Department of Neurology, Vanderbilt University Medical Center, Nashville, TN, USA.
Department of Neurology, Vanderbilt University Medical Center, Nashville, TN, USA.
Neuromodulation. 2023 Feb;26(2):451-458. doi: 10.1016/j.neurom.2022.10.051. Epub 2022 Dec 24.
The deep brain stimulation (DBS) in early-stage Parkinson's disease (PD) pilot clinical trial randomized 30 patients (Hoehn & Yahr II off; medication duration 0.5-4 years; without dyskinesia/motor fluctuations) to optimal drug therapy (ODT) (early ODT) or bilateral subthalamic nucleus (STN) DBS plus ODT (early DBS+ODT). This study aims to report the 11-year outcomes of patients who completed the DBS in early-stage PD pilot clinical trial.
Attempts were made to contact all 29 subjects who completed the two-year trial to participate in an 11-year follow-up study. Mixed-effects models compared overall trend in outcomes for randomization groups (fixed-effects: assigned treatment, year, their interaction; random-effect: subject) to account for repeated measures.
Twelve subjects participated in this 11-year follow-up study (n = 8 early ODT, n = 4 early DBS+ODT). Participating subjects were 70.0 ± 4.8 years old with a PD medication duration of 13.7 ± 1.7 years (early DBS duration 11.5 ± 1.3 years, n = 4). Three early ODT subjects received STN-DBS as standard of care (DBS duration 6.5 ± 2.0 years). Early ODT subjects had worse motor complications (Unified Parkinson's Disease Rating Scale [UPDRS]-IV) than early DBS+ODT subjects over the 11-year follow-up period (between-group difference = 3.5 points; p = 0.03). Early DBS+ODT was well-tolerated after 11 years and showed comparable outcomes to early ODT for other UPDRS domains, Parkinson Disease Questionnaire-39 (PDQ-39), and levodopa equivalent daily dose (LEDD).
Eleven years after randomization, early DBS+ODT subjects had fewer motor complications than early ODT subjects. These results should be interpreted with caution because only 40% of pilot trial subjects participated in this 11-year follow-up study. The Food and Drug Administration has approved the conduct of a pivotal clinical trial evaluating DBS in early-stage PD (IDEG050016).
The Clinicaltrials.gov registration number for the study is NCT00282152.
在早期帕金森病（PD）的临床试验中，深部脑刺激（DBS）随机将 30 名患者（Hoehn 和 Yahr II 期；药物治疗期 0.5-4 年；无运动障碍/运动波动）分为最佳药物治疗（ODT）（早期 ODT）或双侧丘脑底核（STN）DBS 加 ODT（早期 DBS+ODT）。本研究旨在报告完成早期 PD 临床试验的患者 11 年的结果。
试图联系所有完成两年试验的 29 名受试者，参与 11 年的随访研究。混合效应模型比较随机分组的总体趋势（固定效应：分配治疗、年、它们的相互作用；随机效应：受试者）以考虑重复测量。
12 名受试者参加了这项为期 11 年的随访研究（n=8 例早期 ODT，n=4 例早期 DBS+ODT）。参与研究的受试者年龄为 70.0±4.8 岁，PD 药物治疗时间为 13.7±1.7 年（早期 DBS 持续时间为 11.5±1.3 年，n=4）。3 名早期 ODT 受试者因标准治疗（DBS 持续时间 6.5±2.0 年）而接受 STN-DBS。在 11 年的随访期间，早期 ODT 受试者的运动并发症（统一帕金森病评定量表 [UPDRS]-IV）比早期 DBS+ODT 受试者更差（组间差异=3.5 分；p=0.03）。11 年后，早期 DBS+ODT 耐受性良好，在其他 UPDRS 领域、帕金森病问卷-39（PDQ-39）和左旋多巴等效日剂量（LEDD）方面与早期 ODT 结果相当。
在随机分组后 11 年，早期 DBS+ODT 受试者的运动并发症比早期 ODT 受试者少。由于只有 40%的试验受试者参加了这项 11 年的随访研究，因此应谨慎解释这些结果。食品和药物管理局已批准开展一项评估早期 PD 中 DBS 的关键临床试验（IDEG050016）。
该研究的 Clinicaltrials.gov 注册号为 NCT00282152。
