Evaluation of treatment response to enzyme replacement therapy with Velaglucerase alfa in patients with Gaucher disease using whole-body magnetic resonance imaging

被引：7

作者：

Laudemann, K. ^{[1
]}

Moos, L. ^{[1
]}

Mengel, E. ^{[2
]}

Lollert, A. ^{[1
]}

Hoffmann, C. ^{[1
]}

Brixius-Huth, M. ^{[2
]}

Wagner, D. ^{[1
]}

Dueber, C. ^{[3
]}

Staatz, G. ^{[1
]}

机构：

[1] Johannes Gutenberg Univ Mainz, Med Ctr, Sect Pediat Radiol, Dept Diagnost & Intervent Radiol, Langenbeckstr 1, D-55131 Mainz, Germany

[2] Johannes Gutenberg Univ Mainz, Med Ctr, Ctr Pediat & Adolescent Med, Villa Metab, D-55131 Mainz, Germany

[3] Johannes Gutenberg Univ Mainz, Med Ctr, Dept Diagnost & Intervent Radiol, D-55131 Mainz, Germany

来源：

BLOOD CELLS MOLECULES AND DISEASES | 2016年 / 57卷

关键词：

Velaglucerase alfa; Whole-body MRI; Score systems; Gaucher disease; MARROW BURDEN SCORE; BONE-MARROW; SKELETAL INVOLVEMENT; IMIGLUCERASE; QUANTIFICATION; PARAMETER; CHILDREN;

D O I：

10.1016/j.bcmd.2015.11.003

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

Objective: This was a retrospective data analysis to evaluate the treatment response to enzyme replacement therapy (ERT) with Velaglucerase alfa using whole-body magnetic resonance imaging (MRI). Materials and methods: A baseline and follow-up MRI were performed on 18 Gaucher Type 1 patients at an interval of 11.6 months. The MRI score systems determined the Bone-Marrow-Burden (BMB) score, the Dusseldorf-Daucher score (DGS), and the Vertebra-Disc-Ratio (VDR). The Severity Score Index Type 1 (GD-DS3) was also assessed. Results: The baseline MRI medians were: BMB, 7.00; DGS, 3.00; and VDR: 1.70; while, the follow-up MRI medians were: BMB, 7.00; DGS, 3.00; and VDR: 1.73. The baseline GD-DS3 median was 2.40 (BMB excl.: 0.50) and the follow-up median was 2.00 (BMB excl.: 0.50). There was weak statistical significance with the Wilcoxon signed-rank test for the DGS (p = 0.034) and GD-DS3 (p = 0.047) between both MRIs. Conclusion: Velaglucerase alfa therapy is a effective long-term treatment for Gaucher Type 1 patients who are newly diagnosed or switching therapies. Measurements with whole-body MRI and an objective scoring system were reliable tools for detecting early stage bone marrow activity. Further research is needed to evaluate the "Booster-Effect" of Velaglucerase alfa therapy in Gaucher skeletal disease. (C) 2015 Elsevier Inc All rights reserved.

引用

页码：35 / 41

页数：7

共 28 条

[1] Velaglucerase alfa enzyme replacement therapy compared with imiglucerase in patients with Gaucher disease [J].