Pallidal deep brain stimulation in patients with primary generalised or segmental dystonia: 5-year follow-up of a randomised trial

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• 作者：Prof Jens Volkmann ; MD^a ; ^b ; ^{volkmann_j@klinik.uni-wuerzburg.de} ; Alexander Wolters ; MD^c ; Prof Andreas Kupsch ; MD^d ; Prof Jö ; rg Mü ; ller ; MD^c ; ^e ; ^f ; Prof Andrea A Kü ; hn ; MD^d ; Gerd-Helge Schneider ; MD^g ; Prof Werner Poewe ; MD^e ; Sascha Hering ; MD^e ; Prof Wilhelm Eisner ; MD^h ; Jan-Uwe Mü ; ller ; MDⁱ ; Prof Gü ; nther Deuschl ; MD^a ; Marcus O Pinsker ; MD^j ; ^k ; Inger-Marie Skogseid ; MD^l ; Geir Ketil Roeste ; MD^m ; Prof Martin Krause ; MDⁿ ; ^o ; Prof Volker Tronnier ; MD^p ; ^q ; Prof Alfons Schnitzler ; MD^r ; Jü ; rgen Voges ; MD^s ; ^u ; Prof Guido Nikkhah ; MD^k ; Prof Jan Vesper ; MD^r ; Prof Joseph Classen ; MD^b ; ^t ; ^u ; Prof Markus Naumann ; MD^b ; ^v ; Prof Reiner Benecke ; MD^c ; for the DBS study group for dystonia ^&dagger ;
• 刊名：Lancet Neurology
• 出版年：2012
• 出版时间：December, 2012
• 年：2012
• 卷：11
• 期：12
• 页码：1029-1038
• 全文大小：278 K

文摘

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Summary

Background

Severe forms of primary dystonia are difficult to manage medically. We assessed the safety and efficacy of pallidal neurostimulation in patients with primary generalised or segmental dystonia prospectively followed up for 5 years in a controlled multicentre trial.

Methods

In the parent trial, 40 patients were randomly assigned to either sham neurostimulation or neurostimulation of the internal globus pallidus for a period of 3 months and thereafter all patients completed 6 months of active neurostimulation. 38 patients agreed to be followed up annually after the activation of neurostimulation, including assessments of dystonia severity, pain, disability, and quality of life. The primary endpoint of the 5-year follow-up study extension was the change in dystonia severity at 3 years and 5 years as assessed by open-label ratings of the Burke-Fahn-Marsden dystonia rating scale (BFMDRS) motor score compared with the preoperative baseline and the 6-month visit. The primary endpoint was analysed on an intention-to-treat basis. The original trial is registered with ().

Findings

An intention-to-treat analysis including all patients from the parent trial showed significant improvements in dystonia severity at 3 years and 5 years compared with baseline, which corresponded to ?20¡¤8 points (SD 17¡¤1; ?47¡¤9 % ; n=40) at 6 months; ?26¡¤5 points (19¡¤7; ?61¡¤1 % ; n=31) at 3 years; and ?25¡¤1 points (21¡¤3; ?57¡¤8 % ; n=32). The improvement from 6 months to 3 years (-5¡¤7 points [SD 8¡¤4]; ?34 % ) was significant and sustained at the 5-year follow-up (-4¡¤3 [10¡¤4]). 49 new adverse events occurred between 6 months and 5 years. Dysarthria and transient worsening of dystonia were the most common non-serious adverse events. 21 adverse events were rated serious and were almost exclusively device related. One patient attempted suicide shortly after the 6-month visit during a depressive episode. All serious adverse events resolved without permanent sequelae.

Interpretation

3 years and 5 years after surgery, pallidal neurostimulation continues to be an effective and relatively safe treatment option for patients with severe idiopathic dystonia. This long-term observation provides further evidence in favour of pallidal neurostimulation as a first-line treatment for patients with medically intractable, segmental, or generalised dystonia.

Funding

Medtronic.