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 行政核心 – 摘要 原发性免疫缺陷治疗联盟 (PIDTC)，罕见疾病临床协会成员 研究网络 (RDCRN)，是一个由 44 个免疫学和造血干细胞移植组成的联盟 遍布美国和加拿大的中心，成立于 2009 年，旨在研究罕见的遗传性疾病 免疫系统，统称为原发性免疫缺陷疾病（PID）。 PIDTC 的目标 我们一直致力于了解 PID 并确定其最终治疗的最佳方法。在最初的 9 年里， PIDTC 研究了造血细胞移植 (HCT)、基因治疗 (GT) 和 针对严重联合免疫缺陷 (SCID) 患者的酶替代疗法 (ERT)，Wiskott- 奥尔德里奇综合征（WAS）和慢性肉芽肿病（CGD）。这些 PID 是最长寿的 具有威胁性，通常需要 HCT 才能生存。然而，没有一个中心对受影响的情况进行足够的跟踪 个人涵盖这些疾病的全部范围。因此，有必要成立一个联盟来定义 每个 PID 的自然史和多中心研究都需要进行稳健的统计评估，以 不仅比较患者相关变量的影响，还比较治疗相关变量对临床的影响 结果。组织 PIDTC 的大量参与地点和多个项目是一项挑战， 需要精心设计的行政结构。 PIDTC 的行政核心是 制定执行以下任务： 1) PIDTC 的全面管理，包括科学指导 愿景、政策、程序和资金分配； 2）整合和监督内部所有活动 以及 PIDTC 站点之间的通信，包括协调 PIDTC 站点之间的通信并带来 将参与的研究人员聚集到一个有凝聚力的 PIDTC 环境中； 3）为所有人提供生物统计支持 PIDTC 研究，包括对项目、试点以及项目间研究和开发的数据进行分析 计划举措的功率计算和研究设计； 4）作为协调点 RDCRN、RDCRN 数据管理和协调中心 (DMCC) 以及 PID 利益相关者和患者 倡导团体（PAG）； 5) PIDTC 网站和时事通讯的制作和更新，两者 作为我们传播 PIDTC 的使命和成就、职业提升的手段 机会以及对 PID 和 RDCRN 范围资源的认识； 6) 参与 RDCRN 范围内的努力 开发和传播处理临床和研究数据的工具和最佳实践，包括使用 通用数据元素 (CDE)，以及 7) 确保科学家之间相互支持的互动 进行临床研究以进一步实现 PIDTC 的目标。行政核心将 继续加强和扩大 PIDTC 内部和外部的关系，并增加 下一个资助周期的生产力和服务。