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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.

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Acknowledgments

AAK, PK and GHS were supported by a grant from the German Research foundation (DFG), KFO247. KL was supported by a grant from the German Research Foundation (DFG, LO 1555/3-2). AK received Grants from the German Research Council and the German Ministry of Education and Research.

Author information

Correspondence to A. A. Kühn.

Ethics declarations

Conflicts of interest

AA. Kühn was funded by the German Research Foundation (DFG KFO247) and received speakers honoraria or consultancies from Medtronic, Boston Scientific and St. Jude Medical, and travel Grants from Ipsen Pharma and Merz. P. Krause was funded by the German Research Foundation (DFG KFO247). K. Lauritsch: none. A. Horn was funded by the German Research Foundation (DFG KFO247) and received grants from the junior clinical scientist program and the Max-Rubner-Price of the Charité. A. Lipp: None. B. Weschke received a grant from the EC within the Collaborative Project “EPISTOP” of the Seventh Framework Program. A. Kupsch received grants from the German Research Council and the German Ministry of Education and Research, belongs to the Advisory Board of ´Medtronic USA´ and received honoraria from Allergan, Boehringer Ingelheim, Ipsen Pharma, Lundbeck, Medtronic, Merck, Merz Pharmaceuticals, Orion, St. Jude, UCB. G.H. Schneider was supported by a grant from the German Research Foundation (DFG), KFO247. K. Kiening: none.

Funding sources for study

The study was supported by the German Research Foundation (DFG) KFO 247.

Ethical standard

The study was approved by the local ethics committee of the Charité, University Medicine Berlin.

Informed consent

All patients gave written informed consent.

Electronic supplementary material

Below is the link to the electronic supplementary material.

Video legend: Segment 1: Patient 2 with generalized dystonia with predominant phasic movements of the head as well as right upper extremity and dystonic gait disorder with bending of the trunk, inward rotation and involuntary flexion of the legs while walking. At no time did she experience speech and/or swallowing problems. Segment 2: After 21 months of pallidal DBS, phasic involuntary movements of the head and right arm were almost completely suppressed, along with significant improvement of cervical and truncal dystonia as well as relevant reduction of dystonia of the upper extremities and dystonic gait. Segment 3: DBS effects remaining stable for more than 13 years, with sustained relief of disability and cessation of the phasic involuntary head and upper limb movements. Mild residual dystonia of the limbs results in minor limitations of fine motor skills and gait. (WMV 43560 kb)

Video legend: Segment 1: Patient 2 with generalized dystonia with predominant phasic movements of the head as well as right upper extremity and dystonic gait disorder with bending of the trunk, inward rotation and involuntary flexion of the legs while walking. At no time did she experience speech and/or swallowing problems. Segment 2: After 21 months of pallidal DBS, phasic involuntary movements of the head and right arm were almost completely suppressed, along with significant improvement of cervical and truncal dystonia as well as relevant reduction of dystonia of the upper extremities and dystonic gait. Segment 3: DBS effects remaining stable for more than 13 years, with sustained relief of disability and cessation of the phasic involuntary head and upper limb movements. Mild residual dystonia of the limbs results in minor limitations of fine motor skills and gait. (WMV 43560 kb)

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Krause, P., Lauritsch, K., Lipp, A. et al. Long-term results of deep brain stimulation in a cohort of eight children with isolated dystonia. J Neurol 263, 2319–2326 (2016) doi:10.1007/s00415-016-8253-6

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Keywords

  • DYT1 dystonia
  • Pallidal DBS
  • Long-term effects
  • Idiopathic dystonia