Objective: The present study aimed to evaluate the clinical features and clinical outcomes in dystonia patients who underwent bilateral deep brain stimulation of the globus pallidus internus (GPi-DBS) to identify potential factors influencing treatment response.

Background: GPi-DBS is an effective treatment for medically refractory dystonia, improving motor symptoms and quality of life, though clinical outcomes and influencing factors remain debated.

Method: This study included 24 patients with at least 1.5 months of follow-up. Clinical improvement and quality of life (QoL) were assessed using the Global Dystonia Severity Rating Scale (GDS), Fahn–Marsden Dystonia Rating Scale (F-M), and EuroQoL 5-Dimension Questionnaire (EQ5D). Associations between postoperative changes in scores (ΔEQ5D, ΔGDS, ΔF-M) and demographic, clinical, and stimulation-related factors were analyzed.

Results: Twenty-four patients (9 female) with generalized (n=15), cervical (n=6), segmental (n=2), and oromandibular (n=1) dystonia were included. Mean ages at symptom onset and surgery were 24.1±19.6 and 36.3±18.4 years, with a mean disease duration of 12.2±10.4 years and follow-up of 5.78±4.31 years. Significant improvements were observed in all EQ5D subscales except ‘self-care’ (p=0.162) and in GDS and F-M scores except the ‘eye’ subscale (p>0.37). Male sex predicted greater ΔGDS improvement (β=13.53, p=0.010) while married patients showed greater improvement in ΔEQ5D (β = 18.45, p = 0.035). Older age at surgery was associated with worse ΔEQ5D outcomes (β = 0.491, p = 0.044). Higher left-sided pulse width correlated with greater ΔGDS-Trunk improvement (β=0.0384, p=0.023).

Conclusion: Our study corroborates that GPi-DBS improves clinical outcomes and QoL in dystonia patients with sustained long-term efficacy. Greater motor recovery in males and greater QoL improvement in married patients underscore the potential role of sex differences and social support in treatment response. The association between younger age at surgery and QoL gains indicates that earlier intervention may enhance outcomes. Additionally, correlations between PW and symptom-specific improvements highlight the influence of stimulation parameters on motor recovery. Our findings suggest that optimal GPi-DBS outcomes require a holistic approach, tailoring stimulation while accounting for patient-specific demographic and social factors.

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MDS Abstracts - https://www.mdsabstracts.org/abstract/long-term-efficacy-and-determinants-of-motor-and-quality-of-life-outcomes-following-bilateral-gpi-dbs-in-dystonia/