Category: Dystonia: Clinical Trials and Therapy
Objective: To evaluate the motor and quality of life (QoL) outcomes of deep brain stimulation for pediatric dystonia with the following parameters: BFMDRS, total number of anti-dystonia medications, and QoL of the children and their caregivers.
Background: Dystonia causes significant physical disability and impact on children’s quality of life. Pharmacologic therapy is the mainstay of treatment for pediatric dystonia. In drug-resistant dystonia, DBS is proven to be effective. Despite the establishment of DBS as a treatment modality for pediatric dystonia, studies on its clinical outcomes remain scarce.
Method: Children with inherited, acquired or idiopathic dystonia who underwent DBS surgeries from the CHILD-DBS Registry were recruited into this prospective study with parental consent. The clinical outcomes were evaluated at four postoperative timepoints (up to 3-year post-DBS) with BFMDRS, total number of anti-dystonia medications, the Pediatric Quality of Life Inventory (PedsQL) and the Care-related Quality of Life instrument (CarerQol-7D).
Results: 18 children (8 males, 10 females) with drug-resistant dystonia of various etiologies (9 inherited, 8 acquired and 1 idiopathic) were recruited. The median age at diagnosis and age at DBS surgery were 1 and 11 years old respectively. Patients’ median follow-up duration was 1 year. At 1-year post-DBS: BFMDRS – motor improved by 28% while BFMDRS – disability remained unchanged. 50% of patients successfully weaned ≥1 anti-dystonia medications. The scores plateaued after 1-year post-DBS. At 3-year post-DBS, Total PedsQL score improved by 39% (physical domain 144% improvement, psychosocial domain 1% improvement) while CarerQol-7D score improved by 13%. There was a strong negative relationship between the BFMDRS – motor and CarerQol-7D at 1-year post-DBS (Pearson R= -0.85, p=0.03).
Conclusion: DBS is an effective treatment for drug-resistant dystonia in children. It provided significant dystonia relief and reduced the number of anti-dystonia medications at 1-year post-DBS. QoL of the children and their caregivers continued to improve even beyond 1-year post-DBS. Better post-DBS motor outcomes were associated with higher caregiver’s QoL.
To cite this abstract in AMA style:
W K. Lim, C. Gorodetsky, G. Ibrahim, K. Mithani, S. Breitbart, A. Leblanc-Millar, A. Fasano. Motor and Quality of Life Outcomes of Deep Brain Stimulation for Pediatric Dystonia – CHILD-DBS Registry [abstract]. Mov Disord. 2024; 39 (suppl 1). https://www.mdsabstracts.org/abstract/motor-and-quality-of-life-outcomes-of-deep-brain-stimulation-for-pediatric-dystonia-child-dbs-registry/. Accessed October 6, 2024.« Back to 2024 International Congress
MDS Abstracts - https://www.mdsabstracts.org/abstract/motor-and-quality-of-life-outcomes-of-deep-brain-stimulation-for-pediatric-dystonia-child-dbs-registry/