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). 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.
Division of Allergy, Immunology and Transplantation, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Md. Electronic address: LGriffith@niaid.nih.gov.
Linda M
LM
Griffith
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.
Morton J
MJ
Cowan
Division of Immunology, Children's Hospital, and Harvard Stem Cell Institute, Harvard Medical School, Boston, Mass.
Luigi D
LD
Notarangelo
Departments of Microbiology, Immunology & Molecular Genetics and Pediatrics, University of California Los Angeles, Los Angeles, Calif.
Donald B
DB
Kohn
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.
Jennifer M
JM
Puck
Pediatric Allergy & Immunology, Texas Children's Hospital, Baylor College of Medicine, Houston, Tex.
William T
WT
Shearer
Pediatric Hematology/Oncology, Fred Hutchinson Cancer Research Center, University of Washington School of Medicine, Seattle, Wash.
Lauri M
LM
Burroughs
Pediatric Rheumatology, Seattle Children's Research Institute, University of Washington School of Medicine, Seattle, Wash.
Troy R
TR
Torgerson
Pediatric Immunology and Pediatrics, Mother and Child Ste-Justine Hospital, University of Montreal, Montreal, Quebec, Canada.
Hélène
H
Decaluwe
Pediatric Immunology and Pediatrics, Mother and Child Ste-Justine Hospital, University of Montreal, Montreal, Quebec, Canada.
Elie
E
Haddad
D000818:Animals; D000068456:Clinical Studies as Topic; D006801:Humans; D007153:Immunologic Deficiency Syndromes / Q000175:diagnosis* / Q000628:therapy*; D000070516:Intersectoral Collaboration; D014837:Voluntary Health Agencies
Allogeneic hematopoietic cell transplantation; clinical trial; gene therapy; primary immunodeficiency