Pemphigus & Pemphigoid: from the bench to the bedside

描述(申请人提供)：天疱疮和类天疱疮是严重的皮肤起泡疾病。在了解天疱疮和类天疱疮的发病机制以及开发治疗这些疾病的新方法方面取得了快速进展。第一届和第二届天疱疮全国会议于2001年和2005年在国立卫生研究院校园举行，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：从替补席到床边”。它将由国际天疱疮和类天疱疮基金会赞助，并将聚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，特别是天疱疮和类天疱疮的机制和新的治疗方法。目标将是确定最有效地促进我们对天疱疮和类天疱疮的病因和治疗的进一步了解的研究机会领域，并在感兴趣的研究人员中交叉杂交如何实现这些目标的想法。这次会议的具体目的是：1)突出在确定天疱疮和类天疱疮发病机制方面的最新进展；2)讨论建立多国合作努力的可行性，以便：i)确定治疗天疱疮或类天疱疮患者的商定算法；ii)进行多机构随机试验；3)回应患者对更好信息和在治疗这些孤儿疾病方面取得更快进展的要求和需求。来自世界各地的主要研究人员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，将举行一次广泛的小组讨论，讨论最紧迫、与临床相关的问题，这些问题阻碍了更安全的治疗方法的开发，并具体解决了组织多中心水泡性疾病试验的障碍。我们预计这次会议将吸引140-160名致力于研究和治疗天疱疮和类天疱疮的科学家和临床医生。

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.