Department of Neurology, University of Cologne, Faculty of Medicine and University Hospital Cologne, Cologne, Germany.
Department of Neurology and Neurosurgery, Salford Royal NHS Foundation Trust, Manchester Academic Health Science Centre, Manchester, Greater Manchester, UK.
J Neurol Neurosurg Psychiatry. 2024 Jun 17;95(7):630-638. doi: 10.1136/jnnp-2023-332272.
Subthalamic nucleus deep brain stimulation (STN-DBS) for Parkinson's disease (PD) improves quality of life (QoL), motor and non-motor symptoms (NMS). However, in previous studies, 43%-49% of patients did not experience clinically relevant postoperative QoL improvement. To inform individualised prediction of postoperative QoL improvement, we developed a stratification analysis of QoL outcomes based on preoperative non-motor total burden, severity of motor progression and motor response in levodopa challenge tests.
This was a prospective, open-label, multicentre, international study with a 6-month follow-up. A distribution-based threshold identified 'QoL responders' in the PDQuestionnaire-8 Summary Index (PDQ-8 SI). After baseline stratification based on the NMS Scale, Hoehn and Yahr Scale and levodopa response assessed with the Unified PD Rating Scale-III, we compared postoperative QoL response between these strata. To assess the clinical usefulness and statistical feasibility of stratifications, we compared cumulative distribution function curves, respectively PDQ-8 within-stratum variation.
All main outcomes improved postoperatively. Based on the 8.1 points threshold for clinically meaningful PDQ-8 SI improvement, only 80/161 patients were classified as 'QoL responders'. The absolute risk reductions for QoL non-response among respective non-motor, motor progression and levodopa response strata were 23%, 8% and 3%, respectively. Only non-motor stratification reduced PDQ-8 within-stratum variation compared with the overall cohort.
Non-motor stratification, but not motor progression or levodopa response stratification, is clinically useful and statistically feasible for personalised preoperative prediction of postoperative QoL outcome of STN-DBS for PD. Our findings highlight that non-motor assessments are necessary components of a case-based, holistic approach of DBS indication evaluations geared towards optimising postoperative QoL outcomes.
GermanClinicalTrialsRegister: #6735.
丘脑底核深部脑刺激（STN-DBS）治疗帕金森病（PD）可改善生活质量（QoL）、运动和非运动症状（NMS）。然而，在先前的研究中，43%-49%的患者术后 QoL 改善不明显。为了实现术后 QoL 改善的个体化预测，我们基于术前非运动总负担、运动进展严重程度和左旋多巴挑战试验中的运动反应，对 QoL 结局进行了分层分析。
这是一项前瞻性、开放性、多中心、国际研究，随访 6 个月。基于分布的阈值确定了 PDQuestionnaire-8 综合指数（PDQ-8 SI）中的“QoL 应答者”。在基于非运动量表、Hoehn 和 Yahr 量表以及用统一帕金森病评定量表-III 评估的左旋多巴反应进行基线分层后，我们比较了这些分层之间的术后 QoL 反应。为了评估分层的临床有用性和统计学可行性，我们比较了术后 QoL 应答者的累积分布函数曲线和 PDQ-8 分层内变异性。
所有主要结局均在术后改善。基于 PDQ-8 SI 改善 8.1 分的临床意义阈值，仅有 80/161 例患者被归类为“QoL 应答者”。在非运动、运动进展和左旋多巴反应分层中，QoL 无应答的绝对风险降低分别为 23%、8%和 3%。只有非运动分层与整体队列相比降低了 PDQ-8 分层内变异性。
非运动分层，而非运动进展或左旋多巴反应分层，对 STN-DBS 治疗 PD 术后 QoL 结局的个体化术前预测具有临床意义和统计学可行性。我们的研究结果强调，非运动评估是基于病例的、整体 DBS 适应证评估方法的必要组成部分，旨在优化术后 QoL 结局。
德国临床试验注册中心：#6735。
