TY - JOUR
T1 - Primary Immune Deficiency Treatment Consortium (PIDTC) report
AU - Griffith, Linda M.
AU - Cowan, Morton J.
AU - Notarangelo, Luigi D.
AU - Kohn, Donald B.
AU - Puck, Jennifer M.
AU - Pai, Sung Yun
AU - Ballard, Barbara
AU - Bauer, Sarah C.
AU - Bleesing, Jack J.H.
AU - Boyle, Marcia
AU - Brower, Amy
AU - Buckley, Rebecca H.
AU - Van Der Burg, Mirjam
AU - Burroughs, Lauri M.
AU - Candotti, Fabio
AU - Cant, Andrew J.
AU - Chatila, Talal
AU - Cunningham-Rundles, Charlotte
AU - Dinauer, Mary C.
AU - Dvorak, Christopher C.
AU - Filipovich, Alexandra H.
AU - Fleisher, Thomas A.
AU - Bobby Gaspar, Hubert
AU - Gungor, Tayfun
AU - Haddad, Elie
AU - Hovermale, Emily
AU - Huang, Faith
AU - Hurley, Alan
AU - Hurley, Mary
AU - Iyengar, Sumathi
AU - Kang, Elizabeth M.
AU - Logan, Brent R.
AU - Long-Boyle, Janel R.
AU - Malech, Harry L.
AU - McGhee, Sean A.
AU - Modell, Fred
AU - Modell, Vicki
AU - Ochs, Hans D.
AU - O'Reilly, Richard J.
AU - Parkman, Robertson
AU - Rawlings, David J.
AU - Routes, John M.
AU - Shearer, William T.
AU - Small, Trudy N.
AU - Smith, Heather
AU - Sullivan, Kathleen E.
AU - Szabolcs, Paul
AU - Thrasher, Adrian
AU - Torgerson, Troy R.
AU - Veys, Paul
AU - Weinberg, Kenneth
AU - Zuniga-Pflucker, Juan Carlos
N1 - Funding Information:
In 2008, North American experts in the diagnosis and treatment of primary immunodeficiency (PID) diseases met at the National Institutes of Health (NIH) in Bethesda, Maryland, to discuss opportunities for collaboration, the feasibility and prioritization of clinical research questions, and the scope of expertise that would be needed to establish an effective multicenter consortium. 1 Historically, procedures for hematopoietic cell transplantation (HCT) have differed according to local practice, with relatively few patients treated at each center. Therefore multicenter longitudinal retrospective, prospective, and cross-sectional protocols were designed to capture, for the first time in a common comprehensive database, survival and other outcomes after HCT, gene therapy (GT), or enzyme replacement therapy for PIDs performed in North America. The results of these studies were envisioned to become a resource and foundation for the design of future prospective interventional studies. The group has worked together successfully to establish the Primary Immune Deficiency Treatment Consortium (PIDTC), which currently includes 33 centers in North America with expertise in HCT for PIDs and is sponsored by the National Institute of Allergy and Infectious Diseases (NIAID) and the Office of Rare Diseases Research, National Center for Advancing Translational Sciences, NIH. PIDTC clinical studies in severe combined immunodeficiency (SCID) are now well underway, and protocols in Wiskott-Aldrich syndrome (WAS) and chronic granulomatous disease (CGD) are expected to open in 2013 ( Table I ).
Funding Information:
Supported by the Division of Allergy, Immunology and Transplantation, National Institute of Allergy and Infectious Diseases ; the Intramural Research Programs of the National Human Genome Research Institute and the National Institute of Allergy and Infectious Diseases ; and the Office of Rare Diseases Research, National Center for Advancing Translational Sciences, National Institutes of Health, Bethesda, Md ; U54-AI082973 (primary investigator: M. J. Cowan); U54-NS064808 (primary investigator: J. P. Krischer); R13-AI094943 (primary investigators: M. J. Cowan and L. D. Notarangelo). Workshops in April 2011 (San Francisco, Calif) and April 2012 (Boston, Mass) were also supported in part by the Immune Deficiency Foundation, Towson, Md ; the Jeffrey Modell Foundation, New York, NY ; the Robert A. Good Immunology Society, St Petersburg, Fla ; the Manton Center for Orphan Disease Research and the Children’s Hospital Translational Research Program, Children’s Hospital, Boston, Mass ; Baxter International, Deerfield, Ill ; CSL Behring, King of Prussia, Pa ; Sigma-Tau Pharmaceuticals, Gaithersburg, Md ; and Talecris Biotherapeutics, Research Triangle Park, NC .
PY - 2014/2
Y1 - 2014/2
N2 - 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.
AB - 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.
KW - Allogeneic hematopoietic cell transplantation
KW - clinical trial
KW - gene therapy
KW - primary immunodeficiency
UR - http://www.scopus.com/inward/record.url?scp=84895059316&partnerID=8YFLogxK
U2 - 10.1016/j.jaci.2013.07.052
DO - 10.1016/j.jaci.2013.07.052
M3 - Article
C2 - 24139498
AN - SCOPUS:84895059316
SN - 0091-6749
VL - 133
SP - 335-347.e11
JO - Journal of Allergy and Clinical Immunology
JF - Journal of Allergy and Clinical Immunology
IS - 2
ER -