{
  "abstract": "Background The safety and effectiveness of deep brain stimulation of the subthalamic nucleus (STN-DBS) for the treatment of dystonia lack high-level evidence-based medical support. This study aimed to clarify the efficacy and safety of STN-DBS and perform a post hoc analysis comparing it with DBS of the internal globus pallidus (GPi-DBS).Methods This multicentre, randomised, double-blind, controlled trial included 67 patients aged 6–60 years old diagnosed with genetic or idiopathic isolated generalised or segmental dystonia. They were enrolled from seven hospitals in China and randomly assigned to undergo GPi-DBS or STN-DBS. After surgery, they were randomised to receive either neurostimulation or sham stimulation for 3 months. At the 3-month follow-up, neurostimulation was also initiated in the sham stimulation group, and all patients were followed up for more than 3 years after treatment. The primary outcome was the Burke-Fahn-Marsden Dystonia Rating Scale movement (BFMDRS-M) score.Results In the STN group, the neurostimulation subgroup exhibited significant improvement (p<0.001), which is also superior to the sham stimulation subgroup (p=0.028) at 3-month follow-up. At the 6-month and >3-year follow-ups, all patients receiving STN-DBS showed a significant improvement in BFMDRS-M scores (p<0.001). Further post hoc analysis revealed that both STN-DBS and GPi-DBS could produce similar therapeutic effects on motor symptoms (P 6 months=0.865, P>3 years=0.905). There were no ongoing serious adverse events throughout the study.Conclusions For isolated generalised and segmental dystonia patients, the STN is a selectable DBS target with ensured safety and efficacy. STN-DBS and GPi-DBS may achieve comparable therapeutic effects on motor symptoms.Trial registration number NCT03017586.",
  "authors": [
    {
      "affiliations": [
        "Department of Neurology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences, Peking Union Medical College, Beijing, China"
      ],
      "name": "Lin Wang"
    },
    {
      "affiliations": [
        "Department of Neurology, Laboratory of Neurodegenerative Disorders, West China Hospital, Sichuan University, Chengdu, Sichuan, China"
      ],
      "name": "Huifang Shang"
    },
    {
      "affiliations": [
        "Neurotoxin research center of Key Laboratory of Spine and Spinal Cord Injury Repair and Regeneration of Ministry of Education, Department of Neurology, Tongji Hospital Affiliated to Tongji University, Shanghai, China"
      ],
      "name": "Lingjing Jin"
    },
    {
      "affiliations": [
        "Department of Neurology, Union Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, Hubei, China"
      ],
      "name": "Nian Xiong"
    },
    {
      "affiliations": [
        "Department of Neurology, Sir Run Run Shaw Hospital,School of Medicine, Zhejiang University, Hangzhou, Zhejiang, China"
      ],
      "name": "Xingyue Hu"
    },
    {
      "affiliations": [
        "Department of Neurosurgery, West China Hospital of Sichuan University, Chengdu, Sichuan, China"
      ],
      "name": "Wei Wang"
    },
    {
      "affiliations": [
        "Department of Neurology, Qilu Hospital of Shandong University, Jinan, Shandong, China"
      ],
      "name": "Yiming Liu"
    },
    {
      "affiliations": [
        "Department of Geriatric Neurology, Nanjing Medical University Affiliated Brain Hospital, Nanjing, Jiangsu, China"
      ],
      "name": "Jun Yan"
    },
    {
      "affiliations": [
        "Clinical Research Institute, Institute of Advanced Clinical Medicine, Peking University, Beijing, China"
      ],
      "name": "Lingling Gao"
    },
    {
      "affiliations": [
        "Department of Neurosurgery, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences, Peking Union Medical College, Beijing, China"
      ],
      "name": "Yaning Wang"
    },
    {
      "affiliations": [
        "Department of Neurology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences, Peking Union Medical College, Beijing, China"
      ],
      "name": "Yanying Wang"
    },
    {
      "affiliations": [
        "Department of Neurosurgery, Union Hospital, Tongji Medical College of Huazhong University of Science and Technology, Wuhan, Hubei, China"
      ],
      "name": "Peng Fu"
    },
    {
      "affiliations": [
        "Department of Neurology, Sir Run Run Shaw Hospital,School of Medicine, Zhejiang University, Hangzhou, Zhejiang, China"
      ],
      "name": "Huaying Cai"
    },
    {
      "affiliations": [
        "Department of Functional Neurosurgery, Nanjing Medical University Affiliated Brain Hospital, Nanjing, Jiangsu, China"
      ],
      "name": "Wenbin Zhang"
    },
    {
      "affiliations": [
        "Department of Neurosurgery, Shandong University Qilu Hospital, Jinan, Shandong, China"
      ],
      "name": "Shujun Xu"
    },
    {
      "affiliations": [
        "Neurotoxin research center of Key Laboratory of Spine and Spinal Cord Injury Repair and Regeneration of Ministry of Education, Department of Neurology, Tongji Hospital Affiliated to Tongji University, Shanghai, China"
      ],
      "name": "Fei Teng"
    },
    {
      "affiliations": [
        "Department of Neurology, Laboratory of Neurodegenerative Disorders, West China Hospital, Sichuan University, Chengdu, Sichuan, China"
      ],
      "name": "Ruwei Ou"
    },
    {
      "affiliations": [
        "Department of Neurology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences, Peking Union Medical College, Beijing, China"
      ],
      "name": "Lei Qiao"
    },
    {
      "affiliations": [
        "Department of Neurology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences, Peking Union Medical College, Beijing, China"
      ],
      "name": "Yingmai Yang"
    },
    {
      "affiliations": [
        "Department of Neurology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences, Peking Union Medical College, Beijing, China"
      ],
      "name": "Mengyu Zhang"
    },
    {
      "affiliations": [
        "Department of Neurosurgery, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences, Peking Union Medical College, Beijing, China"
      ],
      "name": "Yi Guo"
    },
    {
      "affiliations": [
        "Department of Neurology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences, Peking Union Medical College, Beijing, China"
      ],
      "name": "Xinhua Wan"
    }
  ],
  "title": "Subthalamic deep brain stimulation in isolated generalised or segmental dystonia (RELAX Study): a multicentre, randomised, double-blind, controlled trial",
  "uid": "0944a1e2-e766-52c1-afe4-ba021aa11b0b"
}