Primary Immune Deficiency Treatment Consortium (PIDTC) report

Abstract

The Primary Immune Deficiency Treatment Consortium (PIDTC) is a network of 33 centers in North America that study the treatment of rare and severe primary immunodeficiency diseases. Current protocols address the natural history of patients treated for severe combined immunodeficiency (SCID), Wiskott-Aldrich syndrome, and chronic granulomatous disease through retrospective, prospective, and cross-sectional studies. The PIDTC additionally seeks to encourage training of junior investigators, establish partnerships with European and other International colleagues, work with patient advocacy groups to promote community awareness, and conduct pilot demonstration projects. Future goals include the conduct of prospective treatment studies to determine optimal therapies for primary immunodeficiency diseases. To date, the PIDTC has funded 2 pilot projects: newborn screening for SCID in Navajo Native Americans and B-cell reconstitution in patients with SCID after hematopoietic stem cell transplantation. Ten junior investigators have received grant awards. The PIDTC Annual Scientific Workshop has brought together consortium members, outside speakers, patient advocacy groups, and young investigators and trainees to report progress of the protocols and discuss common interests and goals, including new scientific developments and future directions of clinical research. Here we report the progress of the PIDTC to date, highlights of the first 2 PIDTC workshops, and consideration of future consortium objectives.

Keywords: ADA; Adenosine deaminase; Allogeneic hematopoietic cell transplantation; CGD; CIBMTR; Center for International Blood and Marrow Transplant Research; Chronic granulomatous disease; EBMT; ESID; European Group for Blood and Marrow Transplantation; European Society for Immunodeficiencies; GT; GVHD; Gene therapy; Graft-versus-host disease; HCT; Hematopoietic cell transplantation; IEWP; Inborn Errors Working Party; MAC; MUD; Matched unrelated donor; Myeloablative conditioning; NBS; NIAID; NIH; NK; National Institute of Allergy and Infectious Diseases; National Institutes of Health; Natural killer; Newborn screening for SCID; PID; PIDTC; Primary Immune Deficiency Treatment Consortium; Primary immunodeficiency; RIC; Reduced-intensity conditioning; SCETIDE; SCID; Severe combined immunodeficiency; Stem Cell Transplantation for Immunodeficiencies in Europe; USIDNET; United States Immunodeficiency Network; WAS; Wiskott-Aldrich syndrome; clinical trial; gene therapy; primary immunodeficiency.