Primary Immune Deficiency Treatment Consortium (PIDTC) update

• Published in: Journal of allergy and clinical immunology Vol. 138; no. 2; pp. 375 - 385
• Main Authors: Griffith, Linda M., Cowan, Morton J., Notarangelo, Luigi D., Kohn, Donald B., Puck, Jennifer M., Shearer, William T., Burroughs, Lauri M., Torgerson, Troy R., Decaluwe, Hélène, Haddad, Elie
• Format: Journal Article
• Language: English
• Published: United States Elsevier Inc 01.08.2016; Elsevier Limited
• Subjects: 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; North America; California; 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
• ISSN: 0091-6749; 1097-6825; 1097-6825
• DOI: 10.1016/j.jaci.2016.01.051

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.