Stevens-Johnson Syndrome/Toxic Epidural Necrolysis 2017: Building Multidisciplinary Networks to Drive Science and Translation

项目摘要 史蒂文斯-约翰逊综合征/中毒性表皮坏死松解症(SJS/TEN)是一种免疫调节的疾病，通常 由药物引起的疾病很少见(估计发病率为1-3/100万)，但集体负担很大。这是最严重的 皮肤药物不良反应，通常有30%的死亡率和显著的短期和长期发病率。 在美国和国际上推进SJS/TEN研究、预防和临床护理需要 高度协调多个学科，如重症监护、外科、皮肤科、眼科、临床 药理学、流行病学、监管科学、信息学、免疫学、遗传学、皮肤生物学等领域 基础科学。通过由美国国立卫生研究院组织、NHGRI牵头的为期两天的研讨会，取得了重大进展 2015年3月，与FDA合作，有30名已成立的SJS/TEN研究人员参与，以确定优先事项。的确有 目前，没有专门针对SJS/TEN的持续应急小组或会议允许多学科小组 科学家和临床医生在一次会议上进行交流。该提案寻求对“史蒂文斯-约翰逊法案”的支持 综合症/中毒性表皮坏死松解症2017：构建多学科网络推动科学与翻译 解决这一未得到满足的需求。会议将于2017年3月2日举行，紧接着美国科学院 皮肤科会议(2017年3月3日至6日)，佛罗里达州奥兰多。该计划将吸引约200人参加，并 将鼓励多个科学和临床学科的新研究人员、实习生、妇女和少数族裔， 患者和患者权益倡导团体。为期一天的课程将包括演讲和更多 在SJS/TEN领域或在科学学科中有20多名国内和国际专家，这些专家有很多需要 为推进SJS/TEN作出贡献。该计划将包括各种演示格式，包括Keynote 演讲、圆桌讨论和海报会议。已经成立了一个组委会，它已经 具有杰出的国家和国际代表性，包括妇女、少数群体、新调查员、一名受训人员和 SJS基金会成员。这次会议的作用是1)创建网络；2)确定研究目标和未回答问题的优先顺序 问题；3)为新的研究人员和受训人员提供指导；4)召集主要研究人员介绍 尖端研究；以及5)解决在SJS/TEN研究方面取得短期和长期进展的潜在障碍。它是 预期本次会议的一个重要成果将是建立一个协同的全球研究人员网络 这将有一个持久和富有成效的合作关系。本次会议的记录将分发给 通过网络录音和同行评议的出版物。这与NIAMS和NIH的使命一致 会议将成功召集已建立或正在进行与这一严重生命威胁相关的工作的关键研究人员 皮肤药物不良反应的传播、教育、翻译和燃料方面的重大进展 将显著促进人类健康的SJS/TEN的原因、治疗和预防。

Project Summary Stevens-Johnson syndrome/toxic epidermal necrolysis (SJS/TEN) an immunologically-mediated disease that is usually drug-induced, is rare (estimated incidence 1-3/1,000,000) but has a high collective burden. It is the severest of cutaneous adverse drugs reactions, often with >30% mortality and significant short and long-term morbidity. Advancement in SJS/TEN research, prevention and clinical care within the United States and internationally requires considerable coordination of multiple disciplines such as critical care, surgery, dermatology, ophthalmology,clinical pharmacology, epidemiology, regulatory science, informatics, immunology, genetics, skin biology and other areas of basic science. Significant progress was achieved through a two-day workshop organized by the NIH and led by the NHGRI in collaboration with the FDA in March 2015 which involved 30 established SJS/TEN researchers to set priorities. There is currently no ongoing contingency group or conference dedicated to SJS/TEN that would allow a multidisciplinary group of scientists and clinicians to engage at a single meeting. This proposal seeks support for “Stevens-Johnson Syndrome/Toxic Epidermal Necrolysis 2017: Building Multidisciplinary Networks to Drive Science & Translation” to address this unmet need. The meeting will take place March 2, 2017 immediately preceding the American Academy of Dermatology meeting (March 3-6, 2017) in Orlando, Florida. The program will attract approximately 200 attendees and will encourage new investigators, trainees, women and minorities across multiple scientific and clinical disciplines, patients and patient advocacy groups. The one-day program will include presentations and involvement from more than 20 domestic and international experts both within the area of SJS/TEN or in scientific disciplines that have much to contribute to the advancement of SJS/TEN. The program will include a variety of presentation formats including keynote presentations, round table discussions and a poster session. An organizing committee has been assembled that has outstanding national and international representation and includes women, minorities, new investigators, a trainee and an SJS foundation member. This meeting will serve to 1) Create networks; 2) Prioritize research goals and unanswered questions; 3) Provide mentorship for new investigators and trainees; 4) Assemble key researchers for presentation of cutting edge research; and 5) Address potential barriers to short and long-term progress in SJS/TEN research. It is anticipated that an important outcome of this meeting will be the creation of a synergistic global network of researchers that will have a durable and productive collaborative relationship. The proceedings of this meeting will be disseminated through web-recording and a peer-reviewed publication. Consistent with the mission of the NIAMS and NIH this meeting will successfully assemble key researchers established or doing work of relevance to this severe life-threatening cutaneous adverse drug reaction for dissemination, education, translation and fuel significant progress in defining causes, treatment and prevention of SJS/TEN that will significantly advance human health.