Journal
BONE MARROW TRANSPLANTATION
Volume 31, Issue 4, Pages 229-239Publisher
NATURE PUBLISHING GROUP
DOI: 10.1038/sj.bmt.1703839
Keywords
inherited metabolic storage disease; mucopolysaccharidosis; Hurler syndrome; leukodystrophy; osteopetrosis; hematopoietic cell transplantation
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For the past two decades, hematopoietic cell transplantation (HCT) has been used as effective therapy for selected inherited metabolic diseases (IMD) including Hurler (NIPS IH) and Maroteaux-Lamy (NIPS VI) syndromes, childhood-onset cerebral X-linked adrenoleukodystrophy (X-ALD), globoid-cell leukodystrophy (GLD), metachromatic leukodystrophy (MLD), alpha-mannosidosis, osteopetrosis, and others. Careful pre-HCT evaluation is critical and coordinated, multidisciplinary follow-up is essential in this field of transplantation. The primary goals of HCT for these disorders have been to promote long-term survival with donor-derived engraftment and to optimize the quality of life. Guidelines for HCT and monitoring are provided; a brief overview of long-term results is also presented.
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