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Clinical outcomes following hematopoietic stem cell transplantation for the treatment of mucopolysaccharidosis VI.

Publication ,  Journal Article
Turbeville, S; Nicely, H; Rizzo, JD; Pedersen, TL; Orchard, PJ; Horwitz, ME; Horwitz, EM; Veys, P; Bonfim, C; Al-Seraihy, A
Published in: Mol Genet Metab
February 2011

Mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy Syndrome) is one of approximately 50 known lysosomal storage disorders. MPS VI is characterized by an absence or deficiency of N-acetylgalactosamine 4-sulfatase (arylsulfatase B) resulting in accumulation of dermatan sulfate. Prior to the availability of enzyme replacement therapy (ERT), the clinical management of MPS VI was limited to supportive care and allogeneic hematopoietic stem cell transplantation (HSCT); however, due to the rarity of this disease, little is known about the long-term outcomes of HSCT for MPS VI. The following retrospective study was performed using aggregate data gathered by the Center for International Blood and Marrow Transplant Research (CIBMTR) between 1982 and 2007 to determine survival probability for patients with MPS VI following allogeneic HSCT. This analysis identified 45 MPS VI patients with a median age of 5 years (range, 1-22 years) at the time they received an allogeneic HSCT. Cumulative incidence (95% CI) of acute graft-vs.-host disease at 100 days was 36% (21-53%). Probability of survival was 78% (65-89%) at 100 days and 66% (52-79%) at 1 and 3 years. While these data are based upon small numbers of recipients, they represent the largest series to date and may help clinicians assess the relative risks and benefits of currently available therapies.

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Published In

Mol Genet Metab

DOI

EISSN

1096-7206

Publication Date

February 2011

Volume

102

Issue

2

Start / End Page

111 / 115

Location

United States

Related Subject Headings

  • Young Adult
  • Treatment Outcome
  • Transplantation, Homologous
  • Survival Analysis
  • Retrospective Studies
  • Neutrophils
  • Mucopolysaccharidosis VI
  • Male
  • Infant
  • Incidence
 

Citation

APA
Chicago
ICMJE
MLA
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Turbeville, S., Nicely, H., Rizzo, J. D., Pedersen, T. L., Orchard, P. J., Horwitz, M. E., … Al-Seraihy, A. (2011). Clinical outcomes following hematopoietic stem cell transplantation for the treatment of mucopolysaccharidosis VI. Mol Genet Metab, 102(2), 111–115. https://doi.org/10.1016/j.ymgme.2010.09.010
Turbeville, Sean, Helen Nicely, J Douglas Rizzo, Tanya L. Pedersen, Paul J. Orchard, Mitchell E. Horwitz, Edwin M. Horwitz, Paul Veys, Carmem Bonfim, and Amal Al-Seraihy. “Clinical outcomes following hematopoietic stem cell transplantation for the treatment of mucopolysaccharidosis VI.Mol Genet Metab 102, no. 2 (February 2011): 111–15. https://doi.org/10.1016/j.ymgme.2010.09.010.
Turbeville S, Nicely H, Rizzo JD, Pedersen TL, Orchard PJ, Horwitz ME, et al. Clinical outcomes following hematopoietic stem cell transplantation for the treatment of mucopolysaccharidosis VI. Mol Genet Metab. 2011 Feb;102(2):111–5.
Turbeville, Sean, et al. “Clinical outcomes following hematopoietic stem cell transplantation for the treatment of mucopolysaccharidosis VI.Mol Genet Metab, vol. 102, no. 2, Feb. 2011, pp. 111–15. Pubmed, doi:10.1016/j.ymgme.2010.09.010.
Turbeville S, Nicely H, Rizzo JD, Pedersen TL, Orchard PJ, Horwitz ME, Horwitz EM, Veys P, Bonfim C, Al-Seraihy A. Clinical outcomes following hematopoietic stem cell transplantation for the treatment of mucopolysaccharidosis VI. Mol Genet Metab. 2011 Feb;102(2):111–115.
Journal cover image

Published In

Mol Genet Metab

DOI

EISSN

1096-7206

Publication Date

February 2011

Volume

102

Issue

2

Start / End Page

111 / 115

Location

United States

Related Subject Headings

  • Young Adult
  • Treatment Outcome
  • Transplantation, Homologous
  • Survival Analysis
  • Retrospective Studies
  • Neutrophils
  • Mucopolysaccharidosis VI
  • Male
  • Infant
  • Incidence