ppharmПедиатрическая фармакологияPediatric pharmacology1727-57762500-3089Издательство «ПедиатрЪ»10.15690/pf.v11i3.1014ppharm-103Research ArticleВ ПРАКТИКУ ПЕДИАТРАFor Pediatricians' PracticeОЦЕНКА ЭФФЕКТИВНОСТИ ФЕРМЕНТОЗАМЕСТИТЕЛЬНОЙ ТЕРАПИИ У ДЕТЕЙ С БОЛЕЗНЬЮ ГОШЕ ПО ДАННЫМ МЕЖДУНАРОДНЫХ ИССЛЕДОВАНИЙEVALUATION OF ENZYME REPLACEMENT THERAPY EFFECTIVENESS IN CHILDREN WITH GAUCHER’S DISEASE ACCORDING TO THE INTERNATIONAL STUDIESГундобинаО. С.GundobinaO. S.

кандидат медицинских наук, заведующая отделением восстановительного лечения детей с болезнями органовпищеварительной системы НИИ профилактической педиатрии и восстановительного лечения ФГБУ «Научный центр здоровья детей»

MD, head of the department of medical rehabilitation for children with diseases of digestive organs of the research institute of preventive pediatrics and medical rehabilitation of the Scientific Center of Children’s Health (Federal State Budgetary Institution)

gundobina@nczd.ruМовсисянГ. Б.MovsisyanG. B.Намазова-БарановаЛ. С.Namazova-BaranovaL. S.Научный центр здоровья детей, МоскваРоссияScientific Center of Children’s Health, MoscowRussian FederationНаучный центр здоровья детей, Москва, Российская ФедерацияРоссияScientific Center of Children’s Health, Moscow, Russian FederationRussian Federation201403062014113Педиатрическая фармакология8084Copyright © Гундобина О.С., Мовсисян Г.Б., Намазова-Баранова Л.С., 20142014Гундобина О.С., Мовсисян Г.Б., Намазова-Баранова Л.С.Gundobina O.S., Movsisyan G.B., Namazova-Baranova L.S.Данная работа распространяется под лицензией Creative Commons Attribution 4.0.This work is licensed under a Creative Commons Attribution 4.0 License.https://www.pedpharma.ru/jour/article/view/103

The article presents data on the history of creation of pathogenetic enzyme replacement therapy and its introduction into clinical practice of managing patients with Gaucher’s disease. 2 primary stages are distinguished: beginning of use of enzyme β-D-glycosidase analog obtained from placenta and alglucerase; introduction of recombinant glucocerebrosidase (imiglucerase). The article demonstrates that enzyme replacement therapy is the only efficient method of treating Gaucher’s disease; according to the international studies; it terminates the primary clinical manifestations of the disease, thus improving quality of life of the patients without any marked side effects. Imiglucerase is used at present; it causes hydrolysis of glycolipid glucocerebroside down to glucose and ceramide by the common way of metabolism of membrane lipids. Imiglucerase is indicated for long-term enzyme replacement therapy in patients with confirmed Gaucher’s disease (types 1 and 3).

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The authors declare that there are no conflicts of interest present.