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）：本申请的目标是开发一种新的结果工具，即斑秃疾病活动指数（ALADIN），它有可能用于确定斑秃（AA）的临床相关终点。 AA 是最常见的自身免疫性疾病之一，终生患病风险约为 2%，仅在美国就有 530 万人受影响，其中包括各个年龄段和种族的男性和女性。 AA 是人类脱发的第二常见形式，会给受影响的人造成严重的毁容和心理困扰。 AA 携带其中之一 在所有皮肤病中，这种疾病的负担最高，尤其是自我形象与其外表密切相关的儿童和青少年。在确定药物疗效和比较 AA 药物试验的结果时，需要将患者报告的结果 (PRO) 整合为疾病终点。由于 AA 带来较高的心理负担，因此在开发 AA 新疗法时必须将患者的观点作为核心结果。因此，需要开发能够捕捉疾病对情绪和心理影响的 PRO。据我们所知，任何已完成的随机对照试验研究尚未纳入 AA 对患者健康相关生活质量的情绪和心理影响。为了将 AA 特异性 PRO 纳入疾病进展/状态的评估中，需要存在一种可靠且有效的工具来测量 AA 临床试验中的一组统一结果。在哥伦比亚大学医学中心正在进行的 AA 临床试验中，我们有独特的机会开发斑秃疾病活动指数 (ALADIN) 并将其部署到 AA 临床试验中，我们将能够评估其心理测量特性在 AA 人群中。 ALADIN 将通过整合患者报告的健康相关生活质量工具来构建；医生报告了脱发评估、临床测量、基因型风险评分、基因表达分析和血清生物标志物。 ALADIN 将按照广为接受且广泛验证的克罗恩病活动指数 (CDAI) 建模，该指数同样包含患者报告的测量值；医生报告测量结果和生物数据。我们将在开发 ALADIN 时应用创新的统计方法，创建一个能够定量区分疾病状态、预后和治疗反应的分类器。最终的工具将是一个可用于监测 AA 疾病活动的单一指数，并且将很容易适应多个临床试验地点并允许跨试验进行比较。