[HTML][HTML] Long-term follow-up of IPEX syndrome patients after different therapeutic strategies: an international multicenter retrospective study
F Barzaghi, LCA Hernandez, B Neven, S Ricci… - Journal of Allergy and …, 2018 - Elsevier
Background Immunodysregulation polyendocrinopathy enteropathy x-linked (IPEX)
syndrome is a monogenic autoimmune disease caused by FOXP3 mutations. Because it is a
rare disease, the natural history and response to treatments, including allogeneic
hematopoietic stem cell transplantation (HSCT) and immunosuppression (IS), have not been
thoroughly examined. Objective This analysis sought to evaluate disease onset,
progression, and long-term outcome of the 2 main treatments in long-term IPEX survivors …
syndrome is a monogenic autoimmune disease caused by FOXP3 mutations. Because it is a
rare disease, the natural history and response to treatments, including allogeneic
hematopoietic stem cell transplantation (HSCT) and immunosuppression (IS), have not been
thoroughly examined. Objective This analysis sought to evaluate disease onset,
progression, and long-term outcome of the 2 main treatments in long-term IPEX survivors …