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Primary Immune Deficiency Treatment Consortium (PIDTC) report

Lookup NU author(s): Professor Andrew Cant

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Abstract

The Primary Immune Deficiency Treatment Consortium (PIDTC) is a network of 33 centers in North America that study the treatment of rare and severe primary immunodeficiency diseases. Current protocols address the natural history of patients treated for severe combined immunodeficiency (SCID), Wiskott-Aldrich syndrome, and chronic granulomatous disease through retrospective, prospective, and cross-sectional studies. The PIDTC additionally seeks to encourage training of junior investigators, establish partnerships with European and other International colleagues, work with patient advocacy groups to promote community awareness, and conduct pilot demonstration projects. Future goals include the conduct of prospective treatment studies to determine optimal therapies for primary immunodeficiency diseases. To date, the PIDTC has funded 2 pilot projects: newborn screening for SCID in Navajo Native Americans and B-cell reconstitution in patients with SCID after hematopoietic stem cell transplantation. Ten junior investigators have received grant awards. The PIDTC Annual Scientific Workshop has brought together consortium members, outside speakers, patient advocacy groups, and young investigators and trainees to report progress of the protocols and discuss common interests and goals, including new scientific developments and future directions of clinical research. Here we report the progress of the PIDTC to date, highlights of the first 2 PIDTC workshops, and consideration of future consortium objectives.

Publication metadata

Author(s): Griffith LM, Cowan MJ, Notarangelo LD, Kohn DB, Puck JM, Pai SY, Ballard B, Bauer SC, Bleesing JJH, Boyle M, Brower A, Buckley RH, van der Burg M, Burroughs LM, Candotti F, Cant AJ, Chatila T, Cunningham-Rundles C, Dinauer MC, Dvorak CC, Filipovich AH, Fleisher TA, Gaspar HB, Gungor T, Haddad E, Hovermale E, Huang F, Hurley A, Hurley M, Iyengar S, Kang EM, Logan BR, Long-Boyle JR, Malech HL, McGhee SA, Modell F, Modell V, Ochs HD, O'Reilly RJ, Parkman R, Rawlings DJ, Routes JM, Shearer WT, Small TN, Smith H, Sullivan KE, Szabolcs P, Thrasher A, Torgerson TR, Veys P, Weinberg K, Zuniga-Pflucker JC, Workshop Participants

Publication type: Article

Publication status: Published

Journal: Journal of Allergy and Clinical Immunology

Year: 2014

Volume: 133

Issue: 2

Pages: 335-347

Print publication date: 01/02/2014

ISSN (print): 0091-6749

ISSN (electronic): 1097-6825

Publisher: Mosby, Inc.

URL: http://dx.doi.org/10.1016/j.jaci.2013.07.052

DOI: 10.1016/j.jaci.2013.07.052

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Funding

Funder referenceFunder name
Baxter International, Deerfield, Ill
Division of Allergy, Immunology and Transplantation, National Institute of Allergy and Infectious Diseases
Robert A. Good Immunology Society, St Petersburg, Fla
Talecris Biotherapeutics, Research Triangle Park, NC
Children's Hospital Translational Research Program, Children's Hospital, Boston, Mass
CSL Behring, King of Prussia, Pa
Immune Deficiency Foundation, Towson, Md
Jeffrey Modell Foundation, New York, NY
Manton Center for Orphan Disease Research
National Human Genome Research Institute
National Institute of Allergy and Infectious Diseases
Sigma-Tau Pharmaceuticals, Gaithersburg, Md
R13-AI094943Office of Rare Diseases Research, National Center for Advancing Translational Sciences, National Institutes of Health, Bethesda, Md
U54-AI082973Office of Rare Diseases Research, National Center for Advancing Translational Sciences, National Institutes of Health, Bethesda, Md
U54-NS064808Office of Rare Diseases Research, National Center for Advancing Translational Sciences, National Institutes of Health, Bethesda, Md

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