Abstract
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.
Highlights
The article describes the transplantation of hematopoietic stem cells (HSC) in children with severe form of mucopolysaccharidosis type I – Hurler syndrome
HSC is performed according to traditional high-dose, chemotherapy-based conditioning regimens which provide intense immunosuppression to prevent graft rejection
The severity of clinical manifestations depending on the age of debut of mucopolysaccharidosis type I [1]
Summary
Статья посвящена вопросам трансплантации гемопоэтических стволовых клеток у детей с тяжелой формой мукополисахаридоза I типа – синдромом Гурлер. Согласно клиническим рекомендациям трансплантацию гемопоэтических стволовых клеток проводят в соответствии с традиционными высокодозовыми схемами кондиционирования, которые были разработаны, чтобы обеспечить интенсивную иммуносупрессию для предотвращения отторжения трансплантата. Ключевые слова: дети, мукополисахаридоз I типа (синдром Гурлер), патогенетическое лечение, трансплантация гемопоэтических стволовых клеток. Трансплантация гемопоэтических стволовых клеток у детей с синдромом Гурлер. 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. РОССИЙСКИЙ ВЕСТНИК ПЕРИНАТОЛОГИИ И ПЕДИАТРИИ, 2019; 64:(5) ROSSIYSKIY VESTNIK PERINATOLOGII I PEDIATRII, 2019; 64:(5)
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