晚期帕金森病的临床试验
Clinical trials in late-stage Parkinson's disease.
文献信息
| PMID | 42770427 |
|---|---|
| 原文 | 在 PubMed 查看原文 ↗ |
| 发表日期 | 2026 |
| 作者 | Margherita Fabbri |
| 作者单位 | Department of Clinical Pharmacology and Neurosciences, Clinical Investigation Center CIC1436, Toulouse Parkinson Expert Centre, Toulouse NeuroToul Center of Excellence in Neurodegeneration, French NS-Park/F-CRIN Network, University of Toulouse, CHU of Toulouse, INSERM, Toulouse, France. |
| 期刊 | Journal of Parkinson's disease |
| SCI 分区 | Q1 |
| IF | 5.7 |
| 研究类型 | 综述 Meta · 临床 |
| 所属专科 | 耳科 |
中文摘要
背景： 晚期帕金森病（LSPD）是帕金森病一个未被充分认识和研究的阶段，其特征为严重的轴性运动症状、高非运动症状负担、认知障碍、功能依赖以及显著的照护者参与。尽管其具有重大的临床和社会经济影响，LSPD似乎在很大程度上被排除在临床试验之外，这可能是因为晚期残疾、活动能力受限以及缺乏适当调整的结果测量指标。
方法： 为了更好地描述该人群在研究中的代表性，我们对主要临床试验注册库中列出的临床试验进行了系统综述，重点关注纳入Hoehn和Yahr分期为4或5期参与者的研究。
结果： 截至2026年1月，有353项注册试验允许纳入LSPD个体；然而，其中许多研究应用了与痴呆相关的排除标准。大多数试验研究了针对轴性或整体运动症状的药物或康复干预措施。在已发表的研究中（占总数的27.8%），只有30%实际招募了LSPD参与者。
结论： 我们的发现证实了LSPD个体在临床研究中的纳入有限。放宽入选标准、实施去中心化或居家试验设计、纳入以照护者为中心的结局指标，以及采用认知适应性评估工具，可能有助于建立一个更具包容性和以患者为中心的研究框架。这些努力对于更好地满足这一不断增长且脆弱人群的需求至关重要。
英文摘要
Late-stage Parkinson's disease (LSPD) represents an under-recognized and under-studied phase of PD characterized by severe axial motor symptoms, high non-motor burden, cognitive impairment, functional dependence, and substantial caregiver involvement. Despite its major clinical and socioeconomic impact, LSPD seem to be largely excluded from clinical trials, likely due to advanced disability, mobility limitations, and the lack of appropriately adapted outcome measures.To better characterize the representation of this population in research, we conducted a systematic review of clinical trials listed in major clinical trials registries, focusing on studies that included participants with a Hoehn and Yahr stage of 4 or 5. As of January 2026, 353 registered trials allowed inclusion of individuals with LSPD; however, many of these studies applied exclusion criteria related to dementia. Most trials investigated pharmacological or rehabilitation interventions targeting axial or global motor symptoms. Among the published studies (27.8% of the total), only 30% actually enrolled participants with LSPD.Our findings confirm the limited inclusion of individuals with LSPD in clinical research. Broadening eligibility criteria, implementing decentralized or home-based trial designs, incorporating caregiver-centered outcomes, and adopting cognitively adapted assessment tools may foster a more inclusive and patient-centered research framework. Such efforts are essential to better address the needs of this growing and vulnerable population.