Communication Bridge: Optimizing an evidence-based intervention for individuals with primary progressive aphasia

沟通桥梁：优化针对原发性进行性失语症患者的循证干预措施

• 批准号: 10656037
• 负责人: EMILY J ROGALSKI
• 金额: --
• 依托单位: NORTHWESTERN UNIVERSITY AT CHICAGO
• 依托单位国家: 美国
• 财政年份: 2017
• 资助国家: 美国
• 起止时间: 2017-09-15 至 2023-09-02
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10656037
• 关键词: Address; Alzheimer' s Disease; Aphasia; CNR1 gene; CNR2 gene; Caring; Clinical; Clinical Trials; Communication; Communication impairment; Communities; Complement; Complex; Cost Effectiveness Analysis; Counseling; Custom; Data; Dementia; Development; Disease; Economic Burden; Education; Evaluation; Evidence based intervention; Family; Focus Groups; Frontotemporal Lobar Degenerations; Future; Guidelines; Health; Individual; Intervention; Interview; Language; Life; Measurement; Measures; Modeling; Neurodegenerative Disorders; Outcome; Participant; Patients; Persons; Phase; Physical activity; Pragmatic clinical trial; Primary Progressive Aphasia; Probability; Quality of life; Randomized, Controlled Trials; Readiness; Social Interaction; Speech; Syndrome; Testing; Theoretical model; Training; Work; active control; arm; care giving burden; clinical care; clinical practice; cost; cost effectiveness; efficacy evaluation; experimental arm; functional outcomes; health economics; inclusion criteria; innovation; intervention cost; language impairment; neurodegenerative dementia; neuropathology; novel; personalized medicine; post intervention; pragmatic trial; predicting response; primary outcome; psychosocial; remote monitoring; response; skills; social engagement; success; tool; translational potential; trial design; trial readiness; videoconference; wearable sensor technology; web app

PROJECT SUMMARY/ABSTRACT Language and communication impairments (aphasia) are the defining features of the clinical neurodegenerative dementia syndrome primary progressive aphasia (PPA), which can be caused by Alzheimer’s disease (AD) or frontotemporal lobar degeneration. The devastation of communication negatively impacts quality of life for persons living with PPA and their care partners. Communication difficulties in PPA can also reduce life-space mobility including shrinking social interactions and lowering participation in community activities. Nonpharmacological interventions, including speech-language and psychosocial intervention, may offer significant benefit to the quality of life for persons with PPA and their care partners, but have historically lacked efficacy evidence as well as guidelines to direct best clinical care practices. We have been changing this landscape, through the development of the Communication Bridge (CB) intervention with strategically staged, rigorous clinical trials that are on a path towards implementation. The CB1 trial established feasibility of delivering our intervention at a global scale via telepractice and demonstrated gains in functional communication outcomes that were maintained 6-months post baseline. Next, the CB2 trial provided the first rigorous Phase 2a, Stage II, superiority, randomized controlled trial (RCT) of speech-language intervention in PPA. CB2 interim analysis conditional power results suggest a high probability of trial success (>80%) but also identified a need for greater psychosocial education and counseling. Thus, the current application proposes the CB3 trial designed to optimize the intervention by testing whether the addition of psychosocial education and counselling (PEC) to speech-language intervention (Experimental arm) is superior to an active Control intervention. The RCT will be complemented by Aim 1a, which will maximize pragmatic trial readiness following the Readiness Assessment for Pragmatic Trials (RAPT) tool; and Aim 2, which will assess functional outcomes with novel wearable sensors to objectively measure life participation using a ‘Life-Space Mobility’ framework. Trial outcomes will inform future pragmatic trial design. The translational potential is high since the delivery model and primary outcomes are aligned with current clinical practice. In the absence of a definitive cure for AD and related neurodegenerative diseases, it is important to identify and evaluate strategies that help individuals maximize their quality of life, and this project will help fulfill this need.

项目总结/摘要 语言和交流障碍（失语症）是临床神经退行性疾病的定义特征。 痴呆综合征原发性进行性失语症（PPA），可由阿尔茨海默病（AD）或 额颞叶变性通信的破坏对生活质量产生了负面影响， 患有PPA的人及其护理伴侣。PPA中的沟通困难也会减少生活空间 流动性，包括减少社会互动和降低参与社群活动。 非药物干预，包括言语和心理社会干预，可能会提供 显著改善PPA患者及其护理伙伴的生活质量，但历史上缺乏 疗效证据以及指导最佳临床护理实践的指南。我们一直在改变这一点 景观，通过发展的沟通桥梁（CB）的干预与战略阶段， 严格的临床试验正在走向实施。CB 1试验确定了交付的可行性 我们通过远程实践在全球范围内进行干预，并在功能性沟通成果方面取得了进展 在基线后6个月维持。接下来，CB 2试验提供了第一个严格的2a期，II期， PPA言语-语言干预的随机对照试验（RCT）。CB 2中期分析 条件把握度结果表明试验成功的可能性很高（>80%），但也确定需要更大的 心理社会教育和咨询。因此，本申请提出了CB 3试验，其设计为 通过测试是否增加了心理社会教育和咨询， (PEC)言语-语言干预（实验组）上级主动控制干预。 RCT将由目标1a补充，目标1a将在准备就绪后最大限度地提高实际试验准备就绪程度 实用性试验评估（RAPT）工具;以及目标2，将评估新的 可穿戴传感器使用“生活空间移动性”框架客观地测量生活参与度。审判 结果将为未来务实的试验设计提供信息。翻译潜力很高，因为交付模式 主要结局与当前临床实践一致。在没有明确治愈AD的方法的情况下， 相关的神经退行性疾病，重要的是要确定和评估的战略，帮助个人 最大限度地提高他们的生活质量，这个项目将有助于满足这一需求。