[Transplant of hematopoietic stem cells in X-linked adrenoleukodystrophy].
Over 130 patients with adrenoleukodystrophy have received hematopoietic stem cell transplantation from normal donors. Without treatment, this disease has an inexorable fatal course. On the other hand, all of the engrafted hosts have had a remarkable positive clinical improvement in response to normalization of previously deficient enzymatic activity. The specific indications and methods for transplantation are also included in this article.
Madero
L
L
Sección Onco-hematología/trasplante hematopoyético; Hospital Niño Jesús, Madrid, 28009, España. lmadero@ctv.es
Sevilla
J
J
D000326:Adrenoleukodystrophy / Q000628:therapy*; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans