Hematopoietic cell transplantation for mucopolysaccharidosis IIB (Hunter syndrome).

Abstract

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).

• Authors: C Peters, W Krivit
• Journal: Bone marrow transplantation (Bone Marrow Transplant) Vol. 25 Issue 10 Pg. 1097-9 (May 2000) ISSN: 0268-3369 [Print] England
• PMID: 10828872 (Publication Type: Case Reports, Journal Article, Research Support, Non-U.S. Gov't)

Topics

• Anemia, Hemolytic, Autoimmune (etiology)
• Autoimmune Diseases (etiology)
• Bacteremia (etiology)
• Decision Making
• Ethics, Medical
• Fetal Blood (cytology)
• Graft vs Host Disease (etiology)
• Hematopoietic Stem Cell Transplantation (adverse effects)
• Humans
• Infant, Newborn
• Informed Consent
• Male
• Mucopolysaccharidosis II (complications, therapy)
• Quality of Life
• Transplantation, Homologous (adverse effects)
• Treatment Outcome