PIDTC Administrative Unit

PIDTC 行政单位

• 批准号: 10682531
• 负责人: Jennifer M. Puck
• 金额: $ 168.9万
• 依托单位: UNIVERSITY OF CALIFORNIA, SAN FRANCISCO
• 依托单位国家: 美国
• 财政年份: 2009
• 资助国家: 美国
• 起止时间: 2009-09-12 至 2024-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10682531
• 关键词: Achievement; Affect; Awareness; Biometry; Canada; Caring; Cells; Chronic Granulomatous Disease; Clinical; Clinical Research; Common Data Element; Communication; Consensus; Data; Data Analyses; Data Coordinating Center; Disease; Ensure; Environment; Funding; Goals; Hematopoietic Stem Cell Transplantation; Immune System Diseases; Immunity; Immunology; Individual; Lead; Life; Mission; Multi-Institutional Clinical Trial; Multicenter Studies; Natural History; Newsletter; Outcome; Outcome Study; Patients; Policies; Procedures; Production; Productivity; Rare Diseases; Regimen; Research; Research Design; Research Personnel; Research Project Grants; Resources; Scientist; Services; Severe Combined Immunodeficiency; Site; Source; Statistical Data Interpretation; Structure; Supervision; Update; Vision; Wiskott-Aldrich Syndrome; career; conditioning; congenital immunodeficiency; enzyme replacement therapy; evidence base; experience; gene therapy; hematopoietic cell transplantation; improved; member; organizational structure; patient advocacy group; rare genetic disorder; tool; transplant centers; web site

PIDTC Administrative Core – Abstract The Primary Immune Deficiency Treatment Consortium (PIDTC), a member of the Rare Diseases Clinical Research Network (RDCRN), is a Consortium of 44 immunology and hematopoietic stem cell transplant centers throughout the USA and Canada, which was established in 2009 to study rare genetic disorders of the immune system, collectively known as primary immunodeficiency diseases (PIDs). The goals of the PIDTC have been to understand PIDs and define optimal approaches for their definitive treatment. In its first 9 years, the PIDTC has studied outcomes following hematopoietic cell transplantation (HCT), gene therapy (GT) and enzyme replacement therapy (ERT) for patients with severe combined immunodeficiency (SCID), Wiskott- Aldrich syndrome (WAS) and chronic granulomatous disease (CGD). These PIDs are among the most life- threatening, often requiring HCT for survival. However, no single center has followed enough affected individuals to encompass the full spectrum of these disorders. Therefore, a consortium is essential to define the natural history of each PID and multicenter studies are required for robust statistical assessment to compare impacts not only of patient-related variables, but also of treatment-related variables on clinical outcome. Organizing the large number of participating sites and multiple Projects of the PIDTC is a challenge, requiring a carefully crafted administrative structure. The Administrative Core of the PIDTC has been developed to perform the following tasks: 1) overall administration of the PIDTC, including scientific direction and vision, policies, procedures and allocation of funds; 2) integration and supervision of all activities within and among the PIDTC sites, including coordinating communication among the PIDTC sites and bringing together participating investigators into a cohesive PIDTC environment; 3) providing biostatistical support for all PIDTC research, including analysis of data from Projects, Pilots, and inter-Project studies and developing power calculations and study designs for planned initiatives; 4) serving as the point of coordination with the RDCRN, the RDCRN Data Management and Coordinating Center (DMCC) and PID stakeholders and Patient Advocacy Groups (PAGs); 5) production and updates of the PIDTC website and newsletter, both of which serve as our means to broadcast the mission and achievements of the PIDTC, career enhancement opportunities, and awareness of PID and RDCRN-wide resources; 6) participation in RDCRN-wide efforts to develop and disseminate tools and best practices for handling clinical and research data, including the use of Common Data Elements (CDEs), and 7) ensuring a mutually supportive interaction between the scientists conducting clinical research to further the achievement of the goals of the PIDTC. The Administrative Core will continue strengthening and expanding relationships both within and beyond the PIDTC and increasing productivity and services during the next funding cycle.

PIDTC管理核心 - 摘要 初级免疫缺陷治疗联盟（PIDTC），稀有疾病的成员临床 研究网络（RDCRN）是44个免疫学和造血干细胞移植的财团 整个美国和加拿大的中心成立于2009年，旨在研究罕见的遗传疾病 免疫系统，统称为原发性免疫缺陷疾病（PID）。 PIDTC的目标 已经理解PID并为其确定治疗定义了最佳方法。在最初的9年中 PIDTC在造血细胞移植（HCT），基因治疗（GT）和 严重合并免疫缺陷（SCID）的患者酶替代疗法（ERT），Wiskott- Aldrich综合征（WAS）和慢性肉芽肿性疾病（CGD）。这些PID是一生中最重要的 - 威胁，通常需要HCT生存。但是，没有一个中心遵循足够的影响 个人涵盖了这些疾病的全部范围。因此，财团对于定义至关重要 每种PID和多中心研究的自然病史都是必需的 比较不仅与患者相关的变量的影响，还比较与治疗相关的变量对临床的影响 结果。组织大量参与站点和PIDTC的多个项目是一个挑战， 需要精心制作的行政结构。 PIDTC的管理核心已经 开发以执行以下任务：1）PIDTC的总体给药，包括科学方向 和愿景，政策，程序和资金分配； 2）对所有活动的整合和监督 在PIDTC站点中，包括PIDTC站点之间的协调沟通并带来 共同参与调查人员的凝聚力PIDTC环境； 3）为所有人提供生物统计支持 PIDTC研究，包括对项目，飞行员和项目间研究的数据的分析以及开发 电力计算和计划设计的设计； 4）作为与 RDCRN，RDCRN数据管理和协调中心（DMCC）以及PID利益相关者和患者 倡导组（PAG）； 5）PIDTC网站和新闻通讯的生产和更新都 作为广播PIDTC的使命和成就，职业增强的手段 机会，以及对PID和RDCRN范围内的资源的认识； 6）参加RDCRN范围的努力 开发和传播用于处理临床和研究数据的工具以及最佳实践，包括使用 常见数据元素（CD）和7）确保科学家之间的相互支持 进行临床研究以进一步实现PIDTC的目标。行政核心将 继续加强和扩展PIDTC内外的关系并增加 在下一个资金周期中的生产力和服务。