Primary Immune Deficiency Treatment Consortium (PIDTC) update - 04/08/16
Doi : 10.1016/j.jaci.2016.01.051
Linda M. Griffith, MD, MHS, PhD a, ⁎ 
, Morton J. Cowan, MD b, Luigi D. Notarangelo, MD c, Donald B. Kohn, MD d, Jennifer M. Puck, MD b, William T. Shearer, MD, PhD e, Lauri M. Burroughs, MD f, Troy R. Torgerson, MD, PhD g, Hélène Decaluwe, MD, PhD h, Elie Haddad, MD, PhD h
on behalf of the
, Morton J. Cowan, MD b, Luigi D. Notarangelo, MD c, Donald B. Kohn, MD d, Jennifer M. Puck, MD b, William T. Shearer, MD, PhD e, Lauri M. Burroughs, MD f, Troy R. Torgerson, MD, PhD g, Hélène Decaluwe, MD, PhD h, Elie Haddad, MD, PhD hon 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, Children's Hospital, and Harvard Stem Cell Institute, Harvard Medical School, Boston, Mass
d Departments of Microbiology, Immunology & Molecular Genetics and Pediatrics, University of California Los Angeles, Los Angeles, Calif
e Pediatric Allergy & Immunology, Texas Children's Hospital, Baylor College of Medicine, Houston, Tex
f Pediatric Hematology/Oncology, Fred Hutchinson Cancer Research Center, University of Washington School of Medicine, Seattle, Wash
g Pediatric Rheumatology, Seattle Children's Research Institute, University of Washington School of Medicine, Seattle, Wash
h Pediatric Immunology and Pediatrics, Mother and Child Ste-Justine Hospital, University of Montreal, Montreal, Quebec, Canada
∗Corresponding author: Linda M. Griffith, MD, MHS, PhD, Division of Allergy, Immunology and Transplantation, National Institute of Allergy and Infectious Diseases, National Institutes of Health, 5601 Fishers Ln, Rm 7D49, Bethesda, MD 20892-9828.Division of Allergy, Immunology and TransplantationNational Institute of Allergy and Infectious DiseasesNational Institutes of Health5601 Fishers LnRm 7D49BethesdaMD20892-9828
Abstract |
The Primary Immune Deficiency Treatment Consortium (PIDTC) is a collaboration of 41 North American centers studying therapy for rare primary immune deficiency diseases (PIDs), including severe combined immune deficiency (SCID), Wiskott-Aldrich syndrome (WAS), and chronic granulomatous disease (CGD). An additional 3 European centers have partnered with the PIDTC to study CGD. Natural history protocols of the PIDTC analyze outcomes of treatment for rare PIDs in multicenter longitudinal retrospective, prospective, and cross-sectional studies. Since 2009, participating centers have enrolled more than 800 subjects on PIDTC protocols for SCID, and enrollment in the studies on WAS and CGD is underway. Four pilot projects have been funded, and 12 junior investigators have received fellowship awards. Important publications of the consortium describe the outcomes of hematopoietic cell transplantation for SCID during 2000-2009, diagnostic criteria for SCID, and the pilot project of newborn screening for SCID in the Navajo Nation. The PIDTC Annual Scientific Workshops provide an opportunity to strengthen collaborations with junior investigators, patient advocacy groups, and international colleagues. Funded by the National Institute of Allergy and Infectious Diseases and the Office of Rare Diseases Research, National Center for Advancing Translational Sciences, the PIDTC has recently received renewal for another 5 years. Here we review accomplishments of the group, projects underway, highlights of recent workshops, and challenges for the future.
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, GT, HCT, IEWP, MSD, MUD, NBS, NCATS, NIAID, NIH, ORDR, OS, PAG, PID, PIDTC, RD, SCID, TREC, WAS, X-SCID
Plan
| Supported by the Division of Allergy, Immunology and Transplantation, National Institute of Allergy and Infectious Diseases (NIAID); the Intramural Research Program of the NIAID; and the Office of Rare Diseases Research, National Center for Advancing Translational Sciences, National Institutes of Health, Bethesda, Md: U54-AI082973 (PI: M. J. Cowan), U54-NS064808 and U01-TR001263 (PI: J. P. Krischer), and R13-AI094943 (PIs: M. J. Cowan, L. D. Notarangelo). The workshops were also supported in part by the Immune Deficiency Foundation, Towson, Md; the Jeffrey Modell Foundation, New York, NY; the David Center, Texas Children's Hospital, Houston, Tex; the John P. McGovern Foundation, Houston, Tex; Seattle Cancer Care Alliance, Seattle, Wash; Seattle Children's Research Institute and Seattle Children's Hospital, Seattle, Wash; AbbVie, St-Laurent, Quebec, Canada; ADMA Biologics, Ramsey, NJ; Baxalta, Deerfield, Ill; Bristol-Myers Squibb Canada, St-Laurent, Quebec, Canada; CSL Behring, King of Prussia, Pa; CSL Behring, Ottawa, Ontario, Canada; GRIFOLS Canada, Mississauga, Ontario, Canada; Horizon Pharma USA, Deerfield, Ill; Miltenyi Biotec, Auburn, Calif; Octapharma Canada, Toronto, Ontario, Canada; Otsuka Canada Pharmaceutical, St-Laurent, Quebec, Canada; and Sigma-Tau Pharmaceuticals, Gaithersburg, Md. The Primary Immune Deficiency Treatment Consortium (PIDTC) is a part of the Rare Diseases Clinical Research Network (RDCRN) of the Office of Rare Diseases Research (ORDR), National Center for Advancing Translational Sciences (NCATS), and is sponsored by the ORDR, NCATS, and the Division of Allergy Immunology and Transplantation (DAIT), NIAID. The opinions expressed are those of the authors and do not represent the position of the NIAID, the ORDR, the NCATS, the NIH, or the US Government. |
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| Disclosure of potential conflict of interest: M. J. Cowan has received grants from the National Institute of Allergy and Infectious Diseases (NIAID; U54-AI082973 and R13-AI094943) and the California Institute of Regenerative Medicine (DR2-05365, TR3-05535, and LSP1-08363); has received travel support from the NIAID (U54-AI082973); is on the Scientific Advisory Board for Homology; has received royalties from UpToDate; has stock/stock options in Exogen and Homology; and is on the Data Safety Monitoring Board for Bluebird Bio. L. D. Notarangelo has received grants from the NIAID, National Institutes of Health (NIH), and March of Dimes; is a member of the editorial board for the Journal of Clinical Immunology; is a member of the Data Safety Monitoring Board for Novimmune; has consultant arrangements with Sigma-Tau; is employed by Boston Children's Hospital; and has received royalties from UpToDate. D. B. Kohn has received a grant and travel support from the NIH Primary Immune Deficiency Treatment Consortium. J. M. Puck has received grants from the NIH, Jeffrey Modell Foundation, and California Institute for Regenerative Medicine; has consultant arrangements with the California Department of Public Health; has received payment for lectures from AAI, the Clinical Immunology Society, UC San Diego, IPOPI, JMF, Mt Sinai NY, and Children's Mercy Hospital Kansas; has received royalties from Oxford University Press and UpToDate; and has received travel support from Baxter/Baxalta, and her spouse is employed by Invitae. W. T. Shearer has received grants from the NIAID and the National Heart, Lung, and Blood Institute (NHLBI) and has received travel support from the Primary Immune Deficiency Treatment Consortium. L. M. Burroughs has received a grant from the NIH (P01 HL122173-01 [Storb]). T. R. Torgerson has consultant arrangements with Baxalta Biosciences, CSL Behring, and ADMA Biosciences; has received grants from Baxalta Biosciences, CSL Behring, and the NIH; and has received payment for development of educational presentations from Baxalta Biosciences, CSL Behring, Questcor Pharmaceuticals, and the Robert Wood Johnson Foundation. H. Decaluwe has received a grant and travel support from the NIAID and the Offices of the Rare Diseases Research, NIH. E. Haddad has received grants from Baxalta, CSL Behring, AbbVie, Horizon Pharma USA, ADMA Biologics, GRIFOLS Canada, Octapharma Canada, Bristol-Myers-Squibb Canada, Miltenyi Biotec, and Otsuka Canada Pharmaceutical; has consultant arrangements with CSL Behring and Baxalta; and has received payment for lectures from CSL Behring. L. M. Griffith declares that she has no relevant conflicts of interest. |
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| Information on participants in the PIDTC Leadership Workshop (Bethesda, Md; April 19-20, 2015) and the PIDTC Annual Scientific Workshops (Houston, Tex: May 2-4, 2013 [3rd]; Seattle, Wash: May 1-3, 2014 (4th); and Montreal, Quebec, Canada: April 30-May 2, 2015 (5th) with Education Day (April 29-30, 2015) is provided in Appendices E1 and E2 in this article's Online Repository at www.jacionline.org. |
© 2016
Publié par Elsevier Masson SAS.
Vol 138 - N° 2
P. 375-385 - août 2016 Retour au numéro
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