Hematopoietic cell transplantation for mucopolysaccharidosis IIB (Hunter syndrome).
Hunter syndrome is an X-linked metabolic storage disorder arising from deficiency of iduronate sulfatase enzyme activity. Despite the successful use of hematopoietic cell transplantation for a variety of lysosomal and peroxisomal storage diseases, limited benefit occurs following transplantation in either the severe or mild forms of Hunter syndrome. A brief ethical commentary is provided on the case of a boy with mucopolysaccharidosis IIB (ie the mild form) who received an unrelated umbilical cord blood transplant to improve his future quality of life. Bone Marrow Transplantation (2000).
Peters
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Department of Pediatrics, University of Minnesota, Minneapolis, MN 55455, USA.
Krivit
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W
D000744:Anemia, Hemolytic, Autoimmune / Q000209:etiology*; D001327:Autoimmune Diseases / Q000209:etiology*; D016470:Bacteremia / Q000209:etiology; D003657:Decision Making; D004992:Ethics, Medical; D005312:Fetal Blood / Q000166:cytology*; D006086:Graft vs Host Disease / Q000209:etiology; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects*; D006801:Humans; D007231:Infant, Newborn; D007258:Informed Consent; D008297:Male; D016532:Mucopolysaccharidosis II / Q000150:complications / Q000628:therapy*; D011788:Quality of Life; D014184:Transplantation, Homologous / Q000009:adverse effects; D016896:Treatment Outcome