Treatment of the Immune Dysregulation, Polyendocrinopathy, Enteropathy, X-Linked Syndrome (IPEX) by Allogeneic Bone Marrow Transplantation

The immune dysregulation, polyendocrinopathy, enteropathy, X-linked syndrome (IPEX) is a recessive disorder of early childhood.1 Symptoms of the disease generally appear in infancy and include protracted diarrhea, ichthyosiform dermatitis, insulin-dependent diabetes mellitus, thyroiditis, and hemolytic anemia.2–6 Nephropathy has been reported in two familial cases of enteropathy with insulin-dependent diabetes mellitus.7 X-linked recessive inheritance accounts for the familial cases in the seven kindreds studied to date,2–9 but sporadic cases in boys have also been reported.6,10–12 The results of immunologic investigations of IPEX1 are inconsistent with any of the known X-linked immunodeficiency diseases.13 Severe infections have been observed . . .