Improved metabolic correction in patients with lysosomal storage disease treated with hematopoietic stem cell transplant compared with enzyme replacement therapy

Robert F Wynn; J Ed Wraith; Jean Mercer; Anne O'Meara; Karen Tylee; Margaret Thornley; Heather J Church; Brian W Bigger

doi:10.1016/j.jpeds.2008.11.005

Improved metabolic correction in patients with lysosomal storage disease treated with hematopoietic stem cell transplant compared with enzyme replacement therapy

J Pediatr. 2009 Apr;154(4):609-11. doi: 10.1016/j.jpeds.2008.11.005.

Authors

Robert F Wynn¹, J Ed Wraith, Jean Mercer, Anne O'Meara, Karen Tylee, Margaret Thornley, Heather J Church, Brian W Bigger

Affiliation

¹ Department of Haematology and BMT, Royal Manchester Children's Hospital, Manchester, United Kingdom. robert.wynn@cmmc.nhs.uk

PMID: 19324223
DOI: 10.1016/j.jpeds.2008.11.005

Abstract

We compared substrate reduction in patients with lysosomal storage disorder treated with hematopoietic stem cell transplant and found that it was significantly reduced compared with patients treated with pharmacological enzyme replacement therapy. These data might support the wider application of hematopoietic stem cell transplant in the treatment of lysosomal storage disorders.

Publication types

Clinical Trial
Comparative Study

MeSH terms

Biomarkers / urine
Chondroitin Sulfates / urine
Dermatan Sulfate / urine
Enzyme Therapy
Enzymes / blood
Hematopoietic Stem Cell Transplantation*
Humans
Mucopolysaccharidosis I / therapy*
Mucopolysaccharidosis II / therapy*
Mucopolysaccharidosis VI / therapy*

Substances

Biomarkers
Enzymes
Dermatan Sulfate
Chondroitin Sulfates