Department of Neurology, Amsterdam UMC, University of Amsterdam, Meibergdreef 9, Amsterdam, the Netherlands.
Clinical Research Unit, Amsterdam UMC, University of Amsterdam, Meibergdreef 9, Amsterdam, the Netherlands.
BMC Neurol. 2020 Jan 31;20(1):40. doi: 10.1186/s12883-020-1621-y.
Both Deep Brain Stimulation (DBS) and Continuous intrajejunal Levodopa Infusion (CLI) are effective therapies for the treatment of Parkinson's disease (PD). To our knowledge, no direct head-to-head comparison of DBS and CLI has been performed, whilst the costs probably differ significantly. In the INfusion VErsus STimulation (INVEST) study, costs and effectiveness of DBS and CLI are compared in a randomized controlled trial (RCT) in patients with PD, to study whether higher costs of one of the therapies are justified by superiority of that treatment.
A prospective open label multicentre RCT is being performed, with ancillary patient preference observational arms. Patients with PD who, despite optimal pharmacological treatment, have severe response fluctuations, bradykinesia, dyskinesias, or painful dystonia are eligible for inclusion. A total of 66 patients will be randomized. There is no minimal inclusion in the patient preference arms. The primary health economic outcomes are costs per unit on the Parkinson's Disease Questionnaire-39 (PDQ-39) and costs per unit Quality-Adjusted Life Year (QALY) at 12 months. The main clinical outcome is patient-reported quality of life measured with the PDQ-39 at 12 months. Patients will additionally be followed during 36 months after initiation of the study treatment.
The INVEST trial directly compares the costs and effectiveness of the advanced therapies DBS and CLI.
Dutch Trial Register identifier 4753, registered November 3rd, 2014; EudraCT number 2014-001501-32, Clinicaltrials.gov: NCT02480803.
深部脑刺激（DBS）和连续肠内左旋多巴输注（CLI）都是治疗帕金森病（PD）的有效疗法。据我们所知，尚未对 DBS 和 CLI 进行直接的头对头比较，而成本可能存在显著差异。在 INfusion VErsus STimulation（INVEST）研究中，我们在一项 PD 患者的随机对照试验（RCT）中比较了 DBS 和 CLI 的成本和效果，以研究一种疗法的成本较高是否因其治疗效果更优而具有合理性。
正在进行一项前瞻性开放标签多中心 RCT，并设有患者偏好观察臂。纳入标准为尽管接受了最佳药物治疗，但仍存在严重的运动波动、运动迟缓、运动障碍或疼痛性肌张力障碍的 PD 患者。总共将纳入 66 名患者。在患者偏好臂中没有最低纳入标准。主要卫生经济学结局为帕金森病问卷-39（PDQ-39）单位成本和 12 个月时质量调整生命年（QALY）的单位成本。主要临床结局为 12 个月时患者报告的生活质量，采用 PDQ-39 进行测量。在研究治疗开始后 36 个月内，还将对患者进行随访。
INVEST 试验直接比较了 DBS 和 CLI 这两种先进疗法的成本和效果。
荷兰试验注册中心标识符为 4753，于 2014 年 11 月 3 日注册；EudraCT 编号为 2014-001501-32，Clinicaltrials.gov：NCT02480803。
