# PIDTC Administrative Unit

> **NIH NIH U54** · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · 2020 · $1,667,101

## Abstract

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 se...

## Key facts

- **NIH application ID:** 10018647
- **Project number:** 5U54AI082973-12
- **Recipient organization:** UNIVERSITY OF CALIFORNIA, SAN FRANCISCO
- **Principal Investigator:** Jennifer M. Puck
- **Activity code:** U54 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $1,667,101
- **Award type:** 5
- **Project period:** 2009-09-12 → 2024-08-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10018647

## Citation

> US National Institutes of Health, RePORTER application 10018647, PIDTC Administrative Unit (5U54AI082973-12). Retrieved via AI Analytics 2026-05-23 from https://api.ai-analytics.org/grant/nih/10018647. Licensed CC0.

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