Pemphigus & Pemphigoid: from the bench to the bedside

天疱疮

• 批准号: 7996440
• 负责人: SERGEI A GRANDO
• 金额: $ 2万
• 依托单位: INTERNATIONAL PEMPHIGUS PEMPHIGOID FDN
• 依托单位国家: 美国
• 财政年份: 2010
• 资助国家: 美国
• 起止时间: 2010-07-15 至 2011-06-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7996440
• 关键词: Address; Algorithms; Area; Autoantibodies; Basic Science; Bulla; Bullous Pemphigoid; Clinical; Clinical Investigator; Clinical Trials; Communities; Consensus; Data; Development; Disease; Foundations; Goals; Human; International; Mediating; Multicenter Trials; Orphan Disease; Pathogenesis; Patients; Pemphigus; Physicians; Public Health; Rare Diseases; Research; Research Personnel; Scientist; Severity of illness; Terminology; Touch sensation; United States National Institutes of Health; Work; bench to bedside; clinical application; clinically relevant; effective therapy; interest; meetings; novel; novel strategies; novel therapeutic intervention; randomized trial; response; skin disorder; symposium

DESCRIPTION (provided by applicant): Pemphigus and pemphigoid are serious blistering diseases of the skin. Rapid advances are being made in understanding the pathogenesis of pemphigus and pemphigoid and developing novel approaches to these diseases. The first and second national conferences on pemphigus were held on the NIH campus in 2001 and 2005 and were very successful. We now propose a third conference -- "Pemphigus & Pemphigoid: from the bench to the bedside". It will be sponsored by the International Pemphigus and Pemphigoid Foundation, and will bring together basic scientists and clinical investigators working on the mechanisms of, and novel therapeutic approaches to, human autoantibody mediated diseases in general and pemphigus and pemphigoid, in particular. The goal will be to identify areas of research opportunity that will most effectively promote our further understanding of the causes and the treatments of pemphigus and pemphigoid, and to cross-hybridize ideas on how to reach these goals among interested researchers. The Specific Aims of this conference are to: 1) highlight recent advances in defining the pathogenesis of pemphigus and pemphigoid; 2) discuss the feasibility of setting up cooperative multinational endeavors, so as to: i) define agreed upon algorithm to treat patients with pemphigus or pemphigoid; and ii) conduct multi-institutional randomized trials; and 3) respond to patient's requests and needs for better information and for more rapid progress in the treatment of these orphan diseases. There will be focused presentations of the clinical trial data in patients with pemphigus and pemphigoid by the leading investigators from all over the world. The scientific presentations will be followed by an extended group discussion of the most urgent, clinically relevant issues that hamper development of safer treatments, and specifically address obstacles to organizing multicenter trials for blistering diseases. We expect the conference will attract 140-160 scientists and clinicians dedicated to investigate and treat pemphigus and pemphigoid.

描述（由申请人提供）：天疱疮和类天疱疮是严重的皮肤起泡疾病。在理解天疱疮和类天疱疮的发病机制和开发治疗这些疾病的新方法方面取得了快速进展。2001年和2005年在NIH校园举行了第一次和第二次全国天疱疮会议，非常成功。我们现在提议召开第三次会议-“天疱疮和类天疱疮：从板凳到床边”。它将由国际天疱疮和类天疱疮基金会赞助，并将汇集基础科学家和临床研究人员，研究人类自身抗体介导的疾病的机制和新的治疗方法，特别是天疱疮和类天疱疮。目标是确定最有效地促进我们进一步了解天疱疮和类天疱疮的病因和治疗方法的研究机会领域，并在感兴趣的研究人员中就如何实现这些目标进行交叉杂交。本次会议的具体目的是：1）强调在定义天疱疮和类天疱疮发病机制方面的最新进展; 2）讨论建立合作多国努力的可行性，以便：i）定义商定的治疗天疱疮或类天疱疮患者的算法;和ii）进行多机构随机试验;以及3）响应患者的要求和需要，以获得更好的信息，并在治疗这些孤儿疾病方面取得更快的进展。来自世界各地的主要研究者将重点介绍天疱疮和类天疱疮患者的临床试验数据。在科学报告之后，将进行一次扩大的小组讨论，讨论阻碍更安全治疗方法开发的最紧迫的临床相关问题，并特别讨论组织多中心水泡疾病试验的障碍。我们预计会议将吸引140-160名科学家和临床医生致力于研究和治疗天疱疮和类天疱疮。