Prospective Long-term Follow-up of Focused Ultrasound Unilateral Subthalamotomy for Parkinson Disease
Prospective Long-term Follow-up of Focused Ultrasound Unilateral Subthalamotomy for Parkinson Disease
复制标题
聚焦超声单侧丘脑底核毁损术治疗帕金森病的前瞻性长期随访
DOI：
10.1212/wnl.0000000000206771
复制
发表时间：
2023-03-28
期刊：
影响因子：
9.9
通讯作者：
Obeso, Jose A.
中科院分区：
文献类型：
--
作者：
Martinez-Fernandez, Raul;Natera-Villalba, Elena;Obeso, Jose A.
背景和目的单侧磁共振引导聚焦超声丘脑切除术（FUS-STN）已被证明可以改善帕金森病（PD）的主要运动特征。这种效应是否持续尚不清楚。本研究旨在报告接受单侧 FUS-STN 治疗的 PD 患者的长期结果。方法我们对接受单侧 FUS-STN 治疗的不对称 PD 患者进行了一项前瞻性开放标签研究。所有患者在治疗后 36 个月内均接受评估。主要结局是在停药状态下接受治疗的半身运动障碍协会统一帕金森病评定量表 (MDS-UPDRS) 运动部分 (III) 评分与基线至 FUS-STN 后 36 个月的差异。安全性结果包括随访期间发生的所有不良事件。次要结果是用药后 MDS-UPDRS III 评分的变化；僵硬、运动迟缓、震颤和轴向特征的子评分；总 MDS-UPDRS III；以及 MDS-UPDRS 第四部分。分别使用 MDS-UPDRS II 和 PDQ39 评估功能障碍和生活质量。患者对改变的印象和对治疗的满意度进行自我评估。采用 Wilcoxon 符号秩检验和随后的 Bonferroni 校正进行数据分析。结果 32 例 PD 患者在治疗后 36 个月进行评估。基线时的平均 (+/- SD) 年龄为 56.0 +/- 10.1 岁，平均病程为 6.8 +/- 2.8 年。停药治疗后半身的 MDS-UPDRS III 评分从基线到 3 年提高了 52.3%（评分从 19.0 +/- 3.2 降低到 8.9 +/- 3.3，95% CI 8.7 到 11.6，p < 0.001），并且所有特定运动特征均较基线得到改善。没有报告致残或延迟的不良事件。 3 年时 MDS-UPDRS III 停药总评分比治疗前降低 22.9%（36.8 +/- 7.4 vs 27.4 +/- 6.2，95% CI 6.0 至 11.5，p < 0.001）。 MDS-UPDRS II、IV 和 PDQ39 评分以及左旋多巴剂量与基线相当。讨论单侧 FUS-STN 对 PD 运动特征的益处是长期持续的。经过数年的发展，FUS-STN 有助于更好的临床控制。 NCT02912871/03454425.证据分类本研究提供了关于聚焦超声单侧丘脑下切除术在治疗帕金森病患者中的效用的 IV 级证据。
Background and ObjectivesUnilateral magnetic resonance-guided focused ultrasound subthalamotomy (FUS-STN) has been shown to improve the cardinal motor features of Parkinson disease (PD). Whether this effect is sustained is not known. This study aims to report the long-term outcome of patients with PD treated with unilateral FUS-STN.MethodsWe conducted a prospective open-label study of patients with asymmetrical PD who underwent unilateral FUS-STN. All patients were evaluated up to 36 months after treatment. The primary outcome was the difference from baseline to 36 months after FUS-STN in the score of the Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) motor part (III) for the treated hemibody in the off-medication state. The safety outcome included all adverse events occurring during follow-up. Secondary outcomes were the change in the MDS-UPDRS III score on-medication; subscores of rigidity, bradykinesia, tremor, and axial features; total MDS-UPDRS III; and the MDS-UPDRS part IV. Functional disability and quality of life were assessed using the MDS-UPDRS II and the PDQ39, respectively. Patient impression of change and satisfaction with the treatment were self-assessed. The Wilcoxon signed-rank test with subsequent Bonferroni's correction was used for data analysis.ResultsThirty-two patients with PD were evaluated at 36 months after treatment. The mean (+/- SD) age at baseline was 56.0 +/- 10.1 years, with a mean disease duration of 6.8 +/- 2.8 years. The MDS-UPDRS III score for the treated hemibody off-medication was improved by 52.3% from baseline to 3 years (score reduction from 19.0 +/- 3.2 to 8.9 +/- 3.3, 95% CI 8.7 to 11.6, p < 0.001), and all specific motor features were improved from baseline. No disabling or delayed adverse events were reported. The total MDS-UPDRS III off-medication score was 22.9% lower at 3 years than before treatment (36.8 +/- 7.4 vs 27.4 +/- 6.2, 95% CI 6.0 to 11.5, p < 0.001). The MDS-UPDRS II, IV, and PDQ39 scores and levodopa dose were equivalent to those at baseline.DiscussionThe benefit of unilateral FUS-STN on PD motor features is sustained in the long term. FUS-STN contributes to better clinical control over several years of evolution. NCT02912871/03454425.Classification of EvidenceThis study provides Class IV evidence on the utility of focused ultrasound unilateral subthalamotomy in the treatment of people with Parkinson disease.