Nevler Naomi, Niehoff Debra, Gleixner Amanda M, Dodge Shana, Vogel Adam P, Hill Derek L, Ducharme Simon, Staffaroni Adam M, Yunusova Yana, Au Rhoda, Macwhinney Brian, Wong Bonnie, Parish-Morris Julia, Geronimo Andrew, Hansen Bryan J, Beattie Zachary T, Stephenson Diane T, Boxer Adam L, Dacks Penny, Rohrer Jonathan D
Frontotemporal Degeneration Center, Department of Neurology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.
Digital Science Lab, Abbvie Inc., North Chicago, Illinois, USA.
Alzheimers Dement. 2025 Apr;21(4):e70082. doi: 10.1002/alz.70082.
Frontotemporal degeneration (FTD) is a rare neurodegenerative disease in which patients can present with cognitive, behavioral, motor, and speech impairment. Currently, there are no approved therapies available to slow or halt disease progression. Detection and monitoring of patient symptoms is challenging for this heterogeneous disease and has negatively impacted progress in FTD clinical trials. Rapid technological advancements can promote the development of digital health technologies (DHTs) capable of capturing even the most subtle clinical impairments. DHTs are computing platforms being designed to measure meaningful aspects of disease onset and progression. Here we present some of the numerous tools currently being developed to measure changes in the functional domains that become impaired in FTD, challenges faced by developers, and a proposed roadmap for developing fit-for-purpose DHTs that will aid in the development of effective therapies for FTD. HIGHLIGHTS: DHTs are being developed to assess FTD onset and progression. Tool developers must overcome numerous challenges in creating effective applications. Guidance to tool developers aims to benefit FTD drug development and patient care.
额颞叶变性（FTD）是一种罕见的神经退行性疾病，患者可能会出现认知、行为、运动和言语障碍。目前，尚无获批的疗法可减缓或阻止疾病进展。对于这种异质性疾病，检测和监测患者症状具有挑战性，并且对FTD临床试验的进展产生了负面影响。快速的技术进步能够推动数字健康技术（DHT）的发展，这些技术甚至能够捕捉到最细微的临床损伤。DHT是旨在测量疾病发作和进展的有意义方面的计算平台。在此，我们介绍目前正在开发的众多工具中的一些，这些工具用于测量FTD中受损的功能领域的变化、开发者面临的挑战，以及开发适用于目的的DHT的拟议路线图，这将有助于开发针对FTD的有效疗法。要点：正在开发DHT以评估FTD的发作和进展。工具开发者在创建有效应用程序时必须克服众多挑战。针对工具开发者的指导旨在使FTD药物开发和患者护理受益。
