Hematopoietic stem cell transplantation improves the high incidence of neutralizing allo-antibodies observed in Hurler's syndrome after pharmacological enzyme replacement therapy.

@article{Saif2012HematopoieticSC,
  title={Hematopoietic stem cell transplantation improves the high incidence of neutralizing allo-antibodies observed in Hurler's syndrome after pharmacological enzyme replacement therapy.},
  author={Muhammad Ameer Saif and Brian W Bigger and Karen E Brookes and Jean Mercer and Karen L Tylee and Heather J. Church and Denise K. Bonney and Simon A Jones and James Ed Wraith and Robert Francis Wynn},
  journal={Haematologica},
  year={2012},
  volume={97 9},
  pages={1320-8}
}
BACKGROUND Mucopolysaccharidosis type I is caused by deficiency of α-L-iduronidase. Currently available treatment options include an allogeneic hematopoietic stem cell transplant and enzyme replacement therapy. Exogenous enzyme therapy appears promising but the benefits may be attenuated, at least in some patients, by the development of an immune response to the delivered enzyme. The incidence and impact of alloimmune responses in these patients remain unknown. DESIGN AND METHODS We developed… CONTINUE READING