Creating a multi-center rare disease consortium - the Consortium of Eosinophilic Gastrointestinal Disease Researchers (CEGIR).
Creating a multi-center rare disease consortium - the Consortium of Eosinophilic Gastrointestinal Disease Researchers (CEGIR).
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DOI：
10.3233/trd-170016
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发表时间：
2017-12-18
期刊：
影响因子：
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通讯作者：
Rothenberg ME
中科院分区：
文献类型：
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作者：
Cheng K;Gupta SK;Kantor S;Kuhl JT;Aceves SS;Bonis PA;Capocelli KE;Carpenter C;Chehade M;Collins MH;Dellon ES;Falk GW;Gopal-Srivastava R;Gonsalves N;Hirano I;King EC;Leung J;Krischer JP;Mukkada VA;Schoepfer A;Spergel JM;Straumann A;Yang GY;Furuta GT;Rothenberg ME
嗜酸性胃肠道疾病（EGIDs）影响胃肠道的各个部分。由于这些疾病是罕见的，合作是必不可少的纳入受试者在临床研究和研究更广泛的人群。罕见病临床研究网络（RDCRN）是国家推进转化科学中心（NCATS）的一个项目，于2014年资助了嗜酸性胃肠道疾病研究人员联盟（CEGIR），以推进EGIDs领域的发展。CEGIR促进了各个中心、亚专科、患者、专业组织和患者倡导团体之间的合作，包括14个临床站点。它已经成功地启动了两项大型多中心临床研究，旨在改进EGID的诊断和管理。一些试点研究正在进行中，重点关注egid的各个方面，包括新的治疗干预措施、诊断和监测方法，以及微生物组在发病机制中的作用。CEGIR目前通过职业培训发展计划培养了5名医师学者，自成立以来已发表了40多篇手稿。这篇综述着重介绍了CEGIR的运作模式和进展，以及它如何促进思想交流和促进研究与创新的框架。该联盟为其他潜在临床领域的进展提供了一个模型。
Eosinophilic gastrointestinal disorders (EGIDs) affect various segments of the gastrointestinal tract. Since these disorders are rare, collaboration is essential to enroll subjects in clinical studies and study the broader population. The Rare Diseases Clinical Research Network (RDCRN), a program of the National Center for Advancing Translational Sciences (NCATS), funded the Consortium of Eosinophilic Gastrointestinal Disease Researchers (CEGIR) in 2014 to advance the field of EGIDs. CEGIR facilitates collaboration among various centers, subspecialties, patients, professional organizations and patient-advocacy groups and includes 14 clinical sites. It has successfully initiated two large multi-center clinical studies looking to refine EGID diagnoses and management. Several pilot studies are underway that focus on various aspects of EGIDs including novel therapeutic interventions, diagnostic and monitoring methods, and the role of the microbiome in pathogenesis. CEGIR currently nurtures five physician-scholars through a career training development program and has published more than 40 manuscripts since its inception. This review focuses on CEGIR’s operating model and progress and how it facilitates a framework for exchange of ideas and stimulates research and innovation. This consortium provides a model for progress on other potential clinical areas.