Deep brain stimulation in pediatric dystonia: a systematic review

Abstract

While deep brain stimulation (DBS) treatment is relatively rare in children, it may have a role in dystonia to reduce motor symptoms and disability. Pediatric DBS studies are sparse and limited by small sample size, and thus, outcomes are poorly understood. Thus, we performed a systematic review of the literature including studies of DBS for pediatric (age < 21) dystonia. Patient demographics, disease causes and characteristics, motor scores, and disability scores were recorded at baseline and at last post-operative follow-up. We identified 19 studies reporting DBS outcomes in 76 children with dystonia. Age at surgery was 13.8 ± 3.9 (mean ± SD) years, and 58% of individuals were male. Post-operative follow-up duration was 2.8 ± 2.8 years. Sixty-eight percent of patients had primary dystonia (PD), of whom 56% had a pathological mutation in DYT1 (DYT1+). Across all patients, regardless of dystonia type, 43.8 ± 36% improvement was seen in Burke-Fahn-Marsden Dystonia Rating Scale (BFMDRS) motor (-M) scores after DBS, while 43.7 ± 31% improvement was observed in BFMDRS disability (-D) scores. Patients with PD were more likely to experience ≥ 50% improvement (56%) in BFMDRS-M scores compared to patients with secondary causes of dystonia (21%, p = 0.004). DYT1+ patients were more likely to achieve ≥ 50% improvement (65%) in BFMDRS-D than DTY1- individuals (29%, p = 0.02), although there was no difference in BFMDRS-M ≥ 50% improvement rates between DYT1+ (66%) or DYT1- (43%) children (p = 0.11). While DBS is less common in pediatric patients, individuals with severe dystonia may receive worthwhile benefit with neuromodulation treatment.

Keywords: Deep brain stimulation; Dystonia; Functional neurosurgery; Pediatric neurosurgery; Pediatrics.

Fig. 1

PRISMA outline for systematic review

Fig. 2

Pediatric patients with primary dystonia (PD) were more likely to experience ≥ 50% improvement in Burke-Fahn-Marsden Dystonia Rating Scale motor component (BFMDRS-M) after DBS than patients with secondary causes of dystonia (p = 0.01, chi-square)

Fig. 3

Patients with primary generalized dystonia who carry a DYT1 mutation (DYT1+) were more likely to experience ≥ 50% improvement in BFMDRS-D scores compared to patients without the mutation (DYT1 −) (p = 0.02, chi-square)