Mini Review
Bone Marrow Transplantation (2003) 31, 229–239. doi:10.1038/sj.bmt.1703839
Hematopoietic cell transplantation for inherited metabolic diseases: an overview of outcomes and practice guidelines
C Peters1 and C G Steward2 on behalf of the NMDP, IBMTR, and the Working Party on Inborn Errors of the EBMT
- 1Division of Hematology/Oncology, Blood and Bone Marrow Transplantation, Department of Pediatrics, University of Minnesota School of Medicine, Minneapolis, MN, 55455, USA
- 2Bone Marrow Transplant Unit, Royal Hospital for Children, Upper Maudlin Street, Bristol, BS2 8BJ, UK
Correspondence: Dr Peters, Division of Hematology/Oncology, Blood and Bone Marrow Transplantation, Department of Pediatrics, University of Minnesota, Mayo Mail Code 477, Rm D-580 Mayo Building, 420 Delaware St SE, Minneapolis, MN 55455, USA
Received January 2002; Accepted 12 January 2002.
Abstract
For the past two decades, hematopoietic cell transplantation (HCT) has been used as effective therapy for selected inherited metabolic diseases (IMD) including Hurler (MPS IH) and Maroteaux–Lamy (MPS VI) syndromes, childhood-onset cerebral X-linked adrenoleukodystrophy (X-ALD), globoid-cell leukodystrophy (GLD), metachromatic leukodystrophy (MLD), 
-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.
Keywords:
inherited metabolic storage disease, mucopolysaccharidosis, Hurler syndrome, leukodystrophy, osteopetrosis, hematopoietic cell transplantation
