Abstract | BACKGROUND: METHODS: Between December 1995 and October 2002, 20 consecutive children with Hurler's syndrome received busulfan, cyclophosphamide, and antithymocyte globulin before receiving cord-blood transplants from unrelated donors. The children were subsequently evaluated for engraftment, adverse effects, and effects on disease symptoms. RESULTS: CONCLUSIONS: Cord blood from unrelated donors appears to be an excellent source of stem cells for transplantation in patients with Hurler's syndrome. Sustained engraftment can be achieved without total-body irradiation. Cord-blood transplantation favorably altered the natural history of Hurler's syndrome and thus may be important to consider in young children with this form of the disease.
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Authors | Susan L Staba, Maria L Escolar, Michele Poe, Young Kim, Paul L Martin, Paul Szabolcs, June Allison-Thacker, Susan Wood, David A Wenger, Pablo Rubinstein, John J Hopwood, William Krivit, Joanne Kurtzberg |
Journal | The New England journal of medicine
(N Engl J Med)
Vol. 350
Issue 19
Pg. 1960-9
(May 06 2004)
ISSN: 1533-4406 [Electronic] United States |
PMID | 15128896
(Publication Type: Journal Article, Research Support, U.S. Gov't, P.H.S.)
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Copyright | Copyright 2004 Massachusetts Medical Society |
Chemical References |
- Immunosuppressive Agents
- Iduronidase
- Methylprednisolone
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Topics |
- Blood Donors
- Child Development
- Child, Preschool
- Cognition
- Disease-Free Survival
- Female
- Fetal Blood
(transplantation)
- Follow-Up Studies
- Graft vs Host Disease
(drug therapy, prevention & control)
- Growth
- Hematopoietic Stem Cell Transplantation
- Humans
- Iduronidase
(metabolism)
- Immunosuppressive Agents
(therapeutic use)
- Infant
- Infant, Newborn
- Male
- Methylprednisolone
(therapeutic use)
- Mucopolysaccharidosis I
(physiopathology, psychology, therapy)
- Neutrophils
- Transplantation Conditioning
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