perinatologyРоссийский вестник перинатологии и педиатрииRossiyskiy Vestnik Perinatologii i Pediatrii (Russian Bulletin of Perinatology and Pediatrics)1027-40652500-2228Ltd. “The National Academy of Pediatric Science and Innovation”10.21508/1027-4065-2019-64-5-159-164perinatology-985Research ArticleОБЗОР ЛИТЕРАТУРЫREVIEWSТрансплантация гемопоэтических стволовых клеток у детей с синдромом ГурлерThe hematopoietic stem cell transplantation in children with Hurler syndromehttps://orcid.org/0000-0002-4147-2309ВолгинаС. Я.VolginaS. Ya.

д.м.н., проф. кафедры госпитальной педиатрии,

 

Kazan

Volgina_Svetlana@mail.ruhttps://orcid.org/0000-0002-6662-3548СадыковаД. И.SadykovaD. I.

д.м.н., проф., зав. кафедрой госпитальной педиатрии,

420011 Казань, ул. Бутлерова, д. 49

Kazan

https://orcid.org/0000-0001-7146-7220НиколаеваЕ. А.NikolaevaE. A.

д.м.н., рук. отдела клинической генетики,

125412 Москва, Талдомская ул., д. 2

Moscow

ПаламарчукЕ. И.PalamarchukE. I.

студентка,

119435 Москва, ул. Большая Пироговская, д. 2, стр. 9

Moscow

ФГБОУ ВО «Казанский государственный медицинский университет» Минздрава РоссииРоссияKazan State Medical UniversityRussian FederationОСП «Научно-исследовательский клинический институт педиатрии им. академика Ю.Е. Вельтищева» ФГБОУ ВО РНИМУ им. ПироговаРоссияVeltischev Research and Clinical Institute for Pediatrics of the Pirogov Russian National Research Medical UniversityRussian FederationФГАОУ ВО «Первый московский государственный медицинский университет им. И.М. Сеченова» Минздрава России (Сеченовский университет)РоссияThe First Moscow State Medical University (Sechenov University)Russian Federation201915112019645159164Copyright © Ltd. “The National Academy of Pediatric Science and Innovation”, 20192019Ltd. “The National Academy of Pediatric Science and Innovation”Ltd. “The National Academy of Pediatric Science and Innovation”https://www.ped-perinatology.ru/jour/about/submissions#copyrightNoticehttps://www.ped-perinatology.ru/jour/article/view/985

Статья посвящена вопросам трансплантации гемопоэтических стволовых клеток у детей с тяжелой формой мукополисахаридоза I типа – синдромом Гурлер. Показано, что этот метод лечения рекомендован для применения у пациентов в возрасте до 2,5 года с достаточно высоким коэффициентом психомоторного развития. 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. В настоящее время рекомендуется использовать режим кондиционирования сниженной интенсивности. Международное многоцентровое исследование оценило долгосрочный исход у больных с синдромом Гурлер и подтвердило улучшение качества и увеличение продолжительности жизни пациентов.

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.

детимукополисахаридоз I типа (синдром Гурлер)патогенетическое лечениетрансплантация гемопоэтических стволовых клетокchildrenmucopolysaccharidosis type I (Hurler syndrome)pathogenetic treatmenthematopoietic stem cell transplantationReferencesМукополисахаридоз I типа у детей. Клинические рекомендации, утвержденные Министерством здравоохранения. М., 2016; 36. [Mucopolysaccharidosis type I in children. Clinical guidelines, approved by the Ministry of health. Moscow, 2016; 36 (in Russ.)]Мукополисахаридоз I типа у детей. Клинические рекомендации, утвержденные Министерством здравоохранения. М., 2016; 36. [Mucopolysaccharidosis type I in children. Clinical guidelines, approved by the Ministry of health. Moscow, 2016; 36 (in Russ.)]Clarke L.A., Heppner J. Mucopolysaccharidosis Type I. GeneReviews.2016; http://www.ncbi.nlm.nih.gov/books/NBK1162/.Clarke L.A., Heppner J. Mucopolysaccharidosis Type I. GeneReviews.2016; http://www.ncbi.nlm.nih.gov/books/NBK1162/.Волгина С.Я. 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The authors declare that there are no conflicts of interest present.