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：
8234927
负责人：
MORTON COWAN
金额：
$ 2万
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 2014-02-28
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/8234927
关键词：
Affect Age Autoimmunity Awareness B-Lymphocytes Biological Markers Biological Markers Blood Bone Marrow Transplantation Caring Cell Count Cells Child Chimerism Chronic Granulomatous Disease Clinical Clinical Trials Collaborations Complement Consensus Data Analyses Data Collection Data Reporting Development Diagnosis Disease Education Educational workshop Engraftment Future Goals Health Immune Immune system Immunobiology Immunologics Individual Infection Infection prevention International Knowledge Late Effects Life Lymphocyte Malignant Neoplasms Measures Medical Molecular Mutation Natural Killer Cells Nature Neonatal Screening Newborn Infant North America Outcome Patients Phenotype Predisposition Prospective Studies Protocols documentation Rare Diseases Regimen Registries Research Personnel Risk Risk Factors SCID Mice Severe Combined Immunodeficiency Source T-Lymphocyte Therapeutic Time Toxic effect Transplantation United States Wiskott-Aldrich Syndrome X-Linked Severe Combined Immunodeficiency adenosine deaminase base conditioning enzyme replacement therapy experience gene therapy graft vs host disease granulocyte hematopoietic cell transplantation immune function improved interest international center meetings novel novel strategies patient advocacy group prospective reconstitution

项目摘要

DESCRIPTION (provided by applicant): 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 nation-wide 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 treatment. The proposed annual PIDTC Scientific Workshop will represent 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. The PIDTC Workshop will focus on timely topics to facilitate development of clinical trials aimed at improving treatment of PIDs. The specific objectives of the workshop are as follows: 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 the relevance of biological markers that can 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 discuss newborn screening for SCID and develop protocols for treating newborn infants diagnosed 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 PUBLIC HEALTH RELEVANCE: Severe forms of congenital Primary immune deficiencies (PIDs) are fatal, unless treated by hematopoietic cell transplantation (HCT), enzyme replacement or gene therapy. In 2009, the Primary Immune Deficiency Treatment Consortium (PIDTC) was established in North America with the goal of building multicenter collaboration to improve outcome in PID patients who receive HCT or other forms of treatment. The proposed annual PIDTC Scientific Workshop will assemble experienced and young investigators from all major centers in North America involved in the treatment of these disorders. Discussion will focus on timely topics to facilitate development of multicenter national and international clinical trials aimed at improving treatment of PIDs. Interaction with Patient Advocacy Groups will promote synergy in PID awareness campaigns.

描述（由申请人提供）：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增加。最严重的 PID 形式是致命的，除非通过造血细胞移植 (HCT)、酶替代或基因治疗来实现免疫重建。由于许多与疾病和治疗相关的变量可能影响 HCT 后的结果，因此需要在登记处仔细收集数据并进行多中心合作，以促进结果分析和前瞻性临床试验的开展。 2009 年，初级免疫缺陷治疗联盟 (PIDTC) 在北美成立，目标是建立全国范围的合作，开展回顾性、横断面和前瞻性研究，以确定风险因素、长期结果和后期治疗。对接受 HCT 或其他形式治疗的严重联合免疫缺陷 (SCID)、威斯科特-奥尔德里奇综合征 (WAS) 和慢性肉芽肿病 (CGD) 儿童的影响治疗。拟议的年度 PIDTC 科学研讨会将成为一个独特而重要的论坛，汇集来自北美参与这些疾病治疗的所有主要中心的经验丰富和年轻的研究人员，以及生物统计学家和患者倡导团体。 PIDTC 研讨会将重点关注及时的主题，以促进旨在改善 PID 治疗的临床试验的发展。研讨会的具体目标如下： 1) 传播接受 HCT 或其他形式治疗的严重 PID 患者的生存、临床状态和免疫功能信息 2) 启动其他形式的数据收集和分析可以通过 HCT 治疗的严重 PID 3) 分析可预测 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毒性，同时允许稳健和持久植入和免疫重建 5) 讨论新生儿 SCID 筛查并制定治疗诊断为 SCID 的新生儿的方案。 6) 促进 PID 的 HCT 领域临床试验的发展 7) 促进对 PID 治疗有特定兴趣的年轻研究人员的教育 8) 增强积极参与 PID 宣传活动的患者倡导团体之间的协同作用公共卫生相关性：严重的先天性原发性免疫缺陷 (PID) 是致命的，除非通过造血细胞移植 (HCT)、酶替代或基因疗法进行治疗。 2009 年，原发性免疫缺陷治疗联盟 (PIDTC) 在北美成立，目标是建立多中心合作，以改善接受 HCT 或其他形式治疗的 PID 患者的预后。拟议的年度 PIDTC 科学研讨会将汇集来自北美所有参与这些疾病治疗的主要中心的经验丰富和年轻的研究人员。讨论将集中在及时的主题上，以促进旨在改善 PID 治疗的多中心国家和国际临床试验的发展。与患者权益团体的互动将促进 PID 宣传活动的协同作用。