期刊
PEDIATRIC TRANSPLANTATION
卷 18, 期 1, 页码 E25-E30出版社
WILEY
DOI: 10.1111/petr.12184
关键词
allogeneic hematopoietic stem cell transplantation; enteropathy; Forkhead box protein 3; immune dysregulation; polyendocrinopathy; reduced intensity conditioning; regulatory T cells; X-linked syndrome
资金
- Ministry of Education, Culture, Sports, Science, and Technology of Japan [23591528]
- Ministry of Health, Labour and Welfare of Japan
- Japan Leukemia Research Fund
- SENSHIN Medical Research Foundation
- Grants-in-Aid for Scientific Research [23591528, 23390266] Funding Source: KAKEN
IPEX syndrome is a rare and fatal disorder caused by absence of regulatory T cells (Tregs) due to congenital mutations in the Forkhead box protein 3 gene. Here, we report a patient with IPEX syndrome treated with RIC followed by allogeneic BMT from an HLA-matched sibling donor. We could achieve engraftment and regimen-related toxicity was well tolerated. Although the patient was in mixed chimera and the ratio of donor cells in whole peripheral blood remained relatively low, selective and sustained expansion of Tregs determined as CD4+CD25+Foxp3+ cells was observed. Improvement in clinical symptoms was correlated with expansion of donor-derived Tregs and disappearance of anti-villin autoantibody, which was involved in the pathogenesis of gastrointestinal symptoms in IPEX syndrome. This clinical observation suggests that donor-derived Tregs have selective growth advantage in patients with IPEX syndrome even in mixed chimera after allogeneic BMT and contribute to the control of clinical symptoms caused by the defect of Tregs.
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