PIDTC Administrative Unit

PIDTC管理核心-摘要 原发免疫缺陷治疗联盟(PIDTC)，罕见病临床成员 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 该中心成立于2009年，目的是研究人类罕见的遗传性疾病。 免疫系统疾病，统称为原发免疫缺陷病(PIDs)。PIDTC的目标是 已经了解了PID，并为其最终治疗确定了最佳方法。在最初的9年里， PIDTC研究了造血细胞移植(HCT)、基因治疗(GT)和 严重联合免疫缺陷(SCID)患者的酶替代疗法(ERT) 阿尔德里奇综合征(AS)和慢性肉芽肿病(CGD)。这些ID是最有生命力的- 具有威胁性，通常需要红细胞压积才能生存。然而，没有一个中心受到足够多的影响。 包括这些疾病的全部范围的个体。因此，一个联合体对于界定 为进行可靠的统计评估，需要对每个PID和多中心研究的自然历史进行评估 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， 需要一个精心设计的管理结构。PIDTC的行政核心一直是 为执行以下任务而开发：1)全面管理PIDTC，包括科学指导 以及愿景、政策、程序和资金分配；2)整合和监督内部的所有活动 以及在PIDTC站点之间，包括协调PIDTC站点之间的沟通和将 共同参与PIDTC环境；3)为所有人提供生物统计学支持 PIDTC研究，包括分析来自项目、试点和项目间研究和开发的数据 为计划中的计划进行电力计算和研究设计；4)作为与 RDCRN、RDCRN数据管理和协调中心(DMCC)以及PID利益相关方和患者 宣传团体(PAG)；5)制作和更新PIDTC网站和通讯，这两个网站都 作为我们宣传PIDTC的使命和成就的手段，职业提升 机会，以及对PID和RDCRN范围资源的认识；6)参与RDCRN范围的努力，以 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 公共数据元素(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。行政核心意志 继续加强和扩大PIDTC内外的关系，并不断增加 下一个供资周期的生产力和服务。

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.