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)
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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Long-Boyle, Janel R; Savic, Rada; Yan, Shirley et al. (2015) Population pharmacokinetics of busulfan in pediatric and young adult patients undergoing hematopoietic cell transplant: a model-based dosing algorithm for personalized therapy and implementation into routine clinical use. Ther Drug Monit 37:236-45
Haddad, Elie; Allakhverdi, Zoulfia; Griffith, Linda M et al. (2014) Survey on retransplantation criteria for patients with severe combined immunodeficiency. J Allergy Clin Immunol 133:597-9
Shearer, William T; Dunn, Elizabeth; Notarangelo, Luigi D et al. (2014) Establishing diagnostic criteria for severe combined immunodeficiency disease (SCID), leaky SCID, and Omenn syndrome: the Primary Immune Deficiency Treatment Consortium experience. J Allergy Clin Immunol 133:1092-8
Dvorak, Christopher C; Hassan, Amel; Slatter, Mary A et al. (2014) Comparison of outcomes of hematopoietic stem cell transplantation without chemotherapy conditioning by using matched sibling and unrelated donors for treatment of severe combined immunodeficiency. J Allergy Clin Immunol 134:935-943.e15
Lee, Yu Nee; Frugoni, Francesco; Dobbs, Kerry et al. (2014) A systematic analysis of recombination activity and genotype-phenotype correlation in human recombination-activating gene 1 deficiency. J Allergy Clin Immunol 133:1099-108
Pai, Sung-Yun; Cowan, Morton J (2014) Stem cell transplantation for primary immunodeficiency diseases: the North American experience. Curr Opin Allergy Clin Immunol 14:521-6
Griffith, Linda M; Cowan, Morton J; Notarangelo, Luigi D et al. (2014) Primary Immune Deficiency Treatment Consortium (PIDTC) report. J Allergy Clin Immunol 133:335-47
Medical Advisory Committee of the Immune Deficiency Foundation; Shearer, William T; Fleisher, Thomas A et al. (2014) Recommendations for live viral and bacterial vaccines in immunodeficient patients and their close contacts. J Allergy Clin Immunol 133:961-6
Chinen, Javier; Notarangelo, Luigi D; Shearer, William T (2014) Advances in basic and clinical immunology in 2013. J Allergy Clin Immunol 133:967-76
Pai, Sung-Yun; Logan, Brent R; Griffith, Linda M et al. (2014) Transplantation outcomes for severe combined immunodeficiency, 2000-2009. N Engl J Med 371:434-46

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