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 Blood, 1 January 2007, Vol. 109, No. 1, pp. 383-385.
Prepublished online as a Blood First Edition Paper on September 21, 2006; DOI 10.1182/blood-2006-05-025072.

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TRANSPLANTATION

Brief report

Successful bone marrow transplantation for IPEX syndrome after reduced-intensity conditioning

Aarati Rao1, Naynesh Kamani2, Alexandra Filipovich3, Susan Molleran Lee3, Stella M. Davies3, Jignesh Dalal4, and Shalini Shenoy1,

1 Washington University School of Medicine and St Louis Children's Hospital, MO; 2 Children's National Medical Center, Washington, DC; 3 Children's Hospital Medical Center, Cincinnati, OH; 4 Children's Mercy Hospital, Kansas City, MO

Immune dysregulation, polyendocrinopathy, enteropathy, X-linked (IPEX) syndrome is a rare, fatal autoimmune disorder caused by mutations in the FOXP3 gene leading to the disruption of signaling pathways involved in regulatory T-lymphocyte function. Lifelong multiagent immunosuppression is necessary to control debilitating autoimmune manifestations such as colitis and food allergies. Allogeneic hematopoietic stem cell transplantation (HSCT) can restore T-cell regulatory function but has been previously associated with poor outcome. We describe successful HSCT in 4 patients with IPEX syndrome using a novel reduced-intensity conditioning regimen that resulted in stable donor engraftment, reconstitution of FOXP3+ T regulatory CD4+ cells, and amelioration of gastrointestinal symptoms.


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