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Transplant outcomes in mucopolysaccharidoses.

Publication ,  Journal Article
Prasad, VK; Kurtzberg, J
Published in: Semin Hematol
January 2010

The mucopolysaccharidoses (MPSs) are inherited metabolic disorders (IMDs) caused by single-gene defects leading to progressive cellular accumulation of glycosaminoglycans (GAGs) and damage to multiple organs, including the central nervous, musculoskeletal, cardiorespiratory, and other systems. Hurler syndrome (MPS IH), the most severe form, is the prototypical model. Enzyme replacement therapy (ERT), available for MPS I, II, and VI, is beneficial in some patients. However, ERT does not improve neurocognitive function because of its inability to cross the blood-brain barrier. In contrast, allogeneic hematopoietic stem cell transplantation (HSCT) allows donor-derived, enzyme-producing cells to migrate to the brain and other organs to provide permanent enzyme therapy and thus help somatic organs, improve neurocognitive function and quality of life, and prolong survival, particularly when performed early in the course of the disease. Bone marrow has been the graft source in the past. However, in the last 5 years many patients have been treated with unrelated donor (URD) umbilical cord blood transplant (UCBT), allowing rapid and increased access to transplantation with favorable outcomes. This review describes published and our institutional clinical experiences, discusses the current status of the field, and provides therapy guidelines for patients with MPS.

Duke Scholars

Published In

Semin Hematol

DOI

EISSN

1532-8686

Publication Date

January 2010

Volume

47

Issue

1

Start / End Page

59 / 69

Location

United States

Related Subject Headings

  • Treatment Outcome
  • Transplantation, Homologous
  • Time Factors
  • Practice Guidelines as Topic
  • Patient Selection
  • Mucopolysaccharidoses
  • Immunology
  • Humans
  • Hematopoietic Stem Cell Transplantation
  • Cord Blood Stem Cell Transplantation
 

Citation

APA
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ICMJE
MLA
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Prasad, V. K., & Kurtzberg, J. (2010). Transplant outcomes in mucopolysaccharidoses. Semin Hematol, 47(1), 59–69. https://doi.org/10.1053/j.seminhematol.2009.10.008
Prasad, Vinod K., and Joanne Kurtzberg. “Transplant outcomes in mucopolysaccharidoses.Semin Hematol 47, no. 1 (January 2010): 59–69. https://doi.org/10.1053/j.seminhematol.2009.10.008.
Prasad VK, Kurtzberg J. Transplant outcomes in mucopolysaccharidoses. Semin Hematol. 2010 Jan;47(1):59–69.
Prasad, Vinod K., and Joanne Kurtzberg. “Transplant outcomes in mucopolysaccharidoses.Semin Hematol, vol. 47, no. 1, Jan. 2010, pp. 59–69. Pubmed, doi:10.1053/j.seminhematol.2009.10.008.
Prasad VK, Kurtzberg J. Transplant outcomes in mucopolysaccharidoses. Semin Hematol. 2010 Jan;47(1):59–69.
Journal cover image

Published In

Semin Hematol

DOI

EISSN

1532-8686

Publication Date

January 2010

Volume

47

Issue

1

Start / End Page

59 / 69

Location

United States

Related Subject Headings

  • Treatment Outcome
  • Transplantation, Homologous
  • Time Factors
  • Practice Guidelines as Topic
  • Patient Selection
  • Mucopolysaccharidoses
  • Immunology
  • Humans
  • Hematopoietic Stem Cell Transplantation
  • Cord Blood Stem Cell Transplantation