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अमूर्त

Effect of Deep Brain Stimulation of the Globus Pallidus Internus on Quality of Life in Young Patients with Dyskinetic Cerebral Palsy (STIM-CP): a Prospective Single-Arm Multicenter Trial with a Double-Blind Cross-Over at 12-Months Follow-up

Anne Koy, Andrea A. Kühn, Anne van Riesen, Julius Huebl, Rudolf Korinthenberg, Volker A. Coenen, Joachim K. Krauss, Andreas Wloch, Delia Lorenz, Martin Häussler, Alfons Schnitzler, Jan Vesper, Francois Alesch, Walter Lehmacher, Joerg Mueller, Guntram Borck, Karsten Witt, Tobias Bäumer, Steffen Berweck, Sebastian Schröder, Jürgen

Introduction: Deep brain stimulation of the globus pallidus internus (GPi-DBS) can be effective in patients with dyskinetic cerebral palsy (CP), but outcome in terms of dystonia severity is often less pronounced and more variable compared to patients with inherited monogenic dystonia. A few published case series encompassing retrospective data have shown that GPi-DBS is associated with favorable quality of life outcomes, even in patients without changes in the BFMDRS, suggesting that the sole use of clinical rating scales may not reflect the full effects of DBS, especially not in patients with acquired forms of dystonia such as dyskinetic CP. However, there is no prospective data on DBS effects in children.
We hypothesize an improvement in quality of life by GPi-DBS. Therefore, the mean change in the CPCHILD questionnaire from baseline to 12 months is chosen as the primary outcome parameter. Secondary outcome parameters encompass dystonia severity, motor function, speech, mood, cognition, pain, and quality of life of the carers before and up to 36 months after implantation.
Methods: This is a prospective single-arm study to investigate the effects of GPi-DBS in young patients with dyskinetic CP. 20 patients aged 7-18 years will be recruited in a multicenter setting. 12 months after lead implantation patients are randomized by a double-blind cross-over into two groups: One group starts with ongoing stimulation, in the other group stimulation is turned off. After 24 h they switch stimulation settings. Dystonia severity will be assessed at the end of each phase.
We hypothesize an improvement in quality of life by GPi-DBS. Therefore, the mean change in the CPCHILD questionnaire from baseline to 12 months is chosen as the primary outcome parameter. Secondary outcome parameters encompass dystonia severity, motor function, speech, mood, cognition, pain, and quality of life of the carers before and up to 36 months after implantation.
Discussion: This is the first prospective trial investigating the effects of GPi-DBS on motor and non-motor outcome in a cohort of exclusively pediatric patients in a multicenter setting. Furthermore, long-term data on effects and side effects of GPi-DBS in young patients with dyskinetic cerebral palsy will be generated.