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Hematopoietic Stem Cell Transplantation in Inborn Errors of Metabolism

Authors
  • Tan, Emily Y.1
  • Boelens, Jaap Jan2
  • Jones, Simon A.3
  • Wynn, Robert F.3
  • 1 Faculty of Health and Medical Sciences, University of Western Australia, Perth, WA , (Australia)
  • 2 Stem Cell Transplant and Cellular Therapies, Memorial Sloan Kettering Cancer Center, New York, NY , (United States)
  • 3 Metabolic and Blood and Marrow Transplant Units, Royal Manchester Children's Hospital, Manchester , (United Kingdom)
Type
Published Article
Journal
Frontiers in Pediatrics
Publisher
Frontiers Media SA
Publication Date
Oct 25, 2019
Volume
7
Identifiers
DOI: 10.3389/fped.2019.00433
PMID: 31709204
PMCID: PMC6824291
Source
PubMed Central
Keywords
License
Unknown

Abstract

Hematopoietic stem cell transplantation (HSCT) has been established as an effective therapy for selected inborn errors of metabolism. The success of HSCT in metabolic disease is best exemplified through the treatment of Hurler's syndrome, a lysosomal storage disease. Through the collaborative effort of several international centers, factors that predict successful patient and transplant outcomes have been identified. In this review, we discuss the principles that underlie the use of HSCT in metabolic diseases. We consider the clinical indications, conditioning regimens, and disease-specific follow-up for HSCT in different metabolic diseases. We highlight persisting challenges in HSCT to delay progression of certain organ systems that remain refractory to HSCT and the relatively high rates of aplastic graft failure. Finally, we evaluate the variable applicability of these principles to other inherited metabolic disorders including peroxisomal, mitochondrial, and other lysosomal storage diseases.

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