Primary Immune Deficiency Treatment Consortium (PIDTC)

The Primary Immune Deficiency Treatment Consortium (PIDTC) was established in 2009 to study and define optimal treatments for rare genetic disorders of the immune system, collectively known as primary immunodeficiency diseases. The PIDTC includes 44 immunology and transplantation centers throughout the United States and Canada as well as six patient advocacy groups. In its first nine years, the PIDTC has studied clinical features and 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). Primary immunoregulatory disorders (PIRD) were recently added for the current funding cycle. All these diseases were chosen because they are life-threatening and difficult to treat, often requiring HCT for survival. Because no single center follows enough affected individuals to encompass the full spectrum of these disorders, the consortium has been essential to define their natural history and perform robust statistical assessments to address the impact of patient-related variables, such as genotypes and infections, as well as treatment-related variables on clinical outcomes. PIDTC contributions to understanding pathogenesis and defining which treatments produce optimal survival and quality of life have been published in over 100 papers to date. The PIDTC is working to strengthen its infrastructure for multicenter clinical trials so as to apply new knowledge about these primary immunodeficiencies to achieve advances in care, with the goal of improving the lives of people living with these rare medical conditions.

The Primary Immune Deficiency Treatment Consortium (PIDTC) is part of the Rare Diseases Clinical Research Network (RDCRN), which is funded by the National Institutes of Health (NIH) and led by the National Center for Advancing Translational Sciences (NCATS) through its Office of Rare Diseases Research (ORDR). PIDTC is funded under grant number U54AI082973 as a collaboration between NCATS and the National Institute of Allergy and Infectious Diseases (NIAID). All RDCRN consortia are supported by the network’s Data Management and Coordinating Center (DMCC) (U2CTR002818). Funding support for the DMCC is provided by NCATS and the National Institute of Neurological Disorders and Stroke (NINDS).