Treatment Efficacy and Toxicity in Rheumatoid Arthritis Database and Repository

描述（由申请人提供）：类风湿性关节炎（RA）的治疗在过去10年中有了显著改善。然而，没有一种药物对每个患者都有效，毒性和价格差异很大，从甲氨蝶呤（MTX）的约400美元/年到生物制剂的15，000美元/年。这一建议是基于这样一个前提，即治疗RA的下一个重大进展不是额外的药物，而是大幅提高RA患者使用药物的效率和成本效益。确定RA治疗反应的临床有用标志物的主要障碍之一是缺乏前瞻性收集的治疗反应数据与生物样本相结合的队列。到目前为止，还没有尝试协调美国学术RA研究社区的努力，以创建一个公共资源。因此，我们的建议将满足一个关键的需求：建立一个学术研究者的基础设施，为未来的合作大规模登记奠定基础;并调整和团结许多组织的努力，共同目标是改善RA患者的护理。通过统一学术研究人员的努力，我们可以创建否则无法获得的资源，例如冷冻保存的血细胞库，以允许复杂的免疫学研究来剖析成功治疗RA的分子信号。我们的具体目标是：1）建立一个由致力于收集开始MTX或生物制剂治疗的RA患者的治疗反应数据和生物样品的领先学术研究者组成的合作网络; 2）实施一项协议，以促进统一的数据和标本收集; 3a）通过在10个地点招募少量RA患者进行“概念验证”研究，以确定该基础设施的可行性; 3b）利用关节炎基金会和企业合作伙伴的财政和实物捐助开展试点项目，以促进未来的赠款供资; 4）鼓励联邦资助机构、志愿卫生机构、专业组织和行业伙伴的合作努力，以建立一个大型的，可持续的数据库和知识库，以更好地了解治疗的分子基础，并迅速加速RA的转化研究。

DESCRIPTION (provided by applicant): Treatment of rheumatoid arthritis (RA) has improved dramatically in the last 10 years. No single drug, however, is effective in every patient, and there is great variability in toxicity and price, ranging from ~$400/year for methotrexate (MTX) to up to $15,000/year for biologic agents. This proposal is based on the premise that the next major advance needed in the treatment of RA is not additional drugs, but rather a dramatic improvement in the efficiency and cost-effectiveness of the use of drugs for individual patients with RA. One of the major obstacles to identifying clinically useful markers of treatment response in RA is the lack of cohorts with prospectively collected treatment response data coupled with biological samples. To date, there has been no attempt to harmonize the efforts of the US academic RA research community to create a public resource. Our proposal will thus fill a critical need: establishing an infrastructure of academic investigators to lay the foundation for future collaborative large-scale registries; and to align and unite the efforts of many organizations with the common goal of improving care of RA patients. By unifying the efforts of academic researchers, we can create resources that would not otherwise be available, such as a bank of cryopreserved blood cells to allow sophisticated immunologic research to dissect molecular signals of successful treatment of RA. Our specific aims are: 1) To create a collaborative network of leading academic investigators committed to collecting treatment response data and biologic samples in RA patients starting MTX or biologic agents; 2) To implement a protocol to facilitate uniform data and specimen collection; 3a) To perform a "proof of concept" study to determine the feasibility of this infrastructure by enrolling a small number of RA patients at 10 sites; 3b) To utilize financial and in-kind contributions from the Arthritis Foundation and corporate partners to perform pilot projects to facilitate future grant funding; 4) To stimulate collaborative efforts of federal funding agencies, voluntary health agencies, professional organizations and industry partners to enable creation of a large, sustainable database and repository to better understand the molecular basis of treatment and rapidly accelerate translational research in RA.