Primary Immune Deficiency Treatment Consortium (PIDTC): A resource to drive scientific and clinical advances related to the treatment of inborn errors of immunity

Project Summary/Abstract The Primary Immune Deficiency Treatment Consortium (PIDTC) was founded in 2009 to bring together immunologists, bone marrow transplant (BMT) physicians, and cell/gene therapists to improve treatments and outcomes for patients with inborn errors of immunity (IEI). The PIDTC has aggregated data from 49 sites across

the US and Canada to address key questions related to the treatment and outcomes of the most severe, life- threatening IEIs including Severe Combined Immune Deficiency (SCID), Chronic Granulomatous Disease (CGD), Wiskott- Aldrich Syndrome (WAS), and Primary Immune Regulatory Disorders (PIRD). The PIDTC database, along

with the associated tools that have been built to facilitate acquisition of detailed longitudinal data across a diverse network of clinical sites, and the aggregate experience of the involved clinical and scientific experts, has become an indispensable resource for research, treatment planning, and clinical care advancement within the IEI

community. We propose that the data, materials, and expertise of PIDTC now be organized and made available as a resource to the entire IEI community with key enhancements that will empower the community to easily access and use the PIDTC Resource, and simultaneously ensure the continued growth and sustainment of the PIDTC

Resource. We will expand the PIDTC Resource to increase its utility and improve the power to answer further questions related to the treatment of IEI's including particular genotypes or clinical subtypes of disease. We will consolidate and build tools and infrastructure to accelerate standardized data collection from ongoing and future

studies and trials that are contributing data to the PIDTC Resource. We will provide tools to make the PIDTC data more readily accessible to the broader IEI community including researchers, clinical care providers, patient advocacy groups, patients, foundations, and consortia. This expansion and transformation of the PIDTC into an

open resource will enable diverse users to take advantage of unparalleled IEI datasets to increase understanding and develop better health outcomes for patients as well as conduct new studies with the support of the PIDTC Resource database, expertise and infrastructure. Together, we will further propel the PIDTC's mission to study

therapeutic outcomes in IEI and improve the outcomes of patients with IEI.