Objective: To investigate the role of sex as a biological variable in efficacy and safety of GPi DBS in adults with dystonia.

Background: Sex- related differences in epidemiology, clinical features and treatment response have been described in various neurological diseases. To date, few data are available on sex differences in patients affected by dystonia, and especially information concerning differences in Deep Brain Stimulation (DBS) treatment outcome is lacking.

Method: A double-center retrospective study on patients who underwent GPi DBS for dystonia treatment, followed-up for at least 12 months, was performed. Baseline pre-operative and follow-up data collected included demographic features, disease information, DBS target, stimulation parameters, botulinum treatment. Disease severity was assessed by the Burke-Fahn-Marsden Rating Scale (BFMRS) – Motor and Disability scores. Annual follow-up data from surgery to last visit were recorded. Adverse events were recorded.

Results: 21 patients (14 males/7 females) were excluded due to incomplete data. Sixty-two consecutive patients (24 M/38 F) were enrolled: 32 had idiopathic dystonia (13M/19F), 13 inherited dystonia (4M/9F) and 17 acquired dystonia. Higher age at onset and age at implant was detected in females. No inter-sex difference was detected in BFMDRS motor (mean 45.8±23.3) or disability (14.9±7.4) sub-scales at baseline or in scores’ variation at FUs; disease duration at implant (mean 21.6±12.7 Y) and FU duration (mean 7.2±4.0 years) were comparable among the sexes. Disease duration at implant correlated to basal BFMDRS-M score only in female cohort. Improvement in dystonia severity was comparable in both groups: males improved by 56.5% at 1 year and by 54.1% at last FU (8.2±3.5 years) while females improved by 55.1% at 1 year and by 49.3% at last FU (6.5±4.3 years). A further stratification according to age at dystonia onset, disease duration at implant or dystonia etiology, disability improvement, energy delivered or persistent adverse events, resulted comparable between sexes.

Conclusion: This long-term study suggests that sex may not be a major determinant of efficacy and safety in dystonia patients treated with DBS, indicating equal outcome in women and men. These findings contribute to the debate on the indications for DBS in dystonia syndromes.

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MDS Abstracts - https://www.mdsabstracts.org/abstract/sex-is-not-a-major-determinant-of-pallidus-internus-deep-brain-stimulation-outcome-in-dystonia/