Objective: To evaluate motor and disability outcomes following GPi-DBS in a mixed cohort of primary and secondary dystonia patients treated from a tertiary referral center for movement disorders center.

Background: Globus pallidus interna deep brain stimulation (GPi-DBS) is an established and effective treatment for medically refractory dystonia. However, data regarding clinical outcomes in rare hereditary dystonias remain limited.

Method: Twenty-five patients with dystonia who underwent GPi-DBS were included in this retrospective observational study. Patients were aged 22-64 years and had diagnoses of primary or secondary dystonia. Hereditary dystonia subtypes included neuroacanthocytosis, pantothenate kinase-associated neurodegeneration (PKAN), DYT11, SCA6, and SCA14. Secondary dystonias included tardive dystonia, hemidystonia, and post-anoxic dystonia. Clinical response was assessed by comparing preoperative and postoperative Burke-Fahn-Marsden Dystonia Rating Scale motor (BFMDRS-M) and disability (BFMDRS-D) scores.

Results: Of the 25 patients, 15 (60%) were female and 10 (40%) were male. Mean age was 40.36 years (SD 9.34). Mean disease duration was 18.16 years (range 5-35), and mean disease duration before GPi-DBS was 5.4 years (range 1-9). Nineteen patients (76%) had primary dystonia and 6 (24%) had secondary dystonia; among those with primary dystonia, 9 (47.3%) had hereditary dystonia. Mean improvement was 67% in BFMDRS-M and 62% in BFMDRS-D. Clinical benefit was generally more pronounced in primary dystonia, whereas treatment response was weaker in secondary dystonia. A longer disease duration before GPi-DBS was associated with a lower clinical response.

Conclusion: GPi-DBS provides substantial motor and functional improvement in selected dystonia patients when performed in experienced centers. Treatment response appears more favorable in primary dystonia than in secondary dystonia, and delayed surgery may be associated with poorer clinical outcomes. Genetic characterization may also be important in predicting response, particularly in familial dystonia. Larger multicenter studies are needed to better define predictors of DBS outcomes in rare dystonia syndromes.

References: Rodrigues FB, Duarte GS, Prescott D, Ferreira J, Costa J. Deep brain stimulation for dystonia. Cochrane Database Syst Rev. 2019 Jan 10;1(1):CD012405. doi: 10.1002/14651858.CD012405.pub2. PMID: 30629283; PMCID: PMC6353249.
Tisch S. Deep brain stimulation in dystonia: factors contributing to variability in outcome in short and long term follow-up. Curr Opin Neurol. 2022 Aug 1;35(4):510-517. doi: 10.1097/WCO.0000000000001072. Epub 2022 Jul 5. PMID: 35787538.
Sarva H, Rodriguez-Porcel F, Rivera F, Gonzalez CD, Barkan S, Tripathi S, Gatto E, Ruiz PG; Rare Movement Disorders Study Group of the International Parkinsons and Movement Disorders Society. The role of genetics in the treatment of dystonia with deep brain stimulation: Systematic review and Meta-analysis. J Neurol Sci. 2024 Apr 15;459:122970. doi: 10.1016/j.jns.2024.122970. Epub 2024 Mar 20. PMID: 38520940.
Fan H, Zheng Z, Yin Z, Zhang J, Lu G. Deep Brain Stimulation Treating Dystonia: A Systematic Review of Targets, Body Distributions and Etiology Classifications. Front Hum Neurosci. 2021 Nov 26;15:757579. doi: 10.3389/fnhum.2021.757579. PMID: 34899219; PMCID: PMC8663760.
Beaulieu-Boire I, Aquino CC, Fasano A, Poon YY, Fallis M, Lang AE, Hodaie M, Kalia SK, Lozano A, Moro E. Deep Brain Stimulation in Rare Inherited Dystonias. Brain Stimul. 2016 Nov-Dec;9(6):905-910. doi: 10.1016/j.brs.2016.07.009. Epub 2016 Oct 4. PMID: 27743838.
Horn A, Reich MM, Ewert S, Li N, Al-Fatly B, Lange F, Roothans J, Oxenford S, Horn I, Paschen S, Runge J, Wodarg F, Witt K, Nickl RC, Wittstock M, Schneider GH, Mahlknecht P, Poewe W, Eisner W, Helmers AK, Matthies C, Krauss JK, Deuschl G, Volkmann J, Kühn AA. Optimal deep brain stimulation sites and networks for cervical vs. generalized dystonia. Proc Natl Acad Sci U S A. 2022 Apr 5;119(14):e2114985119. doi: 10.1073/pnas.2114985119. Epub 2022 Mar 31. PMID: 35357970; PMCID: PMC9168456.

« Back to 2026 International Congress

MDS Abstracts - https://www.mdsabstracts.org/abstract/clinical-outcomes-of-globus-pallidus-interna-deep-brain-stimulation-in-primary-and-secondary-dystonia-a-single-center-experience/