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Modeling the effect of enzyme replacement therapy on life-threatening complications in patients with Fabry disease

https://doi.org/10.17749/2070-4909.2018.11.4.038-046

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Abstract

Fabry disease (FD) is a severe lysosome storage disease caused by congenital deficiency of the enzyme α-galactosidase A and characterized by the risk of renal failure combined with cardiovascular and CNS complications. According to the currently available information, the early start of enzyme replacement therapy (ERT) leads to a significant improvement in patient’s condition.

The aim of the study is to assess whether the timely ERT prevents severe FD complications and to calculate the number of prevented cases as depending on the time of ERT start.

Materials and methods. The proposed model is based on the published results on patients with FD, receiving agalsidase alpha as ERT (no data for agalsidase beta was found). The expected number of cases with life-threatening complications was calculated for different starting timepoints and durations of the ERT.

Results. In patients with FD, continuous ERT during five years reduces the number of serious cardiovascular and renal complications by 25%. An early start of ERT makes it possible to additionally (as compared with a late start) prevent the complications in more than 20% of cases.

Conclusion. The early initiation of RPT in patients with FD can significantly reduce the occurrence of severe lifethreatening complications, increase the patients’ survival and improve their quality of life. 

About the Authors

V. I. Ignatyeva
Russian Presidential Academy of National Economy and Public Administration, Federal State Educational Institution of Higher Professional Education
Russian Federation

Viktoria I. Ignatyeva – Researcher at the Center for Health Technology Assessment,

82 Vernadskogo prospect, Moscow 119571



S. V. Moiseev
I.M. Sechenov First Moscow State Medical University, Ministry of Health of Russia
Russian Federation

Sergey V. Moiseev – MD, Doctor of Medical Science, Head of the Department of Internal & Occupational Diseases and Rheumatology,

11/5 Rossolimo Str., Moscow 119435



N. M. Bulanov
I.M. Sechenov First Moscow State Medical University, Ministry of Health of Russia
Russian Federation

Nikolay M. Bulanov – MD, Assistant, Department of Internal & Occupational Diseases and Rheumatology,

Scopus Author ID: 43461093400;

11/5 Rossolimo Str., Moscow 119435



E. A. Karovajkina
I.M. Sechenov First Moscow State Medical University, Ministry of Health of Russia
Russian Federation

Ekaterina A. Karovaikina – MD, Assistant, Department of Internal & Occupational Diseases and Rheumatology,

11/5 Rossolimo Str., Moscow 119435



A S. Moiseev
Lomonosov Moscow State University
Russian Federation

Alexey S. Moiseev – Clinical Intern, Faculty of Fundamental Medicine, 

1 Leninskie gory, Moscow 119991



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For citation:


Ignatyeva V.I., Moiseev S.V., Bulanov N.M., Karovajkina E.A., Moiseev A.S. Modeling the effect of enzyme replacement therapy on life-threatening complications in patients with Fabry disease. FARMAKOEKONOMIKA. Modern Pharmacoeconomic and Pharmacoepidemiology. 2018;11(4):38-46. (In Russ.) https://doi.org/10.17749/2070-4909.2018.11.4.038-046

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