title: Long-term results of deep brain stimulation in a cohort of eight children with isolated dystonia.
abstract: 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.
authors: Department of Neurology, Charité, University Medicine Berlin, Campus Virchow, Berlin, Germany.
authors: P
authors: http://orcid.org/0000-0001-9063-0556
authors: P
authors: Krause
authors: Department of Neurology, Charité, University Medicine Berlin, Campus Virchow, Berlin, Germany.
authors: K
authors: K
authors: Lauritsch
authors: Department of Neurology, Charité, University Medicine Berlin, Campus Virchow, Berlin, Germany.
authors: A
authors: A
authors: Lipp
authors: Department of Neurology, Charité, University Medicine Berlin, Campus Virchow, Berlin, Germany.
authors: A
authors: A
authors: Horn
authors: Department of Neuropediatrics, Charité, University Medicine Berlin, Campus Virchow, Berlin, Germany.
authors: B
authors: B
authors: Weschke
authors: Department of Neurology and Stereotactic Neurosurgery, University Medicine of Magdeburg, Magdeburg, Germany.
authors: A
authors: A
authors: Kupsch
authors: Department of Neurosurgery, Division of Stereotactic Neurosurgery, University Medicine Heidelberg, Heidelberg, Germany.
authors: K L
authors: KL
authors: Kiening
authors: Department of Neurosurgery, Charité, University Medicine Berlin, Campus Virchow, Berlin, Germany.
authors: G-H
authors: GH
authors: Schneider
authors: Department of Neurology, Charité, University Medicine Berlin, Campus Virchow, Berlin, Germany. andrea.kuehn@charite.de.
authors: A A
authors: AA
authors: Kühn
mesh_terms: D000293:Adolescent; D000704:Analysis of Variance; D002648:Child; D015331:Cohort Studies; D046690:Deep Brain Stimulation / Q000379:methods*; D004421:Dystonia / Q000000981:diagnostic imaging / Q000628:therapy*; D005260:Female; D005917:Globus Pallidus / Q000502:physiology*; D006801:Humans; D021621:Imaging, Three-Dimensional; D008279:Magnetic Resonance Imaging; D008297:Male; D017063:Outcome Assessment, Health Care; D012720:Severity of Illness Index; D016896:Treatment Outcome
keywords: DYT1 dystonia; Idiopathic dystonia; Long-term effects; Pallidal DBS