Mestre Tiago A, Stebbins Glenn T, Stephenson Diane, Dexter David, Lee Karen K, Xiao Yuge, Dam Tien, Kopil Catherine M, Simuni Tanya
Ottawa Hospital Research Institute; University of Ottawa Brain and Mind Research Institute; Division of Neurology, Department of Medicine, University of Ottawa, The Ottawa Hospital Ottawa, Ottawa, ON, Canada.
Department of Neurological Sciences, Rush University Medical Center, Chicago, IL, USA.
NPJ Parkinsons Dis. 2024 May 14;10(1):101. doi: 10.1038/s41531-024-00716-z.
Novel therapies with the ability to delay disease progression are a gap in the care of people living with Parkinson disease (PD) today. Clinical outcomes assessments (COAs) that are sensitive to the earliest clinical changes in PD are deemed essential for a successful therapeutic development. To understand the current landscape of COAs use in clinical trials in PD and define priorities for future research in the field, a stakeholder roundtable meeting was held in November 2022. The current paper 1) proposes the collaborative development of patient-centric COAs that can adequately document the effectiveness of disease modification therapies in PD based on key priorities identified during this initial meeting, 2) summarizes the progress made in the subsequent 12 months, and 3) presents the deliverables expected in the near future. Key priorities include 1) the development of a consensus conceptual model of early PD experiences, 2) the adaptation of existing patient-reported outcomes (PROs), 3) the investigation of the role of observer-reported outcomes in addition to 4) enabling diversity in PD research and advocacy, 5) fostering data sharing, and 6) reaching consensus on a biological staging system for PD to drive the development of appropriate PROs for biologically defined populations.
目前，能够延缓疾病进展的新型疗法在帕金森病（PD）患者护理方面尚属空白。对PD最早临床变化敏感的临床结局评估（COA）被认为是成功进行治疗研发的关键。为了解COA在PD临床试验中的应用现状，并确定该领域未来研究的重点，于2022年11月召开了一次利益相关者圆桌会议。本文1）基于首次会议确定的关键重点，提议以患者为中心的COA进行协作开发，以充分记录PD疾病修饰疗法的有效性；2）总结了随后12个月取得的进展；3）介绍了近期预期的可交付成果。关键重点包括：1）建立早期PD体验的共识概念模型；2）改编现有的患者报告结局（PRO）；3）研究观察者报告结局的作用；4）促进PD研究和宣传的多样性；5）推动数据共享；6）就PD的生物学分期系统达成共识，以推动为生物学定义的人群开发合适的PRO。
