Russian pediatric experience on assessing the effectiveness of imiglucerase for long-term enzyme replacement therapy of gaucher disease type 1 in...
Мовсисян Г. Б., Намазова-Баранова Л. С., Савостянов К. В., Гундобина О. С., Семикина Е. Л., Рязанов М. В., Травина М. Л., Черников В. В., Пушков А. А., Букина Т. М.
Вестник Российской академии медицинских наук
Т. 72, Вып. 5, С. 383-392
Опубликовано: 2017
Тип ресурса: Статья
Аннотация:
Background: Today the gold standard for the treatment of Gaucher's disease (GD) is an enzyme replacement therapy (ERT) which allows to stop the main clinical manifestations of the disease and to improve the quality of life in patients. In Russian pediatric practice, there are no publications which assess the effects of long-term ERT in children with GD type 1. Aim: To evaluate the effectiveness of imiglucerase for the treatment of Gaucher's disease of type 1 in child population of the Russian Federation. Materials and methods: An evaluation of the effectiveness of enzyme replacement therapy was carried out by analyzing the monitoring data of 60 patients who were entered in the Russian pediatric registry of Gaucher disease at the National Scientific and Practical Center for Children's Health for the period 2013-2016. Patients received continuous infusions of imiglucerase at a dose of 30-60 U/kg/2 weeks. Among of 60 children with Gaucher's disease type 1, in 35 (group I) were recorded th
Ключевые слова:
Children; Enzyme replacement therapy; Gaucher disease; Imiglucerase; Quality of life
Язык текста: Русский
ISSN: 0869-6047
Мовсисян Г. Б.
Намазова-Баранова Л. С. Лейла Сеймуровна 1963-
Савостянов К. В.
Гундобина О. С.
Семикина Е. Л. Елена Леонидовна 1961-
Рязанов М. В.
Травина М. Л.
Черников В. В.
Пушков А. А.
Букина Т. М.
Movsisyan G. B.
Namazova-Baranova L. S. Lejla Sejmurovna 1963-
Savostyanov K. V.
Gundobina O. S.
Semikina E. L. Elena Leonidovna 1961-
Ryazanov M. V.
Travina M. L.
Chernikov V. V.
Pushkov A. A.
Bukina T. M.
Russian pediatric experience on assessing the effectiveness of imiglucerase for long-term enzyme replacement therapy of gaucher disease type 1 in children
Russian pediatric experience on assessing the effectiveness of imiglucerase for long-term enzyme replacement therapy of gaucher disease type 1 in...
Текст визуальный непосредственный
Вестник Российской академии медицинских наук
Издательство "Педиатръ"
Т. 72, Вып. 5 С. 383-392
2017
Статья
Children Enzyme replacement therapy Gaucher disease Imiglucerase Quality of life
Background: Today the gold standard for the treatment of Gaucher's disease (GD) is an enzyme replacement therapy (ERT) which allows to stop the main clinical manifestations of the disease and to improve the quality of life in patients. In Russian pediatric practice, there are no publications which assess the effects of long-term ERT in children with GD type 1. Aim: To evaluate the effectiveness of imiglucerase for the treatment of Gaucher's disease of type 1 in child population of the Russian Federation. Materials and methods: An evaluation of the effectiveness of enzyme replacement therapy was carried out by analyzing the monitoring data of 60 patients who were entered in the Russian pediatric registry of Gaucher disease at the National Scientific and Practical Center for Children's Health for the period 2013-2016. Patients received continuous infusions of imiglucerase at a dose of 30-60 U/kg/2 weeks. Among of 60 children with Gaucher's disease type 1, in 35 (group I) were recorded th