PIDTC Administrative Unit

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 研究网络（RDCRN）是一个由44个免疫学和造血干细胞移植联盟组成的联盟。 中心遍布美国和加拿大，成立于2009年，研究罕见的遗传性疾病， 免疫系统疾病，统称为原发性免疫缺陷病（PID）。PIDTC的目标 是了解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 酶替代疗法（ERT）用于严重联合免疫缺陷（SCID）患者，Wiskott- 奥尔德里奇综合征（WAS）和慢性肉芽肿病（CGD）。这些人是最有生命力的人， 威胁，通常需要HCT才能生存。然而，没有任何一个中心能够遵循足够的影响 个体以涵盖这些疾病的全部范围。因此，一个联合体是必不可少的， 需要每个PID的自然史和多中心研究进行稳健的统计评估， 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 结果。组织PIDTC的大量参与网站和多个项目是一项挑战， 需要一个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 和愿景，政策，程序和资金分配; 2）整合和监督内部的所有活动 以及PIDTC站点之间，包括协调PIDTC站点之间的通信， 将参与的研究者聚集在一个有凝聚力的PIDTC环境中; 3）为所有参与者提供生物统计学支持 PIDTC研究，包括项目、试点和项目间研究的数据分析， 为计划的倡议进行功率计算和研究设计; 4）作为与 RDCRN、RDCRN数据管理和协调中心（DMCC）、PID利益相关者和患者 宣传小组; 5）制作和更新太平洋岛屿发展理事会网站和通讯， 作为我们传播PIDTC的使命和成就、职业提升 机会，并了解PID和RDCRN范围内的资源; 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做法，包括使用 共同数据元素（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内外的关系， 在下一个融资周期中提高生产力和服务。

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.