Primary Immune Deficiency Treatment Consortium (PIDTC) report

Linda M. Griffith*, Morton J. Cowan, Luigi D. Notarangelo, Donald B. Kohn, Jennifer M. Puck, Sung Yun Pai, Barbara Ballard, Sarah C. Bauer, Jack J.H. Bleesing, Marcia Boyle, Amy Brower, Rebecca H. Buckley, Mirjam Van Der Burg, Lauri M. Burroughs, Fabio Candotti, Andrew J. Cant, Talal Chatila, Charlotte Cunningham-Rundles, Mary C. Dinauer, Christopher C. DvorakAlexandra H. Filipovich, Thomas A. Fleisher, Hubert Bobby Gaspar, Tayfun Gungor, Elie Haddad, Emily Hovermale, Faith Huang, Alan Hurley, Mary Hurley, Sumathi Iyengar, Elizabeth M. Kang, Brent R. Logan, Janel R. Long-Boyle, Harry L. Malech, Sean A. McGhee, Fred Modell, Vicki Modell, Hans D. Ochs, Richard J. O'Reilly, Robertson Parkman, David J. Rawlings, John M. Routes, William T. Shearer, Trudy N. Small, Heather Smith, Kathleen E. Sullivan, Paul Szabolcs, Adrian Thrasher, Troy R. Torgerson, Paul Veys, Kenneth Weinberg, Juan Carlos Zuniga-Pflucker

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

41 Scopus citations

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.

Original languageEnglish (US)
Pages (from-to)335-347.e11
JournalJournal of Allergy and Clinical Immunology
Volume133
Issue number2
DOIs
StatePublished - Feb 2014

Keywords

  • Allogeneic hematopoietic cell transplantation
  • clinical trial
  • gene therapy
  • primary immunodeficiency

ASJC Scopus subject areas

  • Immunology and Allergy
  • Immunology

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