Primary Immune Deficiency Treatment Consortium (PIDTC) report
Linda M Griffith1, Morton J Cowan2, Luigi D Notarangelo3
1Division of Allergy, Immunology and Transplantation, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Md.
The Journal of Allergy and Clinical Immunology
|October 22, 2013
Summary
The Primary Immune Deficiency Treatment Consortium (PIDTC) advances rare disease research through multi-center studies and training. It focuses on severe combined immunodeficiency (SCID) and other primary immunodeficiencies, aiming for optimal treatment strategies.
Area of Science:
- Immunology
- Genetics
- Clinical Research
Background:
- The Primary Immune Deficiency Treatment Consortium (PIDTC) is a North American network of 33 centers.
- It focuses on rare and severe primary immunodeficiency diseases.
Purpose of the Study:
- To report the progress of the PIDTC to date.
- To highlight the first two PIDTC workshops.
- To consider future consortium objectives.
Main Methods:
- Retrospective, prospective, and cross-sectional studies.
- Pilot demonstration projects.
- Collaborations with international colleagues and patient advocacy groups.
Main Results:
- Funded 2 pilot projects: SCID newborn screening in Navajo Native Americans and B-cell reconstitution post-hematopoietic stem cell transplantation.
- Awarded grants to 10 junior investigators.
- Held annual workshops fostering collaboration and discussion on research progress and future directions.
Conclusions:
- The PIDTC is actively progressing in studying and treating primary immunodeficiencies.
- Future goals include prospective treatment studies to determine optimal therapies.
- The consortium fosters research, training, and community awareness for rare immune disorders.
Keywords:
ADAAdenosine deaminaseAllogeneic hematopoietic cell transplantationCGDCIBMTRCenter for International Blood and Marrow Transplant ResearchChronic granulomatous diseaseEBMTESIDEuropean Group for Blood and Marrow TransplantationEuropean Society for ImmunodeficienciesGTGVHDGene therapyGraft-versus-host diseaseHCTHematopoietic cell transplantationIEWPInborn Errors Working PartyMACMUDMatched unrelated donorMyeloablative conditioningNBSNIAIDNIHNKNational Institute of Allergy and Infectious DiseasesNational Institutes of HealthNatural killerNewborn screening for SCIDPIDPIDTCPrimary Immune Deficiency Treatment ConsortiumPrimary immunodeficiencyRICReduced-intensity conditioningSCETIDESCIDSevere combined immunodeficiencyStem Cell Transplantation for Immunodeficiencies in EuropeUSIDNETUnited States Immunodeficiency NetworkWASWiskott-Aldrich syndromeclinical trialgene therapyprimary immunodeficiency
