title: Hematopoietic cell transplantation for inherited metabolic diseases: an overview of outcomes and practice guidelines.
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), 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.
authors.affiliation: Department of Pediatrics, University of Minnesota School of Medicine, Minneapolis, 55455, USA.
authors.forename: C
authors.initials: C
authors.lastname: Peters
authors.forename: C G
authors.initials: CG
authors.lastname: Steward
mesh_terms: D002239:Carbohydrate Metabolism, Inborn Errors / Q000235:genetics / Q000628:therapy; D017408:Guidelines as Topic; D006801:Humans; D008052:Lipid Metabolism, Inborn Errors / Q000235:genetics / Q000628:therapy; D008661:Metabolism, Inborn Errors / Q000235:genetics* / Q000628:therapy*; D009083:Mucopolysaccharidoses / Q000235:genetics / Q000628:therapy; D033581:Stem Cell Transplantation / Q000379:methods / Q000592:standards*; D016896:Treatment Outcome