Developing an Alopecia Areata Disease Activity Index (ALADIN)

制定斑秃疾病活动指数 (ALADIN)

• 批准号: 9117143
• 负责人: Angela M Christiano
• 金额: $ 10万
• 依托单位: COLUMBIA UNIVERSITY HEALTH SCIENCES
• 依托单位国家: 美国
• 财政年份: 2014
• 资助国家: 美国
• 起止时间: 2014-09-01 至 2017-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/9117143
• 关键词: Academic Medical Centers; Adolescent; Affect; Age; Alopecia Areata; Appearance; Autoimmune Diseases; Biological; Biological Markers; Child; Clinical; Clinical Trials; Clinical assessments; Collection; Crohn' s disease; Data; Development; Disease; Disease Progression; Electronics; Emotional; Ethnic group; Female; Futility; Gene Expression; Gene Expression Profiling; Genomics; Genotype; Goals; Hair; Health; Human; Individual; Intervention; Laboratories; Link; Measurement; Measures; Modeling; Molecular; Monitor; Outcome; Patient Outcomes Assessments; Patients; Pharmaceutical Preparations; Physicians; Population; Property; Psychological Impact; Psychometrics; Reporting; Sampling; Self Perception; Serum; Site; Testing; Therapeutic; United States; Weight; clinically relevant; diaries; drug efficacy; health related quality of life; indexing; innovation; insight; instrument; interest; lifetime risk; male; outcome forecast; psychologic; psychological distress; response; risk variant; skin disorder; tool

DESCRIPTION (provided by applicant): The goal of this application is to develop a new outcome instrument, the Alopecia Areata Disease Activity Index (ALADIN) that has the potential for use in the determination of clinically relevant endpoints for Alopecia Areata (AA). AA is one of the most common autoimmune diseases, with a lifetime risk of approximately 2%, affecting 5.3 million individuals in the United States alone, including males and females of all ages and ethnic groups. AA represents the second most common form of human hair loss, and causes significant disfigurement and psychological distress to affected individuals. AA carries one of the highest burdens among any skin diseases, particularly among children and adolescents whose self-image is so closely linked to their appearance. Integration of patient reported outcomes (PROs) as disease endpoints is needed in the determination of drug efficacy and in the comparison of outcomes across drug trials for AA. Because of the high psychological burden associated with AA, it is essential that the patient's perspective be considered as a core outcome in the development of new treatments for AA. For this reason, PROs need to be developed that can capture the emotional and psychological impact of the disease. To our knowledge, the emotional and psychological impacts of AA on patients' health related quality of life have not yet been included in any completed RCT studies. In order to incorporate an AA specific PRO into the assessment of disease progression/status, there needs to exist a reliable and valid instrument that measures a uniform set of outcomes in clinical trials for AA. In the context of our ongoing clinical trials in AA at Columbia University Medical Center, we have the unique opportunity to develop and deploy the Alopecia Areata Disease Activity Index (ALADIN) into clinical trials for AA, where we will be able to assess its psychometric properties in an AA population. ALADIN will be constructed by integrating a patient reported health-related quality of life instrument; physician reported hair loss assessments, clinical measures, a genotype risk score, and gene expression analysis and serum biomarkers. ALADIN will be modeled after the well-accepted and extensively validated Crohn's disease activity index (CDAI) which similarly contains patient reported measures; physician reported measures, and biological data. We will apply innovative statistical approaches in developing ALADIN, to create a classifier that quantitatively distinguishes disease state, prognosis and therapeutic response. The final instrument will be a single index that can be used to monitor disease activity in AA, and will be easily adaptable to multiple clinical trial sites and allow comparison across trials.

描述（由申请人提供）：本申请的目的是开发一种新的结果工具，即Areata Areata疾病活动指数（Aladin），该指数（Aladin）有可能在确定临床相关的Aleata（AA）的临床相关端点。 AA是最常见的自身免疫性疾病之一，终生风险约为2％，仅影响了美国的530万人，包括各个年龄段和种族的男性和女性。 AA代表了人脱发的第二大最常见形式，并给受影响的个体造成严重的毁容和心理困扰。 AA带有一个 任何皮肤疾病的负担最高，尤其是在自我形象与外表密切相关的儿童和青少年中。在确定药物疗效并比较AA药物试验中的结果时，需要将患者报告的结果（PRO）整合为疾病终点。由于与AA相关的心理负担很大，因此必须将患者的观点视为开发AA新疗法的核心结果。因此，需要开发可以捕捉疾病的情感和心理影响的专利人。据我们所知，AA对患者与健康相关的生活质量的情感和心理影响尚未包含在任何完成的RCT研究中。为了将特定于AA的Pro纳入疾病进展/状况的评估中，需要存在一种可靠且有效的工具，该工具在AA的临床试验中测量了一组均匀的结果。在我们在哥伦比亚大学医学中心正在进行的AA临床试验的背景下，我们有独特的机会开发和部署脱发Areata疾病活动指数（Aladin）进入AA的临床试验，我们将能够评估其在AA人群中的心理学特性。阿拉丁将通过整合报告的与健康相关的生活质量工具来建造；医师报告了脱发评估，临床测量，基因型风险评分以及基因表达分析和血清生物标志物。 Aladin将以经过良好认可和广泛验证的克罗恩病活性指数（CDAI）的模型进行建模，该指数（CDAI）类似地包含患者报告的措施；医师报告了措施和生物学数据。我们将采用创新的统计方法来开发阿拉丁，创建一个分类器，以定量区分疾病状态，预后和治疗反应。最终的仪器将是一个单个指数，可用于监测AA中的疾病活动，并且很容易适应多个临床试验地点，并允许在试验中进行比较。