The effect of enzyme replacement therapy on clinical outcomes in male patients with Fabry disease: A systematic literature review by a European...
Germain D. P., Elliott P. M., Falissard B., Fomin V. V., Hilz M. J., Jovanovic A., Kantola I., Linhart A., Mignani R., Namdar M., Nowak A., Oliveira J. -., Pieroni M., Viana-Baptista M., Wanner C., Spada M.
Molecular Genetics and Metabolism Reports
Vol.19, Num.100454
Опубликовано: 2019
Тип ресурса: Обзор
DOI:10.1016/j.ymgmr.2019.100454
Аннотация:
Background: Enzyme replacement therapy (ERT) with recombinant human α-galactosidase has been available for the treatment of Fabry disease since 2001 in Europe and 2003 in the USA. Treatment outcomes with ERT are dependent on baseline patient characteristics, and published data are derived from heterogeneous study populations. Methods: We conducted a comprehensive systematic literature review of all original articles on ERT in the treatment of Fabry disease published up until January 2017. This article presents the findings in adult male patients. Results: Clinical evidence for the efficacy of ERT in adult male patients was available from 166 publications including 36 clinical trial publications. ERT significantly decreases globotriaosylceramide levels in plasma, urine, and in different kidney, heart, and skin cell types, slows the decline in estimated glomerular filtration rate, and reduces/stabilizes left ventricular mass and cardiac wall thickness. ERT also improves nervous system, g
Ключевые слова:
adult male patients; agalsidase alfa; agalsidase beta; enzyme replacement therapy; Fabry disease; systematic literature review
agalsidase alfa; agalsidase beta; galactosidase; globotriaosylceramide; albuminuria; cardiac muscle cell; cerebrovascular accident; clinical outcome; drug efficacy; enzyme replacement; estimated glomerular filtration rate; European; Fabry disease; heart left ventricle mass; human; kidney cell; male; proteinuria; quality of life; Review; systematic review; transient ischemic attack
Язык текста: Английский
ISSN: 2214-4269
Germain D. P.
Elliott P. M.
Falissard B.
Fomin V. V. Viktor Viktorovich 1978-
Hilz M. J.
Jovanovic A.
Kantola I.
Linhart A.
Mignani R.
Namdar M.
Nowak A.
Oliveira J. -. J.-P.
Pieroni M.
Viana-Baptista M.
Wanner C.
Spada M.
Гермаин Д. П.
Еллиотт П. М.
Фалиссард Б.
Фомин В. В. Виктор Викторович 1978-
Хилз М. Й.
Йовановиc А.
Кантола И.
Линхарт А.
Мигнани Р.
Намдар М.
Ноwак А.
Оливеира Й. -. Й.-П.
Пиерони М.
Виана-Баптиста М.
Wаннер C.
Спада М.
The effect of enzyme replacement therapy on clinical outcomes in male patients with Fabry disease: A systematic literature review by a European panel of experts
The effect of enzyme replacement therapy on clinical outcomes in male patients with Fabry disease: A systematic literature review by a European...
Текст визуальный непосредственный
Molecular Genetics and Metabolism Reports
Elsevier Inc.
Vol.19 Num.100454
2019
Обзор
adult male patients agalsidase alfa agalsidase beta enzyme replacement therapy Fabry disease systematic literature review
agalsidase alfa agalsidase beta galactosidase globotriaosylceramide albuminuria cardiac muscle cell cerebrovascular accident clinical outcome drug efficacy enzyme replacement estimated glomerular filtration rate European Fabry disease heart left ventricle mass human kidney cell male proteinuria quality of life Review systematic review transient ischemic attack
Background: Enzyme replacement therapy (ERT) with recombinant human α-galactosidase has been available for the treatment of Fabry disease since 2001 in Europe and 2003 in the USA. Treatment outcomes with ERT are dependent on baseline patient characteristics, and published data are derived from heterogeneous study populations. Methods: We conducted a comprehensive systematic literature review of all original articles on ERT in the treatment of Fabry disease published up until January 2017. This article presents the findings in adult male patients. Results: Clinical evidence for the efficacy of ERT in adult male patients was available from 166 publications including 36 clinical trial publications. ERT significantly decreases globotriaosylceramide levels in plasma, urine, and in different kidney, heart, and skin cell types, slows the decline in estimated glomerular filtration rate, and reduces/stabilizes left ventricular mass and cardiac wall thickness. ERT also improves nervous system, g