Primary Immune Deficiency Treatment Consortium (PIDTC) report Journal Article

Authors: Griffith, L. M.; Cowan, M. J.; Notarangelo, L. D.; Kohn, D. B.; Puck, J. M.; Pai, S. Y.; Ballard, B.; Bauer, S. C.; Bleesing, J. J. H.; Boyle, M.; Brower, A.; Buckley, R. H.; Van Der Burg, M.; Burroughs, L. M.; Candotti, F.; Cant, A. J.; Chatila, T.; Cunningham-Rundles, C.; Dinauer, M. C.; Dvorak, C. C.; Filipovich, A. H.; Fleisher, T. A.; Bobby Gaspar, H.; Gungor, T.; Haddad, E.; Hovermale, E.; Huang, F.; Hurley, A.; Hurley, M.; Iyengar, S.; Kang, E. M.; Logan, B. R.; Long-Boyle, J. R.; Malech, H. L.; McGhee, S. A.; Modell, F.; Modell, V.; Ochs, H. D.; O'Reilly, R. J.; Parkman, R.; Rawlings, D. J.; Routes, J. M.; Shearer, W. T.; Small, T. N.; Smith, H.; Sullivan, K. E.; Szabolcs, P.; Thrasher, A.; Torgerson, T. R.; Veys, P.; Weinberg, K.; Zuniga-Pflucker, J. C.
Article Title: Primary Immune Deficiency Treatment Consortium (PIDTC) report
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. © 2013 American Academy of Allergy, Asthma & Immunology.
Keywords: clinical trial; gene therapy; primary immunodeficiency; allogeneic hematopoietic cell transplantation
Journal Title: Journal of Allergy and Clinical Immunology
Volume: 133
Issue: 2
ISSN: 0091-6749
Publisher: Mosby Elsevier  
Date Published: 2014-02-01
Start Page: 335
End Page: 347
Language: English
DOI: 10.1016/j.jaci.2013.07.052
PROVIDER: scopus
PMCID: PMC3960312
PUBMED: 24139498
Notes: Export Date: 2 April 2014 -- CODEN: JACIB -- Source: Scopus
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MSK Authors
  1. Trudy Small
    215 Small
  2. Richard O'Reilly
    580 O'Reilly