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

• 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
• 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

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PIDs是致命的，除非通过造血细胞移植（HCT）、酶替代或基因治疗实现免疫重建。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和多中心合作，以促进结果分析和前瞻性临床试验的发展。2009年，原发性免疫缺陷治疗联盟（PIDTC）在北美成立，目标是建立一个全国性的合作，开展回顾性、横断面和前瞻性研究，以确定接受HCT或其他形式治疗的患有严重联合免疫缺陷（SCID）、Wiskott-Aldrich综合征（was）和慢性肉芽肿病（CGD）的儿童的风险因素、长期结局和后期影响。拟议的年度PIDTC科学研讨会将代表一个独特而重要的论坛，聚集来自北美所有主要中心参与这些疾病治疗的经验丰富的年轻研究人员，以及生物统计学家和患者倡导团体。PIDTC研讨会将集中讨论及时的主题，以促进旨在改善pid治疗的临床试验的发展。讲习班的具体目标如下：1)传播患者的生存、临床状况、和患者的免疫功能严重PID收到HCT或其他形式的治疗,2)开始在其他形式的数据收集和分析可以治疗严重的PID HCT 3)生物标志物的相关性分析,可以预测成功的免疫重建HCT 4)讨论后的结果为PID HCT,新方法可以最小化transplant-related毒性而允许强劲和持久的移植和免疫重建5)探讨新生儿SCID筛查，制定诊断为SCID的新生儿治疗方案。6)促进HCT治疗PID领域临床试验的发展；7)促进对PID治疗有特殊兴趣的年轻研究人员的教育；8)增加积极参与PID宣传活动的患者倡导团体之间的协同作用