Long-term outcome of Hurler syndrome patients after hematopoietic cell transplantation: an international multicenter study.

@article{Aldenhoven2015LongtermOO,
  title={Long-term outcome of Hurler syndrome patients after hematopoietic cell transplantation: an international multicenter study.},
  author={Mieke Aldenhoven and Robert Francis Wynn and P J Orchard and Anne O'meara and Paul A. Veys and Alain Fischer and Vassili Valayannopoulos and B{\'e}n{\'e}dicte Neven and Attilio Maria Rovelli and V. Prasad and Jakub Tolar and Heather A Allewelt and Simon A Jones and Rossella Parini and Marleen M Renard and Victoria Bordon and Nico Martinus Wulffraat and Tom J. de Koning and Elsa G. Shapiro and Joanne Kurtzberg and Jaap Jan Boelens},
  journal={Blood},
  year={2015},
  volume={125 13},
  pages={
          2164-72
        }
}
Mucopolysaccharidosis type I-Hurler syndrome (MPS-IH) is a lysosomal storage disease characterized by multisystem morbidity and death in early childhood. Although hematopoietic cell transplantation (HCT) has been performed in these patients for more than 30 years, large studies on the long-term outcome of patients with MPS-IH after HCT are lacking. The goal of this international study was to identify predictors of the long-term outcome of patients with MPS-IH after successful HCT. Two hundred… CONTINUE READING
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References

Publications referenced by this paper.
Showing 1-10 of 26 references

Cord-blood transplants from unrelated donors in patients with Hurler's syndrome.

The New England journal of medicine • 2004
View 4 Excerpts
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HSCT in inborn errors of metabolism and osteopetrosis

JJ Boelens, M Bierings, R. Wynn
2012
View 1 Excerpt