Category: Dystonia: Clinical Trials and Therapy
Objective: To compare residual symptoms and disease-specific quality of life (DS-QoL) in CD patients treated with DBS, or BoNT injections alone, for minimum 3 years.
Background: Standard BoNT therapy has shown good efficacy and improved DS-QoL in CD, also in the long-term. However, unsatisfactory outcome is observed in 1/5 to 1/3 [1-3]. DBS of the internal globus pallidus (GPi-DBS) is documented as effective for severe CD in the short term , but documentation beyond 3 years is sparse.
Method: We included isolated CD patients (focal or segmental) treated for minimum 3 years at our hospital with 1)Bilateral GPi-DBS(DBS-group, op.2004-2017), or 2)Regular BoNT-injections (BoNT-group). Primary endpoint was TWSTRS total score at long-term evaluation of the DBS-group compared to the BoNT-group at peak efficacy (4-6 weeks after last injection,V2). The BoNT-group was also evaluated before last injection (V1). Secondary endpoints: Comparison of TWSTRS Severity, Disability and Pain, Cervical Dystonia Impact Profile-58 (CDIP-58) Total scores. Exploratory endpoints: CDIP-58 subscores.
Results: We enrolled 41 patients, 20 DBS-treated/21 BoNT-treated, focal CD in 45/95 %, females 60%/95%, median age at onset/at evaluation 42/37 and 64/65, respectively. After median(range) 7 (4-18) years of DBS and 16 (4-32) of BoNT treatment, primary and secondary endpoints showed similarly low (good) scores in both groups (DBS/BoNT V2): Median TWSTRS total score 15/17, Severity score 7/6, Disability score 5/5, Pain 1/6, CDIP-58 Total score 43/38 (P .16 to .91). In the DBS-group, improvement from pre-operative TWSTRS Total/Severity scores of 56/22 were 73%/68% (both p=<.001). In the BoNT-group, TWSTRS Total and Severity score improved significantly V1 to V2(both p=<.001) When comparing DBS with BoNT at V1, there was a trend towards worse median TWSTRS Total and Severity score (15/11, p=.08/.06), and significantly higher (worse) scores in the CIDP-58 subscore head-neck symptoms (P=.003) at V1. CDIP-58 walking subscore was clearly worse in the DBS group (P=.007,P=.006).
Conclusion: This ultra long-term evaluation showed a similarly low symptom burden/good DS-QoL in the DBS-group when compared with BoNT at peak efficacy. But a higher symptom burden before BoNT injection, confirm the less stable (Yo-yo) effect many BoNT-treated patients experience.
References: 1. Haussermann P, Marczoch S, Klinger C, Landgrebe M, Conrad B, Ceballos-Baumann A. Long-term follow-up of cervical dystonia patients treated with botulinum toxin A. Movement Disorders 2004; 19: 303-308.
2. Skogseid IM, Kerty E. The course of cervical dystonia and patient satisfaction with long-term botulinum toxin A treatment. European Journal of Neurology 2005, 12: 163-170.
3. Colosimo C, Charles D, Misra VP, Maisonobe P, Om S ; INTEREST IN CD2 study group. How satisfied are cervical dystonia patients after 3 years of botulinum toxin type A treatment? Results from a prospective, long-term observational study. J Neurol 2019 Dec; 266(12):3038-46. doi: 10.1007/s00415-019-09527-2. Epub 2019 Sep 9.
4. Volkmann J, Mueller J, Deuschl G, Kühn AA, Krauss JK, Poewe W, Timmermann L, Falk D, Kupsch A, Kivi A, Schneider G-H, Schnitzler A, Südmeyer M, Voges J, Wolters A, Wittstock M, Müller J-U, Hering S, Eisner W, Vesper J, Prokop T, Pinsker M, Schrader C, Kloss M, Kiening K, Boetzel K, Mehrkens J, Skogseid IM, Ramm-Pettersen J, Kemmler G, Bhatia KP, Vitek JL, Benecke R, for the DBS study group for dystonia*. Pallidal neurostimulation in patients with medication-refractory cervical dystonia: a randomised, sham-controlled trial. Lancet Neurol. 2014 Sep;13(9):875-84. doi: 10.1016/S1474-4422(14)70143-7. Epub 2014 Aug 7.
To cite this abstract in AMA style:IM. Wedding, V. Siewers, IM. Skogseid. Ultra long-term outcome of deep brain stimulation (DBS) compared to botulinum toxin (BoNT) treatment in cervical dystonia (CD) [abstract]. Mov Disord. 2023; 38 (suppl 1). https://www.mdsabstracts.org/abstract/ultra-long-term-outcome-of-deep-brain-stimulation-dbs-compared-to-botulinum-toxin-bont-treatment-in-cervical-dystonia-cd/. Accessed September 25, 2023.
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