Unrelated umbilical cord blood transplantation in infancy for mucopolysaccharidosis type IIB (Hunter syndrome) complicated by autoimmune hemolytic anemia.
This report describes unrelated umbilical cord blood transplantation for a 10-month-old infant boy with mucopolysaccharidosis IIB (Hunter syndrome), an X-linked metabolic storage disorder due to deficiency of iduronate sulfatase. Two years after transplant approximately 55% normal plasma enzyme activity has been restored and abnormal urinary excretion of glycosaminoglycans has nearly completely resolved. The boy has exhibited normal growth and development after transplant. Nine months after transplant he developed severe autoimmune hemolytic anemia and required 14 months of corticosteroid treatment to prevent clinically significant anemia. Bone marrow transplantation for Hunter syndrome and post-transplant hemolytic anemia are reviewed. Bone Marrow Transplantation (2000).
Mullen
C A
CA
Department of Pediatrics, University of Texas MD Anderson Cancer Center, Houston, Texas 77030, USA.
Thompson
J N
JN
Richard
L A
LA
Chan
K W
KW
D000305:Adrenal Cortex Hormones / Q000627:therapeutic use; D000744:Anemia, Hemolytic, Autoimmune / Q000188:drug therapy / Q000209:etiology* / Q000276:immunology; D000918:Antibody Specificity; D001327:Autoimmune Diseases / Q000188:drug therapy / Q000209:etiology* / Q000276:immunology; D003298:Coombs Test; D004992:Ethics, Medical; D005312:Fetal Blood / Q000166:cytology*; D006025:Glycosaminoglycans / Q000652:urine; D006086:Graft vs Host Disease / Q000209:etiology; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects*; D006801:Humans; D007066:Iduronate Sulfatase / Q000235:genetics; D007074:Immunoglobulin G / Q000276:immunology; D007166:Immunosuppressive Agents / Q000627:therapeutic use; D007231:Infant, Newborn; D007258:Informed Consent; D008297:Male; D016532:Mucopolysaccharidosis II / Q000150:complications / Q000235:genetics / Q000628:therapy* / Q000652:urine; D017354:Point Mutation; D014184:Transplantation, Homologous / Q000009:adverse effects
D000305:Adrenal Cortex Hormones; D006025:Glycosaminoglycans; D007074:Immunoglobulin G; D007166:Immunosuppressive Agents; D007066:Iduronate Sulfatase