Klinische Pädiatrie
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Klinische Pädiatrie · May 2004
ReviewHematopoietic stem cell transplantation for mucopolysaccharidoses and leukodystrophies.
The only effective treatment for selected metabolic diseases is a successful allogeneic hematopoietic stem cell transplantation (HCT). Best results with HCT are obtained when performed early in the course of the disease. Hence, timely identification and referral are critical. ⋯ Hence, UCB transplant represents an alternative to marrow HCT. Related haploidentical HCT, possibly with very high doses of CD34+ cells, may also represent an option. However, expertise has been developed in very few transplant centers and no large reports are available of its use for patients with inherited metabolic diseases.