Primary Immune Deficiency Treatment Consortium (PIDTC) report

Détails

ID Serval
serval:BIB_554AF113865A
Type
Article: article d'un périodique ou d'un magazine.
Sous-type
Synthèse (review): revue aussi complète que possible des connaissances sur un sujet, rédigée à partir de l'analyse exhaustive des travaux publiés.
Collection
Publications
Titre
Primary Immune Deficiency Treatment Consortium (PIDTC) report
Périodique
J Allergy Clin Immunol
Auteur⸱e⸱s
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., 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., workshop participants
ISSN
1097-6825 (Electronic)
ISSN-L
0091-6749
Statut éditorial
Publié
Date de publication
02/2014
Volume
133
Numéro
2
Pages
335-47
Langue
anglais
Notes
Griffith, Linda M
Cowan, Morton J
Notarangelo, Luigi D
Kohn, Donald B
Puck, Jennifer M
Pai, Sung-Yun
Ballard, Barbara
Bauer, Sarah C
Bleesing, Jack J H
Boyle, Marcia
Brower, Amy
Buckley, Rebecca H
van der Burg, Mirjam
Burroughs, Lauri M
Candotti, Fabio
Cant, Andrew J
Chatila, Talal
Cunningham-Rundles, Charlotte
Dinauer, Mary C
Dvorak, Christopher C
Filipovich, Alexandra H
Fleisher, Thomas A
Bobby Gaspar, Hubert
Gungor, Tayfun
Haddad, Elie
Hovermale, Emily
Huang, Faith
Hurley, Alan
Hurley, Mary
Iyengar, Sumathi
Kang, Elizabeth M
Logan, Brent R
Long-Boyle, Janel R
Malech, Harry L
McGhee, Sean A
Modell, Fred
Modell, Vicki
Ochs, Hans D
O'Reilly, Richard J
Parkman, Robertson
Rawlings, David J
Routes, John M
Shearer, William T
Small, Trudy N
Smith, Heather
Sullivan, Kathleen E
Szabolcs, Paul
Thrasher, Adrian
Torgerson, Troy R
Veys, Paul
Weinberg, Kenneth
Zuniga-Pflucker, Juan Carlos
eng
K12 HD068322/HD/NICHD NIH HHS/
R13 AI094943/AI/NIAID NIH HHS/
Z99 AI999999/Intramural NIH HHS/
P01 HL053749/HL/NHLBI NIH HHS/
U54 AI082973/AI/NIAID NIH HHS/
U54-AI082973/AI/NIAID NIH HHS/
P30 AI045008/AI/NIAID NIH HHS/
P01 AI097100/AI/NIAID NIH HHS/
R13-AI094943/AI/NIAID NIH HHS/
R18 AI048693/AI/NIAID NIH HHS/
U54 NS064808/NS/NINDS NIH HHS/
U54-NS064808/NS/NINDS NIH HHS/
Research Support, N.I.H., Extramural
Research Support, N.I.H., Intramural
Research Support, Non-U.S. Gov't
J Allergy Clin Immunol. 2014 Feb;133(2):335-47. doi: 10.1016/j.jaci.2013.07.052. Epub 2013 Oct 15.
Résumé
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.
Mots-clé
Hematopoietic Stem Cell Transplantation, Humans, *Immunologic Deficiency Syndromes/diagnosis/therapy, Infant, Newborn, Neonatal Screening, Pilot Projects, Societies, Scientific, 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
Pubmed
Création de la notice
01/11/2017 11:29
Dernière modification de la notice
20/08/2019 15:09
Données d'usage