Early-onset Alzheimer’s and other dementia: From natural history to clinical trials

早发性阿尔茨海默病和其他痴呆症：从自然史到临床试验

• 批准号: 10669554
• 负责人: Maria C Carrillo
• 金额: $ 4.42万
• 依托单位: ALZHEIMER'S ASSOCIATION
• 依托单位国家: 美国
• 财政年份: 2021
• 资助国家: 美国
• 起止时间: 2021-04-01 至 2025-03-31
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/10669554
• 关键词: Address; Affect; Age; Alzheimer' s Disease; American; Biological; Biological Markers; Blood Vessels; Caregivers; Classification; Clinical; Clinical Trials; Collaborations; Dedications; Dementia; Diagnosis; Doctor of Philosophy; Early Onset Alzheimer Disease; Emotional; Ensure; Etiology; Evaluation; Face; Family; Family member; Future; Goals; Hearing; Individual; Industry; International; Journals; Late Onset Alzheimer Disease; Lewy Bodies; Life; Life Cycle Stages; Manuscripts; Medical; Memory impairment; Natural History; Pathologic; Pathology; Patients; Persons; Phenotype; Policy Maker; Positioning Attribute; Postdoctoral Fellow; Press Releases; Recommendation; Recording of previous events; Research; Research Personnel; Role; Societies; Source; Symptoms; Therapeutic Trials; Voice; Work; advocacy organizations; career; coping; drug development; early onset; frontotemporal degeneration; genome-wide; human old age (65+); insight; interest; member; patient oriented; patient population; posters; programs; research and development; social media; symposium; trial design; web site; webinar

PROJECT SUMMARY Approximately 5.8 million Americans age 65 and older have Alzheimer's disease, and this number is expected to increase to 13.8 million by 2050. While most develop diagnosable symptoms at or over the age of 65 (e.g. late-onset), 5-10% develop symptoms at age 64 or younger and are classified as early-onset. Early- and late- onset Alzheimer's disease share the same pathologic substrate, but there are critical differences in their clinical presentations, biological phenotypes, and life impact. Moreover, early-onset dementia is more likely than late- onset dementia to be caused by pathologies other than Alzheimer's, including vascular, Lewy body, and frontotemporal degeneration. Because of their young age and atypical symptoms, early-onset patients often face a significant delay to diagnosis. Dementia onset at such a young age also has disproportionately devastating financial and emotional consequences for patients, families, and society. Due to age restrictions or absence of memory deficits, early-onset patients are under-represented in ongoing largescale genome and observational biomarker studies and in therapeutic trials. To date, no large-scale clinical trials have specifically addressed early-onset MCI and dementia. The Alzheimer's Association (AA) proposes to create and implement a two-day conference, to be held annually for 3 years, entitled Early-Onset Alzheimer's and Other Dementia: From Natural History to Clinical Trials. Patients, carers, and family members will be involved in the planning, implementation, and evaluation components of the conference. The short-term goal of the conference is to create a forum for discussion of research, trial design, drug development, and other concerns among key stakeholders. The long-term goal is to increase the likelihood, quality and patient-centeredness of future clinical trials addressing early-onset MCI and dementia. A Conference Workgroup made up of the PI and collaborators, additional leading experts in dementia, and individuals with early-onset MCI or dementia and/or their family members will develop, implement, and oversee all conference, evaluation, and dissemination activities. The conferences will be held in coordination with the annual Alzheimer's Association International Conference (AAIC) and offer didactic, panel discussion, and poster based formats, as well as face-to-face networking opportunities, anticipating 250 attendees. Lunch poster sessions dedicated to the early career investigators will be held on both days of the conference, and multiple approaches will be used to ensure diversity amongst speakers and attendees. Dissemination efforts will include webinars, press releases, social media highlights, and manuscripts documenting conference proceedings and findings.

项目概要 大约有 580 万 65 岁及以上的美国人患有阿尔茨海默病，这个数字是预期的 到 2050 年将增加到 1380 万。虽然大多数人在 65 岁或 65 岁以上时出现可诊断的症状（例如， 晚发型），5-10% 在 64 岁或更年轻时出现症状，被归类为早发型。早-和晚- 阿尔茨海默病的发病具有相同的病理基础，但其临床表现却存在重大差异 表现、生物表型和生活影响。此外，早发性痴呆比晚发性痴呆更有可能 起病性痴呆是由阿尔茨海默病以外的病理引起的，包括血管性痴呆、路易体痴呆和 额颞叶退化。早发患者由于年龄小、症状不典型，常 面临严重的诊断延迟。痴呆症在如此年轻的年龄发病的比例也不成比例 给患者、家庭和社会带来毁灭性的经济和情感后果。由于年龄限制或 由于不存在记忆缺陷，早发患者在正在进行的大规模基因组中代表性不足，并且 观察性生物标志物研究和治疗试验。迄今为止，还没有大规模的临床试验专门针对 解决早发性 MCI 和痴呆症。阿尔茨海默病协会 (AA) 提议创建并 举办为期两天的会议，每年举行一次，为期三年，题为早发性阿尔茨海默病和其他疾病 痴呆症：从自然史到临床试验。患者、护理人员和家属都将参与其中 会议的规划、实施和评估部分。该组织的短期目标 会议旨在创建一个讨论研究、试验设计、药物开发和其他问题的论坛 主要利益相关者之间。长期目标是提高治疗的可能性、质量和以患者为中心的程度 未来针对早发性 MCI 和痴呆症的临床试验。会议工作组由 PI 和 合作者、其他痴呆症领域的领先专家以及患有早发性 MCI 或痴呆症的个人和/或 他们的家庭成员将制定、实施和监督所有会议、评估和传播 活动。这些会议将与国际阿尔茨海默病协会年度会议协调举行 会议 (AAIC) 并提供教学、小组讨论、基于海报的形式以及面对面的形式 交流机会，预计将有 250 名与会者。致力于早期职业生涯的午餐海报会议 会议两天都将进行调查，并将采取多种方式确保 演讲者和与会者之间的多样性。传播工作将包括网络研讨会、新闻稿、社交媒体 媒体亮点以及记录会议记录和调查结果的手稿。