Long-term results of deep brain stimulation in a cohort of eight children with isolated dystonia

• Published in: Journal of neurology 2016-11, Vol.263 (11), p.2319-2326
• Main Authors: Krause, P., Lauritsch, K., Lipp, A., Horn, A., Weschke, B., Kupsch, A., Kiening, K. L., Schneider, G.-H., Kühn, A. A.
• Format: Article
• Language: English
• Subjects: Adolescent; Analysis of Variance; Child; Cohort Studies; Deep brain stimulation; Deep Brain Stimulation - methods; Dystonia; Dystonia - diagnostic imaging; Dystonia - therapy; Electrodes; Female; Globus Pallidus - physiology; Humans; Imaging, Three-Dimensional; Magnetic Resonance Imaging; Male; Medicine; Medicine & Public Health; Movement disorders; Mutation; Neurology; Neuroradiology; Neurosciences; Neurosurgery; Original Communication; Outcome Assessment (Health Care); Patients; Severity of Illness Index; Surgery; Treatment Outcome
• ISSN: 0340-5354; 1432-1459
• DOI: 10.1007/s00415-016-8253-6

Pallidal deep brain stimulation (DBS) is an established treatment for patients with severe isolated dystonia. However, clinical evidence for the long-term use of DBS in children is limited and controlled trials have not yet been conducted. Here, we provide the long-term results of up to 13 years of pallidal DBS in eight pediatric patients with generalized idiopathic or hereditary isolated dystonia (five males, mean age at surgery 12.5 ± 3.5 years), as assessed by retrospective video rating. Video rating was performed at three time points: pre-operative, 1-year short-term follow-up (1y-FU) and long-term last FU (LT-FU, up to 13 years). Symptom severity and disability were assessed using the Burke–Fahn–Marsden Dystonia Rating Scale (BFMDRS). Disability scores were obtained from clinical charts and during the last FU. The mean improvement in BFMDRS motor score was 54.4 ± 8.9 % at 1y-FU and 42.9 ± 11.6 % at LT-FU; the disability scores improved by 59.8 ± 10.3 and 63.3 ± 7.8 %, respectively. Electrode dislocation was noted in one patient and implantable pulse generator dislocation in another, both requiring surgical intervention; no further serious adverse events occurred. Our study presents the first blinded video rating assessment of the short- and long-term effects of pallidal DBS in children with idiopathic or hereditary isolated dystonia. Results confirm that pallidal DBS is a safe and efficacious long-term treatment in children, with overall motor improvement similar to that described in controlled trials in adults.