Donofry Shannon D, O'Hanlon Claire E
Rand Health Q. 2024 Sep 25;11(4):8. eCollection 2024 Sep.
Emerging research suggests that Parkinson's disease (PD) and dementia with Lewy bodies (DLB) share underlying pathology and may represent a single, biologically defined disease spectrum. Cognitive changes are among the most worrisome symptoms for patients with PD, and are the core feature of DLB. While the cognitive changes experienced by individuals with PD and mild cognitive impairment share some clinical characteristics with patients who have undiagnosed or prodromal DLB, these changes are distinct from other types of dementias, such as Alzheimer's disease. To spur the adaptation of existing cognition-focused measures and the development of new ones to underlie clinical trial endpoints in PD and DLB, the PD/DLB Cognition Roundtable was held on January 10 and 11, 2024, in Washington, D.C. The roundtable brought together representatives from academia and industry, as well as with representatives of regulatory agencies, community partners, patient advocates, and research funders, to build consensus and collaborate on the outcome assessment and trial design methods that will support the development of new treatments for early or mild cognitive changes in disorders on the PD/DLB spectrum. The authors of this document summarize the roundtable, discussing the state of the field for clinical trial design and cognition measures in PD and DLB, promising avenues of research, and perspectives of regulatory agencies.
新出现的研究表明,帕金森病(PD)和路易体痴呆(DLB)具有共同的潜在病理,可能代表一种单一的、生物学定义的疾病谱。认知变化是帕金森病患者最令人担忧的症状之一,也是路易体痴呆的核心特征。虽然帕金森病患者和轻度认知障碍患者经历的认知变化与未诊断或前驱期路易体痴呆患者有一些共同的临床特征,但这些变化与其他类型的痴呆,如阿尔茨海默病不同。为了推动现有以认知为重点的测量方法的应用以及开发新的测量方法,以作为帕金森病和路易体痴呆临床试验终点的基础,帕金森病/路易体痴呆认知圆桌会议于2024年1月10日和11日在华盛顿特区举行。圆桌会议汇聚了学术界和产业界的代表,以及监管机构、社区合作伙伴、患者权益倡导者和研究资助者的代表,就结果评估和试验设计方法达成共识并开展合作,这些方法将支持开发针对帕金森病/路易体痴呆谱系疾病早期或轻度认知变化的新疗法。本文档的作者总结了圆桌会议,讨论了帕金森病和路易体痴呆临床试验设计和认知测量的领域现状、有前景的研究途径以及监管机构的观点。