Journal
JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY
Volume 138, Issue 2, Pages 375-385Publisher
MOSBY-ELSEVIER
DOI: 10.1016/j.jaci.2016.01.051
Keywords
Allogeneic hematopoietic cell transplantation; gene therapy; primary immunodeficiency; clinical trial
Categories
Funding
- Division of Allergy, Immunology and Transplantation, National Institute of Allergy and Infectious Diseases (NIAID)
- Intramural Research Program of the NIAID
- Office of Rare Diseases Research, National Center for Advancing Translational Sciences, National Institutes of Health, Bethesda, Md [U54-AI082973, U54-NS064808, U01-TR001263, R13-AI094943]
- Immune Deficiency Foundation, Towson, Md
- Jeffrey Modell Foundation, New York, NY
- David Center, Texas Children's Hospital, Houston, Tex
- 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
- GRI-FOLS 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
- Sigma-Tau Pharmaceuticals, Gaithersburg, Md
- ORDR, NCATS
- Division of Allergy Immunology and Transplantation (DAIT), NIAID
Ask authors/readers for more resources
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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