Open AccessThe hematopoietic stem cell transplantation in children with Hurler syndrome
Author(s) -
С. Я. Волгина,
Д. И. Садыкова,
Е. А. Николаева,
Е. И. Паламарчук
Publication year - 2019
Publication title -
rossijskij vestnik perinatologii i pediatrii
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.139
H-Index - 4
eISSN - 2500-2228
pISSN - 1027-4065
DOI - 10.21508/1027-4065-2019-64-5-159-164
Subject(s) - hurler syndrome , medicine , hematopoietic stem cell transplantation , mucopolysaccharidosis type i , transplantation , stem cell , pediatrics , mucopolysaccharidosis i , immunosuppression , mucopolysaccharidosis , life expectancy , immunology , oncology , enzyme replacement therapy , disease , population , biology , environmental health , genetics
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.