Primary Immune Deficiency Treatment Consortium (PIDTC) report - 30/01/14
Doi : 10.1016/j.jaci.2013.07.052
Linda M. Griffith, MD, PhD a, ⁎ 
, Morton J. Cowan, MD b, Luigi D. Notarangelo, MD c, Donald B. Kohn, MD d, Jennifer M. Puck, MD b, e, Sung-Yun Pai, MD f, Barbara Ballard g, Sarah C. Bauer, MD h, Jack J.H. Bleesing, MD, PhD i, Marcia Boyle g, Amy Brower, PhD j, Rebecca H. Buckley, MD k, Mirjam van der Burg, PhD l, Lauri M. Burroughs, MD m, Fabio Candotti, MD n, Andrew J. Cant, MD o, Talal Chatila, MD p, Charlotte Cunningham-Rundles, MD, PhD q, Mary C. Dinauer, MD, PhD r, Christopher C. Dvorak, MD b, Alexandra H. Filipovich, MD s, Thomas A. Fleisher, MD t, Hubert Bobby Gaspar, MD, PhD u, Tayfun Gungor, MD v, Elie Haddad, MD, PhD w, Emily Hovermale g, Faith Huang, MD x, Alan Hurley y, Mary Hurley y, Sumathi Iyengar, MD z, Elizabeth M. Kang, MD aa, Brent R. Logan, PhD bb, Janel R. Long-Boyle, PharmD, PhD cc, Harry L. Malech, MD aa, Sean A. McGhee, MD dd, Fred Modell ee, Vicki Modell ee, Hans D. Ochs, DM ff, Richard J. O'Reilly, MD gg, Robertson Parkman, MD hh, David J. Rawlings, MD ii, John M. Routes, MD jj, William T. Shearer, MD, PhD kk, Trudy N. Small, MD ll, †, Heather Smith mm, Kathleen E. Sullivan, MD, PhD nn, Paul Szabolcs, MD oo, Adrian Thrasher, MD pp, Troy R. Torgerson, MD, PhD qq, Paul Veys, MD rr, Kenneth Weinberg, MD ss, Juan Carlos Zuniga-Pflucker, PhD tt
on behalf of the
, Morton J. Cowan, MD b, Luigi D. Notarangelo, MD c, Donald B. Kohn, MD d, Jennifer M. Puck, MD b, e, Sung-Yun Pai, MD f, Barbara Ballard g, Sarah C. Bauer, MD h, Jack J.H. Bleesing, MD, PhD i, Marcia Boyle g, Amy Brower, PhD j, Rebecca H. Buckley, MD k, Mirjam van der Burg, PhD l, Lauri M. Burroughs, MD m, Fabio Candotti, MD n, Andrew J. Cant, MD o, Talal Chatila, MD p, Charlotte Cunningham-Rundles, MD, PhD q, Mary C. Dinauer, MD, PhD r, Christopher C. Dvorak, MD b, Alexandra H. Filipovich, MD s, Thomas A. Fleisher, MD t, Hubert Bobby Gaspar, MD, PhD u, Tayfun Gungor, MD v, Elie Haddad, MD, PhD w, Emily Hovermale g, Faith Huang, MD x, Alan Hurley y, Mary Hurley y, Sumathi Iyengar, MD z, Elizabeth M. Kang, MD aa, Brent R. Logan, PhD bb, Janel R. Long-Boyle, PharmD, PhD cc, Harry L. Malech, MD aa, Sean A. McGhee, MD dd, Fred Modell ee, Vicki Modell ee, Hans D. Ochs, DM ff, Richard J. O'Reilly, MD gg, Robertson Parkman, MD hh, David J. Rawlings, MD ii, John M. Routes, MD jj, William T. Shearer, MD, PhD kk, Trudy N. Small, MD ll, †, Heather Smith mm, Kathleen E. Sullivan, MD, PhD nn, Paul Szabolcs, MD oo, Adrian Thrasher, MD pp, Troy R. Torgerson, MD, PhD qq, Paul Veys, MD rr, Kenneth Weinberg, MD ss, Juan Carlos Zuniga-Pflucker, PhD tton behalf of the
workshop participants
a Division of Allergy, Immunology and Transplantation, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Md
b Division of Allergy/Immunology and Blood and Marrow Transplantation, Department of Pediatrics and UCSF Benioff Children’s Hospital, University of California San Francisco, San Francisco, Calif
c Division of Immunology, the Manton Center for Orphan Disease Research, Children’s Hospital, and Harvard Stem Cell Institute, Harvard Medical School, Boston, Mass
d Departments of Microbiology, Immunology and Molecular Genetics, University of California Los Angeles, Los Angeles, Calif
e Institute for Human Genetics, University of California San Francisco, San Francisco, Calif
f Pediatric Hematology/Oncology, Children’s Hospital, Harvard Medical School, Boston, Mass
g Immune Deficiency Foundation, Towson, Md
h Developmental and Behavioral Pediatrics, Lurie Children’s Hospital of Chicago, Northwestern Feinberg School of Medicine, Chicago, Ill
i Department of Pediatrics, Cincinnati Children’s Hospital Medical Center, Cincinnati, Ohio
j Newborn Screening Translational Research Network, American College of Medical Genetics and Genomics, Bethesda, Md
k Pediatric Allergy and Immunology, Duke University School of Medicine, Durham, NC
l Molecular Immunology, Erasmus Medical Center, Rotterdam, The Netherlands
m Pediatric Hematology/Oncology, Fred Hutchinson Cancer Research Center, University of Washington School of Medicine, Seattle, Wash
n Genetics & Molecular Biology Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, Md
o Pediatric Immunology and Infectious Diseases and Pediatric Bone Marrow Transplant, Newcastle General Hospital, Newcastle upon Tyne, United Kingdom
p Pediatric Allergy/Immunology, Children’s Hospital, Harvard Medical School, Boston, Mass
q Pediatric Clinical Immunology, Mount Sinai School of Medicine, New York, NY
r Pediatric Hematology/Oncology, Washington University School of Medicine, St Louis, Mo
s Pediatric Clinical Immunology, Division of Hematology/Oncology, Cincinnati Children’s Hospital Medical Center, Cincinnati, Ohio
t Department of Laboratory Medicine, Clinical Center, National Institutes of Health, Bethesda, Md
u Pediatric Immunology, Center for Immunodeficiency, Institute of Child Health, Great Ormond Street Hospital, University College London, London, United Kingdom
v Pediatric Immunology and Blood and Marrow Transplantation, Children’s Hospital Zurich, University of Zurich, Zurich, Switzerland
w Pediatric Immunology, Mother and Child Ste-Justine Hospital, Montreal, Quebec, Canada
x Pediatric Allergy/Immunology, Mount Sinai Medical Center, New York, NY
y Chronic Granulomatous Disease Association, San Marino, Calif
z Wiskott-Aldrich Foundation, Smyrna, Ga
aa Laboratory of Host Defenses, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Md
bb Center for International Blood and Marrow Transplant Research and Division of Biostatistics, Medical College of Wisconsin, Milwaukee, Wis
cc Department of Clinical Pharmacy, School of Pharmacy, University of California, San Francisco, Calif
dd Pediatric Allergy/Immunology, Lucile Packard Children’s Hospital, Stanford University Medical Center, Stanford, Calif
ee Jeffrey Modell Foundation, New York, NY
ff Center for Immunity and Immunotherapy, Seattle Children’s Hospital Research Institute, University of Washington School of Medicine, Seattle, Wash
gg Pediatrics and Immunology, Memorial Sloan Kettering Cancer Center, New York, NY
hh Division of Research Immunology/B.M.T., Children’s Hospital Los Angeles, Los Angeles, Calif
ii Pediatric Immunology, Seattle Children’s Research Institute, University of Washington School of Medicine, Seattle, Wash
jj Pediatric Allergy and Clinical Immunology, Children’s Hospital of Wisconsin, Medical College of Wisconsin, Milwaukee, Wis
kk Pediatric Allergy & Immunology, Texas Children's Hospital, Baylor College of Medicine, Houston, Tex
ll Pediatric Bone Marrow Transplant Service, Memorial Sloan Kettering Cancer Center, New York, NY
mm SCID Angels for Life Foundation, Lakeland, Fla
nn Pediatric Immunology, Children’s Hospital of Philadelphia, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pa
oo Bone Marrow Transplantation and Cellular Therapies, Children’s Hospital of Pittsburgh, University of Pittsburgh Medical Center, Pittsburgh, Pa
pp Pediatric Immunology, Center for Immunodeficiency, Institute of Child Health, Great Ormond Street Hospital, University College London, London, United Kingdom
qq Pediatric Rheumatology, Seattle Children’s Research Institute, University of Washington School of Medicine, Seattle, Wash
rr Blood and Marrow Transplantation, Institute of Child Health, Great Ormond Street Hospital, London, United Kingdom
ss Pediatric Stem Cell Transplantation and Hematology/Oncology, Lucile Packard Children’s Hospital, Stanford University School of Medicine, Stanford, Calif
tt Department of Immunology, University of Toronto, Toronto, Ontario, Canada
∗Corresponding author: Linda M. Griffith, MD, PhD, Division of Allergy, Immunology and Transplantation, National Institute of Allergy and Infectious Diseases, National Institutes of Health, 6610 Rockledge Dr, Rm 6716, Bethesda, MD 20892-6610.
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.
Le texte complet de cet article est disponible en PDF.Key words : Allogeneic hematopoietic cell transplantation, gene therapy, primary immunodeficiency, clinical trial
Abbreviations used : ADA, CGD, CIBMTR, EBMT, ESID, GT, GVHD, HCT, IEWP, MAC, MUD, NBS, NIAID, NIH, NK, PID, PIDTC, RIC, SCETIDE, SCID, USIDNET, WAS
Plan
| 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. |
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| The opinions expressed are those of the authors and do not represent the position of the National Institute of Allergy and Infectious Diseases, the National Human Genome Research Institute, the Office of Rare Diseases Research, the National Center for Advancing Translational Sciences, the National Institutes of Health, or the US government. |
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| Disclosure of potential conflict of interest: M. J. Cowan has received a grant from the National Institutes of Health (NIH)/National Institute of Allergy and Infectious Diseases (NIAID); has received travel support from the NIH/NIAID; has consultant arrangements with Bluebird Bio; and is on the scientific advisory board for and receives stock options from Exogen Bio. L. D. Notarangelo has received grants from the NIH and the March of Dimes; is a board member for the Immune Disease Institute and the University Pediatric Hospital “Meyer” in Florence, Italy; and has received royalties from UpToDate. D. B. Kohn has received a grant from the Primary Immune Deficiency Treatment Consortium. J. M. Puck has received grants from the Jeffrey Modell Foundation and the NIH. S.-Y. Pai has received a grant from the Venture Philanthropy Network. M. Boyle has received program funding through the Immune Deficiency Foundation from Baxter Healthcare, CSL Behring, Grifols, Biotest Pharmaceuticals, IgG America/ASD, Healthcare/US Bioservices, Octapharma, Kedrion Biopharma, Vidara Therapeutics, and Walgreens–IG Therapy Program. R. H. Buckley has received grants from the NIAID and the NIH, has received travel support from the University of California San Francisco, is employed by Duke University, receives royalties from Paul Immunology (Lippincott Williams & Wilkins), has stock/stock options with TIAA-CREF, and has received travel support from the Clinical Immunology Society. L. M. Burroughs has received grants from the National Heart, Lung, and Blood Institute (NHLBI) and MEDAC. A. J. Cant is a board member for the Bubble Foundation UK, is employed by Newcastle University, and receives royalties from Rook's Textbook of Dermatology (Wiley-Blackwell). M. C. Dinauer has received a grant and travel support from the Primary Immune Deficiency Treatment Consortium. T. Gungoer has received travel support from the Jeffrey Modell Foundation. E. Hovermale has received program funding through the Immune Deficiency Foundation, from Baxter Healthcare, CSL Behring, Grifols, Biotest Pharmaceuticals, IgG America/ASD Healthcare/US Bioservices, Octapharma, Kedrion Biopharma, Vidara Therapeutics, and Walgreens–IG therapy program. S. Iyengar has received travel support from the Primary Immune Deficiency Treatment Consortium. B. R. Logan has received grants from the NIH. S. A. McGhee has received payment for lectures from Baxter. R. O'Reilly has received payment for expert testimony on behalf of Miltenyi Biotech and has received grants from the National Cancer Institute and the NIAID. R. Parkman has received grants and travel support from the NIH. J. M. Routes has received a grant and travel support from the Primary Immune Deficiency Treatment Consortium. H. Smith has received travel support from the Primary Immune Deficiency Treatment Consortium and PerkinElmer and has received a grant from Grifols Pharmaceutical. K. E. Sullivan has received grants from the Primary Immune Deficiency Treatment Consortium, Baxter, and CSL Behring; has consultant arrangements with the Immune Deficiency Foundation and Up To Date; and is employed by UpToDate. P. Szabolcs has received travel support from the Primary Immune Deficiency Treatment Consortium. A. Thrasher has received grants from the Wellcome Trust. T. R. Torgerson has consultant arrangements with Baxter Biosciences; has received grants from Baxter Biosciences and CSL Behring; has received payment for lectures from Baxter Biosciences; and has received payment for development of educational presentations from Baxter Biosciences. P. Veys has received travel support from the Primary Immune Deficiency Treatment Consortium. The rest of the authors declare that they have no relevant conflicts of interest. |
© 2013
Publié par Elsevier Masson SAS.
Vol 133 - N° 2
P. 335 - février 2014 Retour au numéro
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