The results of allogeneic hematopoietic stem cell transplantation with reduced-intensity conditioning regimen doses in patients with Hurler syndrome

The article describes the transplantation of hematopoietic stem cells (HSC) in children with severe form of mucopolysaccharidosis type I – Hurler syndrome. This method is recommended for the patients under 2.5 years with a high rate of psychomotor development. According to clinical guidelines, HSC is performed according to traditional high-dose, chemotherapy-based conditioning regimens which provide intense immunosuppression to prevent graft rejection. Currently, it is recommended to use a reduced-intensity conditioning mode. An international multicenter study assessed the long-term outcome in patients with Hurler syndrome and confirmed improvements in life quality and expectancy.

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The results of allogeneic hematopoietic stem cell transplantation with reduced-intensity conditioning regimen doses in patients with Hurler syndrome

Cited by 2 publications

References 9 publications

Rhinosinusitis in Hurler syndrome patients requiring hematopoietic stem cells transplantation

Rhinosinusitis in Hurler syndrome patients requiring hematopoietic stem cells transplantation

The hematopoietic stem cell transplantation in children with Hurler syndrome

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