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. Dvorak; Alexandra 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

doi:10.1016/j.jaci.2013.07.052

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

Pediatrics

Research output: Contribution to journal › Article › peer-review

44 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 language	English (US)
Pages (from-to)	335-347.e11
Journal	Journal of Allergy and Clinical Immunology
Volume	133
Issue number	2
DOIs	https://doi.org/10.1016/j.jaci.2013.07.052
State	Published - Feb 2014

Keywords

Allogeneic hematopoietic cell transplantation
clinical trial
gene therapy
primary immunodeficiency

ASJC Scopus subject areas

Immunology and Allergy
Immunology

Access to Document

10.1016/j.jaci.2013.07.052

Cite this

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. H., 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., ... Zuniga-Pflucker, J. C. (2014). Primary Immune Deficiency Treatment Consortium (PIDTC) report. Journal of Allergy and Clinical Immunology, 133(2), 335-347.e11. https://doi.org/10.1016/j.jaci.2013.07.052

@article{7c06133b9dbc4d3499ecb98723405c80,

title = "Primary Immune Deficiency Treatment Consortium (PIDTC) report",

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.",

keywords = "Allogeneic hematopoietic cell transplantation, clinical trial, gene therapy, primary immunodeficiency",

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

note = "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: 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. 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{\textquoteright}s Hospital Translational Research Program, Children{\textquoteright}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 . ",

year = "2014",

month = feb,

doi = "10.1016/j.jaci.2013.07.052",

language = "English (US)",

volume = "133",

pages = "335--347.e11",

journal = "Journal of Allergy and Clinical Immunology",

issn = "0091-6749",

publisher = "Mosby Inc.",

number = "2",

}

Griffith, LM, Cowan, MJ, Notarangelo, LD, Kohn, DB, Puck, JM, Pai, SY, Ballard, B, Bauer, SC, Bleesing, JJH, Boyle, M, Brower, A, Buckley, RH, Van Der Burg, M, Burroughs, LM, Candotti, F, Cant, AJ, Chatila, T, Cunningham-Rundles, C, Dinauer, MC, Dvorak, CC, Filipovich, AH, Fleisher, TA, Bobby Gaspar, H, Gungor, T, Haddad, E, Hovermale, E, Huang, F, Hurley, A, Hurley, M, Iyengar, S, Kang, EM, Logan, BR, Long-Boyle, JR, Malech, HL, McGhee, SA, Modell, F, Modell, V, Ochs, HD, O'Reilly, RJ, Parkman, R, Rawlings, DJ, Routes, JM, Shearer, WT, Small, TN, Smith, H, Sullivan, KE, Szabolcs, P, Thrasher, A, Torgerson, TR, Veys, P, Weinberg, K & Zuniga-Pflucker, JC 2014, 'Primary Immune Deficiency Treatment Consortium (PIDTC) report', Journal of Allergy and Clinical Immunology, vol. 133, no. 2, pp. 335-347.e11. https://doi.org/10.1016/j.jaci.2013.07.052

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: 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. 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

UR - http://www.scopus.com/inward/citedby.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 -

Primary Immune Deficiency Treatment Consortium (PIDTC) report

Abstract

Keywords

ASJC Scopus subject areas

Access to Document

Other files and links

Fingerprint

Cite this