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Ryzhov A.O.

Center for Expertise and Quality Control of Medical Care

Fedyaeva V.K.

Center for Expertise and Quality Control of Medical Care;
Financial Research Institute

Enzyme replacement therapy at reduced doses in pediatric patients with Gaucher disease type 1: a systematic review

Authors:

Ryzhov A.O., Fedyaeva V.K.

More about the authors

Journal: Medical Technologies. Assessment and Choice. 2020;42(4): 75‑86

DOI: 10.17116/medtech20204204175

Read: 1071 times

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Table of contents

ENZYME REPLACEMENT THERAPY AT REDUCED DOSES IN PEDIATRIC PATIENTS WITH GAUCHER DISEASE TYPE 1: A SYSTEMATIC REVIEW
ENZYME REPLACEMENT THERAPY AT REDUCED DOSES IN PEDIATRIC PATIENTS WITH GAUCHER DISEASE TYPE 1: A SYSTEMATIC REVIEW

To cite this article:

Ryzhov AO, Fedyaeva VK. Enzyme replacement therapy at reduced doses in pediatric patients with Gaucher disease type 1: a systematic review. Medical Technologies. Assessment and Choice. 2020;42(4):75‑86. (In Russ.)
https://doi.org/10.17116/medtech20204204175

Подписка 2026 Медицинские технологии. Оценка и выбор (Medical Technologies. Assessment and Choice)
МСФ на ЯМ
Использование инфографики авторами научных работ
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Abstract:

OBJECTIVE

To analyze clinical efficacy of enzyme replacement therapy (ERT) at reduced doses in children with Gaucher disease type 1.

MATERIAL AND METHODS

A systematic review is based on 7 studies. Inclusion criteria were the following: Gaucher disease type 1 in pediatric patients, enzyme replacement therapy at a low dosage compared to standard ones. We analyzed laboratory parameters (hemoglobin, platelet count, serum chitotriosidase), liver and spleen dimensions. Data are described as mean and confidence interval.

RESULTS

In most studies, there were no significant differences between the low-dose ERT and standard-dose ERT at the initial and final stage of follow-up.

CONCLUSION

Efficacy of low-dose and standard-dose ERT is comparable in terms of the most important clinical indicators. Pooled analysis of data should be considered in order to increase statistical power, as well as to analyze comparative safety of these approaches.

Keywords:

Gaucher disease type 1
enzyme replacement therapy
children

Authors:

Ryzhov A.O.

Center for Expertise and Quality Control of Medical Care

Fedyaeva V.K.

Center for Expertise and Quality Control of Medical Care;
Financial Research Institute

Received:

30.07.2020

Accepted:

22.08.2020

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References:

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