no concerns or serious adverse events. There an average improvement 21.5% BFMDRS motor subscale score, but only 1.6% according BADS score. Following video review, severity ratings even modest. outcomes, however, encouraging, disability score improving 15.7%, PedsQL total 27%, UPDRS-II 19.3%. Neuropsychological assessment findings unchanged year after surgery. CONCLUSIONS Bilateral neuromodulation well tolerated. Primary secondary showed highly variable effect sizes comparable those population. As previously described, improvements not reflected scales, suggesting development scales specifically dystonia.Clinical trial registration no.: NCT03078816 (clinicaltrials.gov).