Primary Immune Deficiency Treatment Consortium Annual Scientific Meeting

原发性免疫缺陷治疗联盟年度科学会议

基本信息

批准号：
9330521
负责人：
MORTON COWAN
金额：
$ 1.8万
依托单位：
UNIVERSITY OF CALIFORNIA, SAN FRANCISCO
依托单位国家：
美国
项目类别：
财政年份：
2011
资助国家：
美国
起止时间：
2011-03-02 至 2018-02-28
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/9330521
关键词：
Advanced Development Affect Age American Asia Autoimmunity Awareness B-Lymphocytes Biological Markers Boston Caring Cell Count Cells Child Chimerism Chronic Granulomatous Disease Clinical Clinical Research Clinical Trials Collaborations Complement Consensus Data Analyses Data Collection Deficiency Diseases Development Diagnosis Disease Education Educational workshop Engraftment Enzymes Europe European Faculty Family Focus Groups Funding Generic Drugs Goals Immune Immune System Diseases Immune system Immunobiology Immunologics Immunologist Individual Infection Infection prevention International Knowledge Late Effects Life Los Angeles Lymphocyte Malignant - descriptor Malignant Neoplasms Measures Minority Molecular Mutation National Institute of Allergy and Infectious Disease Natural Killer Cells Nature Newborn Infant Non-Malignant North America Outcome Participant Patient-Focused Outcomes Patients Phenotype Predisposition Primary Health Care Prospective Studies Protocols documentation Rare Diseases Regimen Registries Research Personnel Risk Risk Factors Role SCID Mice San Francisco Scientist Severe Combined Immunodeficiency Source South America South American South Asian Specialist Supportive care T-Lymphocyte Therapeutic Time Toxic effect Transplantation Treatment Protocols United States National Institutes of Health Wiskott-Aldrich Syndrome Woman autoinflammation autoinflammatory base clinical development conditioning experience gene therapy graft vs host disease granulocyte hematopoietic cell transplantation immune function improved improved outcome interest meetings microbiome novel strategies patient advocacy group predictive marker prospective reconstitution synergism

项目摘要

PROJECT SUMMARY Primary immune deficiencies (PIDs) are a group of rare disorders of the immune system, resulting in increased susceptibility to infections, autoimmunity and malignancies. The most severe forms of PIDs are fatal, unless immune reconstitution is achieved by hematopoietic cell transplantation (HCT), enzyme replacement or gene therapy. Because of the many disease- and treatment-related variables that may affect outcome after HCT, both careful collection of data in Registries and multicenter collaboration are needed to facilitate analysis of outcome and development of prospective clinical trials. In 2009, the Primary Immune Deficiency Treatment Consortium (PIDTC) was established in North America with the goal of building a North American collaboration to carry-out retrospective, cross-sectional and prospective studies that would define risk factors, long term outcomes and late effects in children with Severe Combined Immunodeficiency (SCID), Wiskott-Aldrich Syndrome (WAS) and Chronic Granulomatous Disease (CGD) who receive HCT or other forms of definitive treatment. In April 2011 the first annual PIDCT Scientific Workshop was held in San Francisco followed by workshops in Boston (2012), Houston (2013), Seattle (2014), Montreal (2015) and Los Angeles (2016). These workshops and Education Days provide a unique and critical forum to assemble experienced and young investigators from all major centers in North America involved in the treatment of these disorders, as well as biostatisticians and Patient Advocacy Groups and have been responsible for initiating the development of national and international collaborations. The proposed 7th PIDTC Scientific Workshop and Education Day in Bethesda, MD (PIDTC2017) will focus on new timely topics to further advance the development of clinical trials aimed at improving treatment of PIDs, immunobiology of PIDs, the microbiome, gene therapy, and collaborations with Patient Advocacy Groups. The specific objectives of the workshops are: 1) To disseminate information on survival, clinical status, and immune function in patients with severe PIDs who have received HCT or other forms of treatment 2) To initiate data collection and analysis in other forms of severe PIDs that can be treated by HCT 3) To analyze relevance of biological markers that predict successful immune reconstitution following HCT 4) To discuss the results of novel approaches to HCT for PID that may minimize transplant-related toxicity while allowing robust and durable engraftment and immune reconstitution 5) To develop optimal treatment protocols for newborns with SCID. 6) To prompt development of clinical trials in the field of HCT for PIDs 7) To promote education of young investigators with a specific interest in treatment of PIDs 8) To increase synergy among Patient Advocacy Groups actively involved in PID awareness campaigns

项目概要原发性免疫缺陷 (PID) 是一组罕见的免疫系统疾病，导致对感染、自身免疫和恶性肿瘤的易感性。最严重的 PID 形式是致命的，除非免疫重建通过造血细胞移植（HCT）、酶替代或基因实现治疗。由于许多与疾病和治疗相关的变量可能影响 HCT 后的结果，需要在登记处仔细收集数据并进行多中心协作，以促进分析前瞻性临床试验的结果和发展。 2009年，初级免疫缺陷治疗联盟（PIDTC）在北美成立，目标是建立北美合作进行回顾性、横断面和前瞻性研究，以确定长期风险因素严重联合免疫缺陷 (SCID) 儿童的结果和后期影响，Wiskott-Aldrich 接受 HCT 或其他形式的明确治疗的综合征 (WAS) 和慢性肉芽肿病 (CGD) 治疗。 2011 年 4 月，第一届年度 PIDCT 科学研讨会在旧金山举行，随后在波士顿（2012 年）、休斯顿（2013 年）、西雅图（2014 年）、蒙特利尔（2015 年）和洛杉矶（2016 年）举办了研讨会。这些研讨会和教育日提供了一个独特而重要的论坛来聚集经验丰富的人和年轻人来自北美所有主要中心的研究人员参与这些疾病的治疗，以及生物统计学家和患者倡导团体，并负责启动开发国家和国际合作。拟议的第七届 PIDTC 科学研讨会和教育日马里兰州贝塞斯达（PIDTC2017）将重点关注新的及时主题，进一步推进临床试验的发展旨在改善 PID 的治疗、PID 的免疫生物学、微生物组、基因治疗和与患者权益团体合作。研讨会的具体目标是： 1) 传播重症患者的生存、临床状态和免疫功能信息接受过 HCT 或其他形式治疗的 PID 2) 启动可通过 HCT 治疗的其他形式的严重 PID 的数据收集和分析 3) 分析预测 HCT 后成功免疫重建的生物标志物的相关性 4) 讨论 HCT 治疗 PID 的新方法的结果，该方法可以最大限度地减少移植相关的毒性同时允许坚固耐用的植入和免疫重建 5) 为患有 SCID 的新生儿制定最佳治疗方案。 6) 促进PID HCT领域临床试验的开展 7) 促进对 PID 治疗有特殊兴趣的年轻研究者的教育 8) 增强积极参与 PID 宣传活动的患者权益团体之间的协同作用