Use of Enzyme Replacement Therapy (Laronidase) before Hematopoietic Stem Cell Transplantation for Mucopolysaccharidosis I: Experience in 18 Patients

Autor:	Robert Wynn, Trevor F. Carr, Joanne Page, J. Edmond Wraith, Simon Jones, Jean Mercer
Rok vydání:	2009
Předmět:	Male medicine.medical_specialty Transplantation Conditioning Mucopolysaccharidosis I medicine.medical_treatment Mucopolysaccharidosis Hematopoietic stem cell transplantation Iduronidase Mucopolysaccharidosis type I medicine Humans business.industry Hematopoietic Stem Cell Transplantation Infant Enzyme replacement therapy medicine.disease Survival Analysis Neoadjuvant Therapy Surgery Treatment Outcome Graft-versus-host disease Pediatrics Perinatology and Child Health Female Stem cell business
Zdroj:	The Journal of Pediatrics. 154:135-139
ISSN:	0022-3476
DOI:	10.1016/j.jpeds.2008.07.004
Popis:	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.
Databáze:	OpenAIRE
Externí odkaz:	https://explore.openaire.eu/search/publication?articleId=doi_dedup___::711e12c437a0ffe27a584e81202e6e1a https://doi.org/10.1016/j.jpeds.2008.07.004 Zobrazit plný text záznamu Full Text from ScienceDirect