Fourth Dense Deposit Disease Focus Group Meeting

描述(申请人提供)：第四届国际致密沉积病(DDD)焦点小组会议将于2011年8月20日在荷兰莱顿举行，紧接着第13届欧洲人类疾病补体会议。这款R13将用于支持将参加本次会议的北美研究生、初级研究科学家和初级教职员工。第四届国际DDD专题组会议的目的和目标是：1.介绍我们对C3肾小球疾病(包括DDD和原发性肾小球肾炎伴C3沉积(GN-C3))的病理生理学研究的进展；2.介绍检测能力的进展以及C3肾病因子和其他抗体，如因子H自身抗体(FHAA)和B因子自身抗体(FBAA)在DDD中的可能影响；3.介绍DDD的遗传学研究，并讨论遗传数据是否可以用于预测临床结果；4.评估抗补体治疗在DDD中的作用。这些目的和目标是前三届DDD国际会议的合乎逻辑的延伸，并将通过以下方式实现：1.聚集具有补体专业知识的临床医生和基础科学家；在DDD方面有专业知识的肾病病理学家；在肾小球肾炎和临床试验方面有专业知识的临床肾病医生；在DDD方面有专业知识的人类遗传学家；以及在糖胺多糖和肾小球基底膜(GBM)方面有专业知识的生物化学家；2.集中研究导致DDD的致病机制，特别是最近发现的涉及C3b和末端补体级联的分解产物在致密沉积物形成中的作用；3.评估C3Nef、FHAA和FBAA在DDD中的临床相关性；4.介绍我们对DDD作为一种复杂疾病的理解方面的遗传学进展，包括在一大批DDD患者中评估所有补体基因编码序列的研究；5.回顾eculizumab在DDD治疗中的现状；6.介绍其他抗补体疗法治疗DDD的数据；7.为研究人员会议计划欧洲/北美DDD临床试验奠定基础。可能会公布第四次DDD焦点小组会议的结果。值得注意的是，在之前三次会议中的两次会议之后，共识声明发表在《美国肾病学会杂志》上(阿佩尔等人，2005年；史密斯等人，2007年)，并对DDD患者的护理产生了影响(见附录)。 与公共卫生相关：这笔赠款用于支持将参加8月20日在荷兰莱顿举行的第四届DDD焦点小组会议的北美研究生、初级研究科学家和初级教员。这些人还将参加紧随DDD会议之后举行的第13届欧洲人类疾病补体会议。

DESCRIPTION (provided by applicant): The Fourth International Dense Deposit Disease (DDD) Focus Group Meeting will be held in Leiden, The Netherlands on August 20th, 2011, immediately before the 13th European Meeting on Complement in Human Disease. This R13 wil be used to support graduate students, junior research scientists and junior faculty from North America who will attend this meeting. The aims and objectives of the Fourth International DDD Focus Group Meeting are: 1. To present advances in our understanding of the pathophysiology of C3-glomerulopathies, which include DDD and primary glomerulonephritis with C3 deposits (GN-C3); 2. To present advances in testing capabilities and the possible impact of C3 nephritic factors (C3Nefs) and other antibodies such as factor H autoantibodies (FHAA) and factor B autoantibodies (FBAA) in DDD; 3. To present genetic studies of DDD and discuss whether genetic data can be used to predict clinical outcome; 4. To evaluate the role of anti-complement therapies,such as eculizumab, in DDD. These aims and objectives are a logical extension of the prior three international conferences on DDD and will be achieved by: 1. Bringing together clinicians and basic scientists with expertise in complement; nephropathologists with expertise in DDD; clinical nephrologists with expertise in glomerulonephritis and clinical trials; human geneticists with expertise in DDD; and biochemists with expertise in glycosaminoglycans and the glomerular basement membrane (GBM); 2. Focusing on the pathogenic mechanisms that lead to DDD, with special emphasis on recent discoveries that implicate breakdown products of both C3b and the terminal complement cascade in the formation of the dense deposits; 3. Evaluating the clinical relevance of C3Nefs, FHAA and FBAA in DDD; 4. Presenting genetic advances in our understanding of DDD as a complex disease, including studies that have evaluated coding sequence of all complement genes in a large cohort of DDD patients; 5. Reviewing the current status of eculizumab in the treatment of DDD; 6. Presenting data on other anti-complement therapeutics as treatments for DDD; 7. Laying the groundwork for investigators meetings to plan a European/North American DDD clinical trail. The outcome of the Fourth DDD Focus Group Meeting may be published. Of note, after two of the three prior meetings, consensus statements were published in the Journal of the American Society of Nephrology (Appel et al., 2005; Smith et al., 2007) and have impacted the care of patients with DDD (see Appendix). PUBLIC HEALTH RELEVANCE: This grant is to support graduate students, junior research scientists and junior faculty from North America who will attend the Fourth DDD Focus Group Meeting to be held August 20th in Leiden, The Netherlands. These persons will also attend the 13th European Meeting on Complement in Human Disease immediately following the DDD conference.