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, workshop participants, Morton J Cowan, Linda M Griffith, Donald B Kohn, Luigi D Notarangelo, Jennifer M Puck, Victor Aquino, Barbara Ballard, Sarah C Bauer, Jack J H Bleesing, Marcia Boyle, Rebecca H Buckley, Lauri M Burroughs, Fabio Candotti, Andrew J Cant, Alice Chan, Ka Wah Chan, Karin Chen, Charlotte Cunningham-Rundles, Geoff Cuvelier, Mary C Dinauer, Elizabeth Dunn, Christopher C Dvorak, Alexandra H Filipovich, Thomas A Fleisher, Lisa R Forbes, Ramsay L Fuleihan, Hubert Bobby Gaspar, Frederick Goldman, Rashmi Gopal-Srivastava, Eyal Grunebaum, Elie Haddad, Lisa Harewood, Jennifer R Heimall, Faith Huang, Alan Hurley, Mary Hurley, Sumathi Iyengar, Theodore S Johnson, Soma Jyonouchi, Elizabeth M Kang, Neena Kapoor, Alan P Knutsen, Jason Law, Brett J Loechelt, Harry L Malech, Caridad Martinez, Sean A McGhee, Theodore B Moore, Maria Teresa de la Morena, Megan M Morsheimer, Sarah K Nicholas, Hans D Ochs, Richard J O'Reilly, Sung-Yun Pai, Robertson Parkman, Sherrie Pryber, Michael A Pulsipher, David J Rawlings, John M Routes, Jacob Rozmus, Marlis Schroeder, Kirk R Schultz, Suzanne Skoda-Smith, William T Shearer, Evan B Shereck, Chu Ri Shin, Angela Smith, Heather Stefanski, Paul Szabolcs, Troy R Torgerson, William Tse, Paul Veys, Kenneth Weinberg, Jennifer M Puck, John M Routes, Neena Kapoor, Angela Smith, Morton J Cowan, Sung-Yun Pai, Brent R Logan, Rebecca H Buckley, Lauri Burroughs, Christopher C Dvorak, Donald B Kohn, Janel R Long-Boyle, Richard J O'Reilly, Michael A Pulsipher, Christopher C Dvorak, Caridad Martinez, Janel R Long-Boyle, Suzanne Skoda-Smith, Angela Smith, Thomas Fleisher, Trudy Small, Jack J H Bleesing, Luigi D Notarangelo, Paul Szabolcs, Richard J O'Reilly, Morton J Cowan, Linda M Griffith, Donald B Kohn, Luigi D Notarangelo, Sung-Yun Pai, Jennifer M Puck, Stephanie Albin, T Prescott Atkinson, Barbara Ballard, Jeffrey J Bednarski, Marcia Boyle, Amy Brower, Rebecca H Buckley, Mirjam van der Burg, Lauri Burroughs, Fabio Candotti, Ka Wah Chan, Talal Chatila, Joshua A Checketts, Anne Marie Comeau, James Albert Connelly, Charlotte Cunningham-Rundles, Geoff Cuvelier, Agnieszka Czechowicz, Shaffina Dhume, Elizabeth Dunn, Christopher C Dvorak, Kerstin Felgentreff, Alexandra H Filipovich, Thomas A Fleisher, Lisa R Forbes, Patricia C Fulkerson, Alfred P Gillio, Frederick Goldman, Rashmi Gopal-Srivastava, Nancy S Green, Tayfun Gungor, Elie Haddad, Imelda Celine Hanson, Jeanette Haugh, Jennifer R Heimall, Emily Hovermale, Mary Hurley, Sumathi Iyengar, Elizabeth M Kang, Neena Kapoor, Renna Killen, Alan P Knutsen, James LaBelle, Jason Law, Brent R Logan, Janel R Long-Boyle, Maxim Lutskiy, Harry L Malech, Rebecca A Marsh, Caridad Martinez, Fred Modell, Vicki Modell, Maria Teresa de la Morena, Sarah K Nicholas, Hans D Ochs, Satiro de Oliveira, Richard J O'Reilly, Kenneth Paris, Robertson Parkman, Matthew H Porteus, Michael A Pulsipher, David J Rawlings, John M Routes, Jacob Rozmus, Holly Ruhlig, Kirk R Schultz, Suzanne Skoda-Smith, William T Shearer, Trudy N Small, Angela Smith, Heather Smith, Heather Stefanski, Kathleen E Sullivan, Megan Sykes, Paul Szabolcs, Adrian Thrasher, Troy R Torgerson, Irit Tirosh, Stefano Volpi, Jolan E Walter, Katja G Weinacht, Juan Carlos Zuniga-Pflucker, 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, workshop participants, Morton J Cowan, Linda M Griffith, Donald B Kohn, Luigi D Notarangelo, Jennifer M Puck, Victor Aquino, Barbara Ballard, Sarah C Bauer, Jack J H Bleesing, Marcia Boyle, Rebecca H Buckley, Lauri M Burroughs, Fabio Candotti, Andrew J Cant, Alice Chan, Ka Wah Chan, Karin Chen, Charlotte Cunningham-Rundles, Geoff Cuvelier, Mary C Dinauer, Elizabeth Dunn, Christopher C Dvorak, Alexandra H Filipovich, Thomas A Fleisher, Lisa R Forbes, Ramsay L Fuleihan, Hubert Bobby Gaspar, Frederick Goldman, Rashmi Gopal-Srivastava, Eyal Grunebaum, Elie Haddad, Lisa Harewood, Jennifer R Heimall, Faith Huang, Alan Hurley, Mary Hurley, Sumathi Iyengar, Theodore S Johnson, Soma Jyonouchi, Elizabeth M Kang, Neena Kapoor, Alan P Knutsen, Jason Law, Brett J Loechelt, Harry L Malech, Caridad Martinez, Sean A McGhee, Theodore B Moore, Maria Teresa de la Morena, Megan M Morsheimer, Sarah K Nicholas, Hans D Ochs, Richard J O'Reilly, Sung-Yun Pai, Robertson Parkman, Sherrie Pryber, Michael A Pulsipher, David J Rawlings, John M Routes, Jacob Rozmus, Marlis Schroeder, Kirk R Schultz, Suzanne Skoda-Smith, William T Shearer, Evan B Shereck, Chu Ri Shin, Angela Smith, Heather Stefanski, Paul Szabolcs, Troy R Torgerson, William Tse, Paul Veys, Kenneth Weinberg, Jennifer M Puck, John M Routes, Neena Kapoor, Angela Smith, Morton J Cowan, Sung-Yun Pai, Brent R Logan, Rebecca H Buckley, Lauri Burroughs, Christopher C Dvorak, Donald B Kohn, Janel R Long-Boyle, Richard J O'Reilly, Michael A Pulsipher, Christopher C Dvorak, Caridad Martinez, Janel R Long-Boyle, Suzanne Skoda-Smith, Angela Smith, Thomas Fleisher, Trudy Small, Jack J H Bleesing, Luigi D Notarangelo, Paul Szabolcs, Richard J O'Reilly, Morton J Cowan, Linda M Griffith, Donald B Kohn, Luigi D Notarangelo, Sung-Yun Pai, Jennifer M Puck, Stephanie Albin, T Prescott Atkinson, Barbara Ballard, Jeffrey J Bednarski, Marcia Boyle, Amy Brower, Rebecca H Buckley, Mirjam van der Burg, Lauri Burroughs, Fabio Candotti, Ka Wah Chan, Talal Chatila, Joshua A Checketts, Anne Marie Comeau, James Albert Connelly, Charlotte Cunningham-Rundles, Geoff Cuvelier, Agnieszka Czechowicz, Shaffina Dhume, Elizabeth Dunn, Christopher C Dvorak, Kerstin Felgentreff, Alexandra H Filipovich, Thomas A Fleisher, Lisa R Forbes, Patricia C Fulkerson, Alfred P Gillio, Frederick Goldman, Rashmi Gopal-Srivastava, Nancy S Green, Tayfun Gungor, Elie Haddad, Imelda Celine Hanson, Jeanette Haugh, Jennifer R Heimall, Emily Hovermale, Mary Hurley, Sumathi Iyengar, Elizabeth M Kang, Neena Kapoor, Renna Killen, Alan P Knutsen, James LaBelle, Jason Law, Brent R Logan, Janel R Long-Boyle, Maxim Lutskiy, Harry L Malech, Rebecca A Marsh, Caridad Martinez, Fred Modell, Vicki Modell, Maria Teresa de la Morena, Sarah K Nicholas, Hans D Ochs, Satiro de Oliveira, Richard J O'Reilly, Kenneth Paris, Robertson Parkman, Matthew H Porteus, Michael A Pulsipher, David J Rawlings, John M Routes, Jacob Rozmus, Holly Ruhlig, Kirk R Schultz, Suzanne Skoda-Smith, William T Shearer, Trudy N Small, Angela Smith, Heather Smith, Heather Stefanski, Kathleen E Sullivan, Megan Sykes, Paul Szabolcs, Adrian Thrasher, Troy R Torgerson, Irit Tirosh, Stefano Volpi, Jolan E Walter, Katja G Weinacht, Juan Carlos Zuniga-Pflucker
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: ADA; Adenosine deaminase; Allogeneic hematopoietic cell transplantation; CGD; CIBMTR; Center for International Blood and Marrow Transplant Research; Chronic granulomatous disease; EBMT; ESID; European Group for Blood and Marrow Transplantation; European Society for Immunodeficiencies; GT; GVHD; Gene therapy; Graft-versus-host disease; HCT; Hematopoietic cell transplantation; IEWP; Inborn Errors Working Party; MAC; MUD; Matched unrelated donor; Myeloablative conditioning; NBS; NIAID; NIH; NK; National Institute of Allergy and Infectious Diseases; National Institutes of Health; Natural killer; Newborn screening for SCID; PID; PIDTC; Primary Immune Deficiency Treatment Consortium; Primary immunodeficiency; RIC; Reduced-intensity conditioning; SCETIDE; SCID; Severe combined immunodeficiency; Stem Cell Transplantation for Immunodeficiencies in Europe; USIDNET; United States Immunodeficiency Network; WAS; Wiskott-Aldrich syndrome; clinical trial; gene therapy; primary immunodeficiency.
Published by Mosby, Inc.
Source: PubMed