Primary immune deficiencies (PIDs) are a group of rare disorders of the immune system that result in increased susceptibility to infections, autoimmunity and malignancies. The most severe forms of PIDs are fatal, unless immune reconstitution is achieved by hematopoietic cell transplantation (HCT), enzyme replacement or gene therapy; other PIDs may be treated with biologics or immune modulation. Because of the many disease- and treatment-related variables that may affect outcome after HCT, both careful collection of data in Registries and multicenter collaboration are needed to facilitate analysis of outcome and development of prospective clinical trials. In 2009, the Primary Immune Deficiency Treatment Consortium (PIDTC) was established with the goal of building a North American collaboration to carry-out retrospective, cross-sectional and prospective studies that would define risk factors, long term outcomes and late effects in children with Severe Combined Immunodeficiency (SCID), Wiskott-Aldrich Syndrome (WAS), Chronic Granulomatous Disease (CGD) and auto-inflammatory diseases who receive HCT or other forms of definitive treatment. In April 2011 the first annual PIDCT Scientific Workshop was held in San Francisco followed by workshops in Boston (2012), Houston (2013), Seattle (2014), Montreal (2015), Los Angeles (2016) and Bethesda (2017). These workshops and Education Days provide a unique and critical forum to assemble experienced and young investigators from all major centers in North America involved in the treatment of these disorders, as well as biostatisticians and Patient Advocacy Groups and have been responsible for initiating the development of national and international collaborations. The proposed 8th PIDTC Scientific Workshop and Education Day in Philadelphia, PA (PIDTC2018) will focus on new timely topics to further advance the development of clinical trials aimed at improving treatment of PIDs, immunobiology of PIDs, development of safer pre-transplant conditioning regimens, autoinflammatory and immune dysregulatory diseases, gene therapy, and collaborations with Patient Advocacy Groups. The specific objectives of the workshops are: 1) To disseminate information on survival, clinical status, and immune function in patients with severe PIDs who have received HCT or other forms of treatment 2) To initiate data collection and analysis in other forms of severe PIDs that can be treated by HCT 3) To analyze relevance of biological markers that predict successful immune reconstitution following HCT 4) To discuss the results of novel approaches to HCT for PID that may minimize transplant-related toxicity while allowing robust and durable engraftment and immune reconstitution 5) To develop optimal treatment protocols for newborns with SCID. 6) To prompt development of clinical trials in the field of HCT for PIDs 7) To promote education of young investigators with a specific interest in treatment of PIDs 8) To actively recruit and train under-represented minority physicians to the field of PID 9) To increase synergy among Patient Advocacy Groups actively involved in PID awareness campaigns

Public Health Relevance

The Primary Immune Deficiency Treatment Consortium (PIDTC) was established in North America with the goal of building multicenter collaboration to improve outcome in primary immune deficiency (PID) patients who receive hematopoietic cell transplantation (HCT) or other forms of treatment. A vital activity of the PIDTC is the Annual Scientific Workshop at which members from the affiliated Centers convene to review progress on the PIDTC studies, to discuss and formulate plans for future investigations, and to jointly educate themselves to new and emerging scientific areas related to PIDs. The proposed 8th Annual PIDTC Scientific Workshop and Education Day in Philadelphia in May 2018 will assemble experienced and young investigators from all major centers in North America in addition to local and international experts involved in the study and treatment of PIDs as well as patient advocacy groups focused on patients with PID and their families. The major scientific theme will be methods to ?personalize transplants', by evaluating and employing novel pre-transplant conditioning methods to minimize toxicity while obtaining robust engraftment, in addition to in-depth presentations on the major PID.

National Institute of Health (NIH)
National Institute of Allergy and Infectious Diseases (NIAID)
Conference (R13)
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Special Emphasis Panel (ZAI1)
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Miller, Lara R
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University of California San Francisco
Schools of Medicine
San Francisco
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