Thus, in conclusion, a patient with IPEX, LRBA deficiency, and probably also CTLA4 deficiency should undergo alloHSCT evaluation and donor search upon diagnosis, and, ideally, an attempt to ameliorate disease severity by targeted immunosuppressive treatment should be undertaken before alloHSCT. This evidence concerns the gene CTLA4 and immune dysregulation-polyendocrinopathy-enteropathy-X-linked syndrome.