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：
8434252
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/8434252
关键词：
Affect Age Autoimmunity Awareness B-Lymphocytes 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

描述（由申请人提供）：原发性免疫缺陷（PID）是免疫系统的一组罕见疾病，导致对感染，自身免疫和恶性肿瘤的敏感性增加。除非通过造血细胞移植（HCT），酶替代或基因治疗实现免疫结构，否则最严重的PID形式是致命的。由于可能影响HCT后可能影响结果的许多疾病和治疗相关的变量，因此需要仔细的注册表和多中心协作收集，以促进对预期的分析和前瞻性临床试验的发展。 2009年，在北美建立了主要的免疫缺陷治疗联盟（PIDTC），其目的是建立一项全国范围的合作，以进行回顾性，横断面和前瞻性研究，以定义风险因素，长期结果和迟到的影响。治疗。拟议的年度PIDTC科学研讨会将代表一个独特而关键的论坛，以组建北美所有主要中心的经验丰富和年轻的研究人员，涉及这些疾病的治疗，以及生物统计学家和患者倡导组织。 PIDTC研讨会将重点介绍及时的主题，以促进旨在改善PID治疗的临床试验。研讨会的具体目标如下：1）在接受HCT或其他形式治疗的严重PID患者中传播有关生存，临床状况和免疫功能的信息，以启动数据收集和分析的其他形式的严重PID，可以通过HCT 3）进行HCT 3）进行生物学标记的相关性，以预测HIMSITION的相关性，以预测HCT 4）的相关性。最小化与移植相关的毒性，同时允许健壮和耐用的植入和免疫重建剂5）讨论SCID的新生儿筛查，并开发用于治疗被诊断为SCID的新生婴儿的方案。 6）促使PID的HCT领域的临床试验开发7）促进对PID治疗特别兴趣的年轻研究人员的教育8），以增加积极参与PID意识运动的患者倡导小组之间的协同作用