The Administrative Unit (AU) of the Primary Immune Deficiency Treatment Consortium (PIDTC) is responsible forthe overall development and achievement of the scientific goals of the PIDTC. The goals of the AU are 1) to provide leadership in order to achieve the PIDTC objectives;2) to provide support and direction for each of the research protocols including development of protocols and consents, regulatory management, establishment and achievement of goals for case report form (CRF) completion, and maintaining datasets and statistical analysis of data for manuscript preparation;and 3) to facilitate development of future initiatives by the PIDTC in order to maintain its leadership role in the field of primary immune deficiency (PID). To this end, the AU has directed and initiated comprehensive studies of Severe Combined Immunodeficiency Disease, Chronic Granulomatous Disease and Wiskott Aldrich Syndrome in addition to organizing and leading the 33 PIDTC centers.
The Specific Aim ofthe AU is: To provide overall leadership to the PIDTC through organization of the Steering Committee, Scientific Planning Committee, Training committee. Pilot Project Committee, Protocol Planning Committees, the Publication Committee and the PAG Committee. Through this organization the AU will: a) Ensure that the protocols and consents are efficiently developed and implemented and that centers achieve their goals of enrollment and completion of CRFs in a timely manner;b) Maintain acfive collaboration with the Data Management and Coordinating Committee (DMCC) for implementation of protocols and CRFs, audifing of centers, import/export of data, and training of study personnel at PIDTC centers;c) Maintain active communications with PAGs, to ensure PAG representation on PIDTC committees, and to develop a productive scientific collaborafion with the PAGs in order to maximize our understanding of PIDs;d) Foster interest and participation of trainees and junior faculty in the PIDTC to generate the next generafion of leaders in the field;e) Ensure that results of scientific progress made by each of the research projects are effectively communicated to all PIDTC members, PAGs, and the rest ofthe scientific community;f) Promote the collaborafion of the PIDTC with colleagues outside of North America;and g) Continually promote the development of new ideas and inifiatives within the PIDTC scientific community, in particular, the design and implementation of prospective multicenter clinical trials that will optimize the care and definitive cure of PIDs.

Public Health Relevance

The ongoing and proposed PIDTC studies that will lead to identification of powerful biomarkers that predict outcome of hematopoietic cell transplantation for children with these rare life-threatening diseases and form the basis for future clinical trials to optimize definitive care could only be accomplished through the leadership and organizafion ofthe PIDTC and its Administrative Unit.

National Institute of Health (NIH)
National Institute of Allergy and Infectious Diseases (NIAID)
Specialized Center--Cooperative Agreements (U54)
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Special Emphasis Panel (ZTR1)
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University of California San Francisco
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Punwani, Divya; Kawahara, Misako; Yu, Jason et al. (2017) Lentivirus Mediated Correction of Artemis-Deficient Severe Combined Immunodeficiency. Hum Gene Ther 28:112-124
Heimall, Jennifer; Logan, Brent R; Cowan, Morton J et al. (2017) Immune reconstitution and survival of 100 SCID patients post-hematopoietic cell transplant: a PIDTC natural history study. Blood 130:2718-2727
Heimall, Jennifer; Puck, Jennifer; Buckley, Rebecca et al. (2017) Current Knowledge and Priorities for Future Research in Late Effects after Hematopoietic Stem Cell Transplantation (HCT) for Severe Combined Immunodeficiency Patients: A Consensus Statement from the Second Pediatric Blood and Marrow Transplant Consortium Biol Blood Marrow Transplant 23:379-387
Wahlstrom, Justin; Patel, Kiran; Eckhert, Erik et al. (2017) Transplacental maternal engraftment and posttransplantation graft-versus-host disease in children with severe combined immunodeficiency. J Allergy Clin Immunol 139:628-633.e10
Dorsey, Morna J; Dvorak, Christopher C; Cowan, Morton J et al. (2017) Treatment of infants identified as having severe combined immunodeficiency by means of newborn screening. J Allergy Clin Immunol 139:733-742
Dietz, Andrew C; Duncan, Christine N; Alter, Blanche P et al. (2017) The Second Pediatric Blood and Marrow Transplant Consortium International Consensus Conference on Late Effects after Pediatric Hematopoietic Cell Transplantation: Defining the Unique Late Effects of Children Undergoing Hematopoietic Cell Transplantation f Biol Blood Marrow Transplant 23:24-29
Kumánovics, Attila; Lee, Yu Nee; Close, Devin W et al. (2017) Estimated disease incidence of RAG1/2 mutations: A case report and querying the Exome Aggregation Consortium. J Allergy Clin Immunol 139:690-692.e3
Hoenig, Manfred; Lagresle-Peyrou, Chantal; Pannicke, Ulrich et al. (2017) Reticular dysgenesis: international survey on clinical presentation, transplantation, and outcome. Blood 129:2928-2938
Lee, Yu Nee; Frugoni, Francesco; Dobbs, Kerry et al. (2016) Characterization of T and B cell repertoire diversity in patients with RAG deficiency. Sci Immunol 1:
Griffith, Linda M; Cowan, Morton J; Notarangelo, Luigi D et al. (2016) Primary Immune Deficiency Treatment Consortium (PIDTC) update. J Allergy Clin Immunol 138:375-85

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