Stable hematopoietic cell engraftment after low-intensity nonmyeloablative conditioning in patients with immune dysregulation, polyendocrinopathy, enteropathy, X-linked syndrome.

Publication Type:

Journal Article

Source:

The Journal of allergy and clinical immunology, Volume 126, Issue 5, p.1000-5 (2010)

Keywords:

2010, Adolescent, Cell Separation, Center-Authored Paper, Child, Clinical Research Division, Cyclosporine, Flow Cytometry Core Facility, Forkhead Transcription Factors, Genetic Diseases, X-Linked, Graft vs Host Disease, hematopoietic stem cell transplantation, Humans, Immunologic Deficiency Syndromes, Immunosuppressive Agents, Infant, Male, Mycophenolic Acid, Myeloablative Agonists, Research Trials Office Core Facility - Biostatistics Service, Reverse Transcriptase Polymerase Chain Reaction, Shared Resources, Syndrome, Transplantation Conditioning, Vaccine and Infectious Disease Division, Vidarabine, Whole-Body Irradiation

Abstract:

Immune dysregulation, polyendocrinopathy, enteropathy, X-linked (IPEX) syndrome is characterized by severe systemic autoimmunity caused by mutations in the forkhead box protein 3 (FOXP3) gene. Hematopoietic cell transplantation is currently the only viable option for long-term survival, but patients are frequently very ill and may not tolerate traditional myeloablative conditioning regimens.