Protocol for combined N-of-1 trials to assess cerebellar neurostimulation for movement disorders in children and young adults with dyskinetic cerebral palsy.
| العنوان: | Protocol for combined N-of-1 trials to assess cerebellar neurostimulation for movement disorders in children and young adults with dyskinetic cerebral palsy. |
|---|---|
| المؤلفون: | San Luciano M; University of California, San Francisco, Weill Institute for Neurosciences., Oehrn CR; University of California, San Francisco., Wang SS; University of California, San Francisco., Tolmie JS; University of California, San Francisco., Wiltshire A; University of California, San Francisco., Graff RE; University of California, San Francisco., Zhu J; University of California, San Francisco., Starr PA; University of California, San Francisco. |
| المصدر: | Research square [Res Sq] 2024 Apr 01. Date of Electronic Publication: 2024 Apr 01. |
| نوع المنشور: | Preprint |
| اللغة: | English |
| بيانات الدورية: | Country of Publication: United States NLM ID: 101768035 Publication Model: Electronic Cited Medium: Internet NLM ISO Abbreviation: Res Sq Subsets: PubMed not MEDLINE |
| مستخلص: | Background: Movement and tone disorders in children and young adults with cerebral palsy are a great source of disability. Deep brain stimulation (DBS) of basal ganglia targets has a major role in the treatment of isolated dystonias, but its efficacy in dyskinetic cerebral palsy (DCP) is lower, due to structural basal ganglia and thalamic damage and lack of improvement of comorbid choreoathetosis and spasticity. The cerebellum is an attractive target for DBS in DCP since it is frequently spared from hypoxic ischemic damage, it has a significant role in dystonia network models, and small studies have shown promise of dentate stimulation in improving CP-related movement and tone disorders. Methods: Ten children and young adults with DCP and disabling movement disorders with or without spasticity will undergo bilateral DBS in the dorsal dentate nucleus, with the most distal contact ending in the superior cerebellar peduncle. We will implant Medtronic Percept, a bidirectional neurostimulator that can sense and store brain activity and deliver DBS therapy. The efficacy of cerebellar DBS in improving quality of life and motor outcomes will be tested by a series of N-of-1 clinical trials. Each N-of-1 trial will consist of three blocks, each consisting of one month of effective stimulation and one month of sham stimulation in a random order with weekly motor and quality of life scales as primary and secondary outcomes. In addition, we will characterize abnormal patterns of cerebellar oscillatory activity measured by local field potentials from the intracranial electrodes related to clinical assessments and wearable monitors. Pre- and 12-month postoperative volumetric structural and functional MRI and diffusion tensor imaging will be used to identify candidate imaging markers of baseline disease severity and response to DBS. Discussion: Our goal is to test a cerebellar neuromodulation therapy that produces meaningful changes in function and well-being for people with CP, obtain a mechanistic understanding of the underlying brain network disorder, and identify physiological and imaging-based predictors of outcomes useful in planning further studies. Trial Registration: ClinicalTrials.gov NCT06122675, first registered November 7, 2023. Competing Interests: Competing interests The authors declare no competing interests. |
| التعليقات: | Update in: BMC Neurol. 2024 Apr 29;24(1):145. (PMID: 38684956) |
| معلومات مُعتمدة: | UH3 NS128297 United States NS NINDS NIH HHS |
| فهرسة مساهمة: | Keywords: Dyskinetic cerebral palsy; cerebellum; children; deep brain stimulation; dentate nucleus; electrophysiology; young adults |
| سلسلة جزيئية: | ClinicalTrials.gov NCT06122675 |
| تواريخ الأحداث: | Date Created: 20240422 Latest Revision: 20240513 |
| رمز التحديث: | 20240513 |
| مُعرف محوري في PubMed: | PMC11030503 |
| DOI: | 10.21203/rs.3.rs-4077387/v1 |
| PMID: | 38645256 |
| قاعدة البيانات: | MEDLINE |
| DOI: | 10.21203/rs.3.rs-4077387/v1 |
|---|