Primary Immune Deficiency Treatment Consortium (PIDTC) update
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)....
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| Published in | Journal of allergy and clinical immunology Vol. 138; no. 2; pp. 375 - 385 |
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| Main Authors | , , , , , , , , , |
| Format | Journal Article |
| Language | English |
| Published |
United States
Elsevier Inc
01.08.2016
Elsevier Limited |
| Subjects | |
| Online Access | Get full text |
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| 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. |
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| AbstractList | 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. 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. The Primary Immune Deficiency Treatment Consortium (PIDTC) is a collaboration of 41 North American centers studying therapy for rare primary immune deficiency diseases (PID) 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 PID in multicenter longitudinal retrospective, prospective and cross-sectional studies. Since 2009, participating centers have enrolled over 800 subjects on PIDTC protocols for SCID, and enrollment on the studies in 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 outcomes of hematopoietic cell transplantation (HCT) for SCID during 2000–2009, diagnostic criteria for SCID, and the pilot project of newborn screening (NBS) 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 NIAID and ORDR, NCATS, 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. 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.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. |
| Author | Griffith, Linda M. Burroughs, Lauri M. Haddad, Elie Cowan, Morton J. Notarangelo, Luigi D. Kohn, Donald B. Torgerson, Troy R. Decaluwe, Hélène Shearer, William T. Puck, Jennifer M. |
| AuthorAffiliation | d Departments of Microbiology, Immunology & Molecular Genetics and Pediatrics, University of California Los Angeles, Los Angeles, CA g Pediatric Rheumatology, Seattle Children’s Research Institute, University of Washington School of Medicine, Seattle, WA a Division of Allergy, Immunology and Transplantation, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD c Division of Immunology, Children’s Hospital, and Harvard Stem Cell Institute, Harvard Medical School, Boston, MA f Pediatric Hematology/Oncology, Fred Hutchinson Cancer Research Center, University of Washington School of Medicine, Seattle, WA 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, CA e Pediatric Allergy & Immunology, Texas Children’s Hospital, Baylor College of Medicine, Houston TX h Pediatric Immunology and Pediatrics, Mother and Child Ste-J |
| AuthorAffiliation_xml | – name: c Division of Immunology, Children’s Hospital, and Harvard Stem Cell Institute, Harvard Medical School, Boston, MA – name: f Pediatric Hematology/Oncology, Fred Hutchinson Cancer Research Center, University of Washington School of Medicine, Seattle, WA – name: h Pediatric Immunology and Pediatrics, Mother and Child Ste-Justine Hospital, University of Montreal, Montreal, QC, Canada – name: d Departments of Microbiology, Immunology & Molecular Genetics and Pediatrics, University of California Los Angeles, Los Angeles, CA – name: a Division of Allergy, Immunology and Transplantation, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD – name: e Pediatric Allergy & Immunology, Texas Children’s Hospital, Baylor College of Medicine, Houston TX – name: g Pediatric Rheumatology, Seattle Children’s Research Institute, University of Washington School of Medicine, Seattle, WA – name: 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, CA |
| Author_xml | – sequence: 1 givenname: Linda M. surname: Griffith fullname: Griffith, Linda M. email: LGriffith@niaid.nih.gov organization: Division of Allergy, Immunology and Transplantation, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Md – sequence: 2 givenname: Morton J. surname: Cowan fullname: Cowan, Morton J. organization: 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 – sequence: 3 givenname: Luigi D. surname: Notarangelo fullname: Notarangelo, Luigi D. organization: Division of Immunology, Children's Hospital, and Harvard Stem Cell Institute, Harvard Medical School, Boston, Mass – sequence: 4 givenname: Donald B. surname: Kohn fullname: Kohn, Donald B. organization: Departments of Microbiology, Immunology & Molecular Genetics and Pediatrics, University of California Los Angeles, Los Angeles, Calif – sequence: 5 givenname: Jennifer M. surname: Puck fullname: Puck, Jennifer M. organization: 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 – sequence: 6 givenname: William T. surname: Shearer fullname: Shearer, William T. organization: Pediatric Allergy & Immunology, Texas Children's Hospital, Baylor College of Medicine, Houston, Tex – sequence: 7 givenname: Lauri M. surname: Burroughs fullname: Burroughs, Lauri M. organization: Pediatric Hematology/Oncology, Fred Hutchinson Cancer Research Center, University of Washington School of Medicine, Seattle, Wash – sequence: 8 givenname: Troy R. surname: Torgerson fullname: Torgerson, Troy R. organization: Pediatric Rheumatology, Seattle Children's Research Institute, University of Washington School of Medicine, Seattle, Wash – sequence: 9 givenname: Hélène surname: Decaluwe fullname: Decaluwe, Hélène organization: Pediatric Immunology and Pediatrics, Mother and Child Ste-Justine Hospital, University of Montreal, Montreal, Quebec, Canada – sequence: 10 givenname: Elie surname: Haddad fullname: Haddad, Elie organization: Pediatric Immunology and Pediatrics, Mother and Child Ste-Justine Hospital, University of Montreal, Montreal, Quebec, Canada |
| BackLink | https://www.ncbi.nlm.nih.gov/pubmed/27262745$$D View this record in MEDLINE/PubMed |
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| Keywords | NCATS WAS X-SCID SCID PIDTC ORDR CIBMTR IEWP primary immunodeficiency MUD NBS MSD clinical trial CGD EBMT OS gene therapy PID NIAID NIH GT RD Allogeneic hematopoietic cell transplantation PAG HCT TREC ADA Severe combined immune deficiency X-linked severe combined immune deficiency National Institutes of Health T-cell receptor excision circle Center for International Blood and Marrow Transplant Research Reticular dysgenesis Hematopoietic cell transplantation Inborn Errors Working Party Office of Rare Diseases Research Matched sibling donor Omenn syndrome Wiskott-Aldrich syndrome Chronic granulomatous disease European Group for Blood and Marrow Transplantation Newborn screening Matched unrelated donor National Center for Advancing Translational Sciences Primary Immune Deficiency Treatment Consortium Patient advocacy group Adenosine deaminase National Institute of Allergy and Infectious Diseases Primary immune deficiency disease |
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| SubjectTerms | Allergy and Immunology Allogeneic hematopoietic cell transplantation Animals Chemotherapy Clinical Studies as Topic clinical trial Collaboration Consortia Disease Funding gene therapy Humans Immunologic Deficiency Syndromes - diagnosis Immunologic Deficiency Syndromes - therapy Intersectoral Collaboration Newborn babies Patients primary immunodeficiency Studies Transplants & implants Voluntary Health Agencies |
| Title | Primary Immune Deficiency Treatment Consortium (PIDTC) update |
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