Objective: To evaluate the clinical outcomes of deep brain stimulation (DBS) in Malaysian children with pharmacoresistant dystonia.

Background: Dystonia is a common paediatric movement disorder which can be due to an underlying genetic mutation, or an acquired brain insult such as hypoxia, infection, or trauma. A proportion of the patients may develop drug-resistant dystonia despite optimal pharmacological treatment. DBS has emerged as a promising therapeutic option for pediatric pharmacoresistant dystonia. In February 2025, our center performed the country’s first pediatric DBS procedure and is currently the only pediatric DBS center in Malaysia.

Method: All children (<19 years) who underwent DBS for pharmacoresistant dystonia at our centre between February 2025 and February 2026 were included in this retrospective cohort study. Demographic and clinical data were obtained through a retrospective review of medical records. DBS outcomes were assessed by comparing pre- and post-DBS changes in the Burke–Fahn–Marsden Dystonia Rating Scale Movement (BFMDRS-M) and Disability (BFMDRS-D) subscales, the number of dystonia medications, and the frequency of status dystonicus episodes.

Results: Four patients (1 male, 3 females) with pharmacoresistant dystonia underwent DBS at a median age of 6.6 years (IQR 4.8). They were all diagnosed with genetic dystonia: GNAO1 (50%), PANK2 (25%), and SLC16A2 (25%). Two patients experienced frequent status dystonicus prior to DBS. The pre-DBS median BFMDRS-M and BFMDRS-D scores were 76.3 (IQR 9.3) and 30.0 (IQR 1.5). At 3 months post-DBS, the median BFMDRS-M and BFMDRS-D scores were 35.5 (IQR 19.5) and 30.0 (IQR 2.0). BFMDRS-M scores improved by 53.5% (p = 0.020), whereas BFMDRS-D scores and the number of dystonia medications remained unchanged. Median body weight increased by 8.2% (p = 0.098). No post-DBS complications were reported. All patients were free of status dystonicus after DBS.

Conclusion: DBS resulted in a marked reduction in dystonia severity in our cohort with significant improvement in BFMDRS-M scores at 3 months. The procedure was well tolerated with no reported complications. Although functional disability and medication burden remained unchanged in the short term, these findings highlight the potential of DBS as a safe and effective therapeutic option for severe pediatric dystonia. Larger studies with longer follow-up are needed to confirm its long-term functional benefits.

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MDS Abstracts - https://www.mdsabstracts.org/abstract/clinical-outcomes-of-deep-brain-stimulation-in-malaysian-children-with-pharmacoresistant-dystonia/