Data Sheet 1_Effectiveness and safety of magnetic resonance–guided unilateral focused ultrasound subthalamotomy for Parkinson’s disease: a systematic review and meta-analysis of prospective studies.docx

BackgroundPatients with asymmetric, medication-refractory Parkinson’s disease (PD) often continue to experience disabling motor symptoms despite optimized pharmacological management. Magnetic resonance–guided focused ultrasound subthalamotomy (FUS-STN) has recently emerged as a promising, non-invasive alternative for improving motor function. However, its overall clinical efficacy and long-term safety remain the subject of active investigation.
MethodsWe systematically searched PubMed, Cochrane Library, Embase, Web of Science, and ClinicalTrials.gov from their inception to 30 November 2024. Prospective studies that assessed unilateral FUS-STN in patients with PD were included. Data were pooled using RevMan 5.3 for mean differences (MD) with 95% confidence intervals (CIs).
ResultsFour prospective studies (n = 69) were included. Unilateral FUS-STN significantly reduced the Movement Disorders Society-Unified Parkinson’s Disease Rating Scale (MDS-UPDRS) Part III scores for the treated hemibody in both the off-medication [MD = −11.01, 95% CI (−12.23, −9.80), p < 0.001] and on-medication states [MD = −6.51, 95% CI (−7.57, −5.42), p < 0.001]. The MDS-UPDRS II (MD = −3.05, p < 0.01) and 39-item Parkinson’s disease questionnaire summary index (PDQ-39SI) scores (MD = −6.99, p < 0.01) also improved. Levodopa equivalent daily dose (LEDD) was reduced in the short term (MD = −149.5 mg, p < 0.001), although it was attenuated at 12 months (p = 0.09). No significant improvement was observed in MDS-UPDRS IV scores (MD = −3.29, p = 0.64). In all included studies, adverse events (AEs) were frequent during and after the procedure, such as postoperative gait and speech disturbance, facial asymmetry, and dyskinesia. However, the majority of AEs were resolved during the 6–12 month follow-up period.
ConclusionUnilateral FUS-STN may offer symptomatic benefits and a general safety profile in selected patients with asymmetric PD. Future investigations should emphasize large-scale, longitudinal, multicenter, and symptom-specific randomized controlled trials to assess the long-term benefits and risks of unilateral FUS-STN in PD patients.
Systematic review registrationhttps://www.crd.york.ac.uk/PROSPERO/view/CRD420251002754, identifier PROSPERO (CRD420251002754).

背景：伴不对称性表现的药物难治性帕金森病（Parkinson’s disease, PD）患者，即便经优化药物治疗后，仍常持续出现致残性运动症状。磁共振引导聚焦超声丘脑底核毁损术（Magnetic resonance–guided focused ultrasound subthalamotomy, FUS-STN）近年来作为改善运动功能的极具前景的非侵入性替代疗法崭露头角，但其整体临床疗效与长期安全性仍为当前积极探索的研究课题。
方法：本研究系统检索了PubMed、Cochrane图书馆、Embase、Web of Science及ClinicalTrials.gov数据库自建库至2024年11月30日的相关文献，纳入评估单侧FUS-STN治疗PD患者的前瞻性研究。采用RevMan 5.3软件对数据进行合并分析，以均数差（mean difference, MD）及95%置信区间（confidence interval, CI）作为效应量。
结果：最终纳入4项前瞻性研究，共69例患者。单侧FUS-STN可显著改善治疗侧肢体的运动障碍学会统一帕金森病评定量表（Movement Disorders Society-Unified Parkinson’s Disease Rating Scale, MDS-UPDRS）第三部分评分，在停药状态下[MD = −11.01, 95% CI (−12.23, −9.80), p < 0.001]与服药状态下[MD = −6.51, 95% CI (−7.57, −5.42), p < 0.001]均有显著降低。此外，MDS-UPDRS第二部分评分[MD = −3.05, p < 0.01]与39项帕金森病问卷综合指数（39-item Parkinson’s disease questionnaire summary index, PDQ-39SI）[MD = −6.99, p < 0.01]亦得到改善。左旋多巴等效日剂量（Levodopa equivalent daily dose, LEDD）在短期随访中显著降低[MD = −149.5 mg, p < 0.001]，但在12个月随访时这种获益有所减弱（p = 0.09）。MDS-UPDRS第四部分评分未观察到显著改善[MD = −3.29, p = 0.64]。纳入的所有研究均显示，术中及术后常见不良事件（adverse events, AEs）包括术后步态与言语障碍、面部不对称及异动症，但多数不良事件可在6~12个月的随访期内缓解。
结论：针对经筛选的不对称性PD患者，单侧FUS-STN可带来症状获益，且整体安全性良好。未来研究应着重开展大规模、纵向、多中心且针对特定症状的随机对照试验，以评估单侧FUS-STN治疗PD患者的长期获益与风险。
系统评价注册信息：本系统评价已在PROSPERO平台注册，注册编号为CRD420251002754，注册链接：https://www.crd.york.ac.uk/PROSPERO/view/CRD420251002754。