Category: Pediatric Movement Disorders
Objective: STIM-CP is a prospective single-arm multicenter study to investigate the effects of deep brain stimulation of the globus pallidus internus (GPi-DBS) in pediatric patients with pharmacorefractory dyskinetic cerebral palsy (DCP).
Background: GPi-DBS can be effective in patients with DCP, but improvement of dystonia severity is often less pronounced compared to patients with inherited isolated dystonia. It can be associated with favorable quality of life outcomes, even in patients without any overt changes in dystonia severity, suggesting that the sole use of clinical rating scales may not reflect the full treatment effects.
Method: 16 Patients with DCP due to perinatal asphyxia (age 7-18 years) were recruited by seven clinical sites in order to systematically assess motor and non-motor domains before and after GPi-DBS. We hypothesized a 10 points improvement in quality of life assessed by the Caregiver Priorities & Child Health Index of Life with Disabilities (CPCHILD) questionnaire as the primary endpoint from baseline to 12 months under active stimulation. After 12 months patients were randomly assigned to a blinded crossover to receive active or sham stimulation for 24 hours each. Severity of dystonia and chorea were assessed by two masked experts using standardized videos.
Results: At 12 months the CPCHILD increased by 4.2 points ± 10.4 (p=0.125). Among secondary outcome parameters, there was a significant improvement in the Canadian Occupational Performance Measure performance score and in the physical health component of the SF-36, indicated by both, the patients and the caregivers (patients: 5.1 points ± 6.2, p=0.028; caregivers: 4.6 points ± 7.3, p=0.029). The Gross Motor Function Measure-66 increased by 1.2 points ± 3.2 (p=0.052). During cross-over dystonia and chorea severity did not change. All serious adverse events resolved without sequelae.
Conclusion: The increase in quality of life did not reach statistical significance. However, GPi-DBS improved motor function after 12 months. Extended follow-up in larger cohorts will ascertain the impact of DBS on pediatric DCP patients to guide treatment decisions in these often-severely disabled patients. There is not enough evidence yet to recommend GPi-DBS as routine treatment to improve quality of life in these patients.
To cite this abstract in AMA style:A. Koy, A. Kühn, J. Huebl, G-H. Schneider, A. van Riesen, M. Eckenweiler, C. Rensing-Zimmermann, V. Coenen, J. Krauss, A. Saryyeva, H. Hartmann, M. Haeussler, J. Volkmann, C. Matthies, A. Horn, A. Schnitzler, J. Vesper, A. Gharabaghi, D. Weiss, A. Bevot, W. Marks, A. Pomykal, E. Monbaliu, G. Borck, J. Mueller, R. Prinz-Langenohl, T. Dembek, V. Visser-Vandewalle, J. Wirths, P. Schiller, M. Hellmich, L. Timmermann. Effect of bilateral pallidal stimulation on quality of life of pediatric patients with dyskinetic cerebral palsy (STIM-CP): A prospective single-arm multicenter study with a subsequent randomized double-blind crossover phase [abstract]. Mov Disord. 2021; 36 (suppl 1). https://www.mdsabstracts.org/abstract/effect-of-bilateral-pallidal-stimulation-on-quality-of-life-of-pediatric-patients-with-dyskinetic-cerebral-palsy-stim-cp-a-prospective-single-arm-multicenter-study-with-a-subsequent-randomized-doub/. Accessed December 5, 2023.
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