J Allergy Clin Immunol. 2013 Oct 15. pii: S0091-6749(13)01369-9. doi: 10.1016/j.jaci.2013.07.052. [Epub ahead of print]
Primary Immune Deficiency Treatment Consortium (PIDTC) report.
Griffith LM, Cowan MJ, Notarangelo LD, Kohn DB, Puck JM, Pai SY, Ballard B, Bauer SC, Bleesing JJ, 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, Bobby Gaspar H, 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; on behalf of the workshop participants.
Source
Division of Allergy, Immunology and Transplantation, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Md. Electronic address: LGriffith@niaid.nih.gov .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.
Copyright © 2013 American Academy of Allergy, Asthma & Immunology. Published by Mosby, Inc. All rights reserved.
Copyright © 2013 American Academy of Allergy, Asthma & Immunology. Published by Mosby, Inc. All rights reserved.
KEYWORDS:
ADA, Adenosine deaminase, Allogeneic hematopoietic cell transplantation, CGD, CIBMTR, Center for International Blood and Marrow Transplant Research, Chronic granulomatous disease, EBMT, ESID, European Group for Blood and Marrow Transplantation, European Society for Immunodeficiencies, GT, GVHD, Gene therapy, Graft-versus-host disease, HCT, Hematopoietic cell transplantation, IEWP, Inborn Errors Working Party, MAC, MUD, Matched unrelated donor, Myeloablative conditioning, NBS, NIAID, NIH, NK, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Natural killer, Newborn screening for SCID, PID, PIDTC, Primary Immune Deficiency Treatment Consortium, Primary immunodeficiency, RIC, Reduced-intensity conditioning, SCETIDE, SCID, Severe combined immunodeficiency, Stem Cell Transplantation for Immunodeficiencies in Europe, USIDNET, United States Immunodeficiency Network, WAS, Wiskott-Aldrich syndrome, clinical trial, gene therapy, primary immunodeficiency- PMID:
- 24139498
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