Combination of enzyme replacement and hematopoietic stem cell transplantation as therapy for Hurler syndrome.
Bone Marrow Transplant
; 41(6): 531-5, 2008 Mar.
Article
en En
| MEDLINE
| ID: mdl-18037941
ABSTRACT
Hurler syndrome (mucopolysaccharidosis type I, MPS IH) is characterized by a deficiency of alpha-L-iduronidase resulting in progressive multiorgan dysfunction. We sought to determine whether enzyme replacement therapy (ERT) with iduronidase in the peritransplant period affects outcome of hematopoietic stem cell transplantation (HSCT) for MPS IH. Seven children with MPS IH at a median age of 1.5 years at the time of myeloablative HSCT were eligible. All patients had null mutations in IDUA gene. Iduronidase (0.58 mg/kg per dose) was administered intravenously in 11-14 weekly doses before HSCT and 8 weekly doses after HSCT. The infusions were well tolerated. All patients developed antibodies to iduronidase but all engrafted with >90% donor hematopoiesis. A majority of patients had significant pulmonary complications before ERT and HSCT but all are alive and well with a median follow-up of more than 1 year after HSCT. This suggests that ERT prior to HSCT is unlikely to alter engraftment. In addition, morbidity was acceptable, despite a previous history of pulmonary difficulties that suggested that these patients were high risk for these complications. Therefore, we recommend treatment of MPS IH patients with combination of ERT and HSCT therapy to further investigate its potential to enhance outcomes with HSCT.
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Colección:
01-internacional
Base de datos:
MEDLINE
Asunto principal:
Mucopolisacaridosis I
/
Trasplante de Células Madre Hematopoyéticas
/
Iduronidasa
Tipo de estudio:
Etiology_studies
Límite:
Humans
/
Infant
Idioma:
En
Revista:
Bone Marrow Transplant
Asunto de la revista:
TRANSPLANTE
Año:
2008
Tipo del documento:
Article
País de afiliación:
Estados Unidos