ArticleThe Journal of allergy and clinical immunology2010
Stable hematopoietic cell engraftment after low-intensity nonmyeloablative conditioning in patients with immune dysregulation, polyendocrinopathy, enteropathy, X-linked syndrome.
Article in The Journal of allergy and clinical immunology, 2010. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 27 papers.
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Who cites it
27 citing papers in PubMed, 63 citations in OpenAlex.
- Long-term follow-up of IPEX syndrome patients after different therapeutic strategies: An international multicenter retrospective study.The Journal of allergy and clinical immunology · 2018Trial
- Treosulfan-based conditioning and hematopoietic cell transplantation for nonmalignant diseases: a prospective multicenter trial.Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation · 2014Trial
- Sirolimus alleviated intractable diarrhea of IPEX syndrome: a case report and literature review.BMC pediatrics · 2024Review
- A novel FOXP3 mutation in a Chinese child with IPEX-associated membranous nephropathy.Molecular genetics & genomic medicine · 2022Review
- Molecular diagnosis of childhood immune dysregulation, polyendocrinopathy, and enteropathy, and implications for clinical management.The Journal of allergy and clinical immunology · 2022Article
- IPEX Syndrome: Improved Knowledge of Immune Pathogenesis Empowers Diagnosis.Frontiers in pediatrics · 2021Review
- Atypical Presentations of IPEX: Expect the Unexpected.Frontiers in pediatrics · 2021Review
- Very Early Onset Inflammatory Bowel Disease: A Clinical Approach With a Focus on the Role of Genetics and Underlying Immune Deficiencies.Inflammatory bowel diseases · 2020Review
- Conditioning Regimens for Hematopoietic Cell Transplantation in Primary Immunodeficiency.Current allergy and asthma reports · 2019Review
- Diagnosis and treatment of a boy with IPEX syndrome presenting with diabetes in early infancy.Clinical case reports · 2019Article
- Autoimmune Enteropathy: An Updated Review with Special Focus on Stem Cell Transplant Therapy.Digestive diseases and sciences · 2019Review
- Molecular Mechanisms Controlling Foxp3 Expression in Health and Autoimmunity: From Epigenetic to Post-translational Regulation.Frontiers in immunology · 2019Review
- Hematopoietic Stem Cell Transplantation in Primary Immunodeficiency Diseases: Current Status and Future Perspectives.Frontiers in pediatrics · 2019Review
- Clinical, Immunological, and Molecular Heterogeneity of 173 Patients With the Phenotype of Immune Dysregulation, Polyendocrinopathy, Enteropathy, X-Linked (IPEX) Syndrome.Frontiers in immunology · 2018Article
- Alternative Splicing of FOXP3-Virtue and Vice.Frontiers in immunology · 2018Review
- Atypical Late-Onset Immune Dysregulation, Polyendocrinopathy, Enteropathy, X-Linked Syndrome with Intractable Diarrhea: A Case Report.Frontiers in pediatrics · 2017Article
- Resetting microbiota by Lactobacillus reuteri inhibits T reg deficiency-induced autoimmunity via adenosine A2A receptors.The Journal of experimental medicine · 2017Article
- Rare phenotypes in the understanding of autoimmunity.Immunology and cell biology · 2016Review
- Successful hematopoietic cell transplantation in a patient with X-linked agammaglobulinemia and acute myeloid leukemia.Pediatric blood & cancer · 2015Article
- Renal involvement in the immunodysregulation, polyendocrinopathy, enteropathy, X-linked (IPEX) disorder.Pediatric nephrology (Berlin, Germany) · 2015Article
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Authors and funding
11 authors at 3 institutions in 2 countries.
Funding
Abstract
backgroundImmune 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.
objectiveHere we present the outcome of hematopoietic cell transplantation using a low-intensity, nonmyeloablative conditioning regimen in 2 patients with IPEX syndrome and significant pretransplant risk factors.
methodsTwo high-risk patients with IPEX syndrome received HLA-matched related bone marrow or unrelated peripheral blood stem cell grafts following conditioning with 90 mg/m(2) fludarabine and 4 Gy total body irradiation. Postgrafting immunosuppression consisted of mycophenolate mofetil and cyclosporine. Immune reconstitution and immune function was evaluated by measurement of donor chimerism, regulatory T-cell numbers, absolute lymphocyte subsets, and T-cell proliferation assays.
resultsBoth patients experienced minimal conditioning toxicity and successfully engrafted after hematopoietic cell transplantation. With a follow-up of 4 and 1 years, respectively, patients 1 and 2 have full immune function and normal FOXP3 protein expression.
conclusionA low-intensity, nonmyeloablative conditioning regimen can establish stable engraftment and correct the life-threatening immune deficiency and enteropathy of IPEX syndrome despite the presence of comorbidities that preclude conventional hematopoietic cell transplantation.
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