丘脑下核深部脑刺激治疗帕金森病的5年疗效

IF 21.3 1区医学 Q1 CLINICAL NEUROLOGY

JAMA neurology Pub Date : 2025-09-15 DOI:10.1001/jamaneurol.2025.3373

Philip A Starr,Rajat S Shivacharan,Edward Goldberg,Alexander I Tröster,Paul A House,Monique L Giroux,Adam O Hebb,Donald M Whiting,Timothy A Leichliter,Jill L Ostrem,Leo Verhagen Metman,Sepehr Sani,Jessica A Karl,Mustafa S Siddiqui,Stephen B Tatter,Ihtsham Ul Haq,Andre G Machado,Michal Gostkowski,Michele Tagliati,Adam N Mamelak,Michael S Okun,Kelly D Foote,Guillermo Moguel-Cobos,Francisco A Ponce,Rajesh Pahwa,Kelly Lyons,Cathrin M Buetefisch,Robert E Gross,Corneliu C Luca,Jonathan R Jagid,Gonzalo J Revuelta,Istvan Takacs,Michael H Pourfar,Alon Y Mogilner,Andrew P Duker,George T Mandybur,Joshua M Rosenow,Cindy Zadikoff,Suketu M Khandhar,Mark Sedrak,Fenna T Phibbs,Joseph Neimat,Jennifer Durphy,Adolfo Ramirez-Zamora,Julie G Pilitsis,Ryan J Uitti,Robert Wharen,Michael C Park,Jerrold L Vitek,

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Patients were implanted and followed up with the Vercise DBS system from May 2013 to December 2022. Eligibility required diagnosis of bilateral idiopathic PD with more than 5 years of motor symptoms, more than 6 hours per day of poor motor function, modified Hoehn and Yahr Scale scores higher than 2, Unified Parkinson's Disease Rating Scale (UPDRS-III) score of 30 or higher (medication-off state), and 33% or higher improvement in UPDRS-III medication-on score.\r\n\r\nIntervention\r\nBilateral STN-DBS for moderate to advanced PD.\r\n\r\nMain Outcomes and Measures\r\nPrimary outcomes included changes in UPDRS and dyskinesia scores, quality-of-life measures, and safety assessments. Exploratory analyses included medication reduction and DBS association with motor signs.\r\n\r\nResults\r\nA total of 313 patients were enrolled with 191 receiving the DBS system, and 137 participants (72%) completed the study. The study population had a mean (SD) age of 60 (7.9) years, with 139 (73%) male participants. Motor function without medication as measured by UPDRS-III improved from a mean (SD) of 42.8 (9.4) to 21.1 (10.6) at year 1 (51%; 95% CI, 49%-53%; P < .001) and 27.6 (11.6) at year 5 (36%; 95% CI, 33%-38%; P < .001). Activities of daily living without medication as measured by UPDRS-III improved from a mean (SD) of 20.6 (6.0) to 12.4 (6.1) at year 1 (41%; 95% CI, 38%-42%; P < .001) and 16.4 (6.5) at year 5 (22%; 95% CI, 18%-23%; P < .001). Dyskinesia scores decreased from 4.0 (5.1) to 1.0 (2.1) at year 1 (75%; 95% CI, 73%-75%; P < .001) and to 1.2 (2.1) at year 5 (70%; 95% CI, 63%-75%; P < .001). The levodopa equivalent dose was reduced by 28% at year 1, remaining stable at year 5 (28%; 95% CI, 26%-31%; P < .001). The most common serious adverse event was infection (9 participants). 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引用次数: 0

摘要

植入式神经刺激器治疗帕金森氏病（INTREPID）试验是一项随机、双盲、假对照的研究，目的是通过丘脑底核（STN）深部脑刺激（DBS）治疗帕金森氏病（PD）。目的评价STN-DBS治疗PD的长期（5年）疗效和安全性。设计、环境和参与者这是一项前瞻性、随机（3:1）、为期12周的双盲假对照研究，在美国23个运动障碍中心进行了为期5年的开放标签随访。患者于2013年5月至2022年12月植入并随访Vercise DBS系统。资格要求诊断为双侧特发PD，运动症状超过5年，每天运动功能不良超过6小时，改良Hoehn和Yahr量表评分高于2分，统一帕金森病评定量表（UPDRS-III）评分为30分或更高（停药状态），UPDRS-III用药评分改善33%或更高。干预：双侧STN-DBS治疗中晚期PD。主要结果和测量方法主要结果包括UPDRS和运动障碍评分的变化、生活质量测量和安全性评估。探索性分析包括药物减少和DBS与运动体征的关联。结果共纳入313例患者，其中191例接受了DBS系统，137例（72%）完成了研究。研究人群的平均（SD）年龄为60岁（7.9岁），其中139名（73%）男性参与者。UPDRS-III测量的无药运动功能在第1年从平均（SD） 42.8（9.4）改善到21.1 (10.6)(51%;95% CI, 49%-53%; P <。001)和27.6(11.6)在5年(36%;95%可信区间,33% -38%;P <措施)。UPDRS-III测量的无药日常生活活动在第1年从平均（SD） 20.6（6.0）改善到12.4 (6.1)(41%;95% CI, 38%-42%; P <。001)和16.4(6.5)在5年(22%;95%可信区间,18% -23%;P <措施)。运动障碍评分在第一年从4.0（5.1）下降到1.0 (2.1)(75%;95% CI, 73%-75%; P <。001)和1.2(2.1)在5年(70%;95%可信区间,63% -75%;P <措施)。左旋多巴等效剂量在第1年减少28%，在第5年保持稳定（28%;95% CI, 26%-31%; P < .001）。最常见的严重不良事件是感染（9名参与者）。报告了10例死亡，但都与该研究无关。结论和相关性尽管STN-DBS结果略有下降，可能是由于疾病的进行性，PD患者的运动和日常生活活动评分持续显着改善，同时抗帕金森药物治疗在5年随访期间稳定减少。

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Five-Year Outcomes from Deep Brain Stimulation of the Subthalamic Nucleus for Parkinson Disease.

Importance The Implantable Neurostimulator for the Treatment of Parkinson's Disease (INTREPID) trial was a randomized, double-blind, sham-controlled study of subthalamic nucleus (STN) deep brain stimulation (DBS) for the treatment of Parkinson disease (PD). Objective To evaluate the long-term (5-year) outcomes and safety of STN-DBS for PD. Design, Setting, and Participants This was a prospective, randomized (3:1), 12-week double-blind sham-controlled study at 23 movement disorder centers across the US with an open-label 5-year follow-up. Patients were implanted and followed up with the Vercise DBS system from May 2013 to December 2022. Eligibility required diagnosis of bilateral idiopathic PD with more than 5 years of motor symptoms, more than 6 hours per day of poor motor function, modified Hoehn and Yahr Scale scores higher than 2, Unified Parkinson's Disease Rating Scale (UPDRS-III) score of 30 or higher (medication-off state), and 33% or higher improvement in UPDRS-III medication-on score. Intervention Bilateral STN-DBS for moderate to advanced PD. Main Outcomes and Measures Primary outcomes included changes in UPDRS and dyskinesia scores, quality-of-life measures, and safety assessments. Exploratory analyses included medication reduction and DBS association with motor signs. Results A total of 313 patients were enrolled with 191 receiving the DBS system, and 137 participants (72%) completed the study. The study population had a mean (SD) age of 60 (7.9) years, with 139 (73%) male participants. Motor function without medication as measured by UPDRS-III improved from a mean (SD) of 42.8 (9.4) to 21.1 (10.6) at year 1 (51%; 95% CI, 49%-53%; P < .001) and 27.6 (11.6) at year 5 (36%; 95% CI, 33%-38%; P < .001). Activities of daily living without medication as measured by UPDRS-III improved from a mean (SD) of 20.6 (6.0) to 12.4 (6.1) at year 1 (41%; 95% CI, 38%-42%; P < .001) and 16.4 (6.5) at year 5 (22%; 95% CI, 18%-23%; P < .001). Dyskinesia scores decreased from 4.0 (5.1) to 1.0 (2.1) at year 1 (75%; 95% CI, 73%-75%; P < .001) and to 1.2 (2.1) at year 5 (70%; 95% CI, 63%-75%; P < .001). The levodopa equivalent dose was reduced by 28% at year 1, remaining stable at year 5 (28%; 95% CI, 26%-31%; P < .001). The most common serious adverse event was infection (9 participants). Ten deaths were reported, none related to the study. Conclusions and Relevance Although STN-DBS outcomes declined slightly, possibly due to the progressive nature of the disease, patients with PD sustained significant improvement in motor and activities of daily living scores, along with a stable reduction in anti-parkinsonian medication over the 5-year follow-up period.

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来源期刊

JAMA neurology CLINICAL NEUROLOGY-

CiteScore

41.90

自引率

1.70%

发文量

250

期刊介绍： JAMA Neurology is an international peer-reviewed journal for physicians caring for people with neurologic disorders and those interested in the structure and function of the normal and diseased nervous system. The Archives of Neurology & Psychiatry began publication in 1919 and, in 1959, became 2 separate journals: Archives of Neurology and Archives of General Psychiatry. In 2013, their names changed to JAMA Neurology and JAMA Psychiatry, respectively. JAMA Neurology is a member of the JAMA Network, a consortium of peer-reviewed, general medical and specialty publications.