Use of enzyme replacement therapy (Laronidase) before hematopoietic stem cell transplantation for mucopolysaccharidosis I: experience in 18 patients.
case_series · Level IV
Where this comes from
- Record sourced from PubMed, PMID 19187736.
- Also identified by DOI 10.1016/j.jpeds.2008.07.004.
- No licence information is recorded for this record.
- Because redistribution is not established, this page shows the abstract only. Follow the links below for the full text.
Abstract
We describe the use of enzyme replacement therapy in conjunction with hematopoietic stem cell transplantation in 18 consecutive patients with severe mucopolysaccharidosis type I. The survival and engraftment rate was 89% overall and 93% for the 15 patients who received full-intensity conditioning.
Medical subject headings
- Hematopoietic Stem Cell Transplantation
- Iduronidase
- Mucopolysaccharidosis I
- Neoadjuvant Therapy