Magnetic resonance imaging of bone marrow changes in Gaucher disease during enzyme replacement therapy: first German long-term results

2001 ◽  
Vol 30 (9) ◽  
pp. 496-503 ◽  
Author(s):  
L. W. Poll ◽  
J.-A. Koch ◽  
S. vom Dahl ◽  
R.. Willers ◽  
A. Scherer ◽  
...  
Keyword(s):  
Magnetic Resonance Imaging ◽  
Bone Marrow ◽  
Magnetic Resonance ◽  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
Gaucher Disease ◽  
Long Term Results ◽  
Resonance Imaging ◽  
Enzyme Replacement

Bone marrow involvement in Gaucher disease at MRI : what long-term evolution can we expect under enzyme replacement therapy?

2015 ◽  
Vol 25 (10) ◽  
pp. 2969-2975 ◽  
Author(s):  
Benjamin Fedida ◽  
Sébastien Touraine ◽  
Jerôme Stirnemann ◽  
Nadia Belmatoug ◽  
Jean-Denis Laredo ◽  
...  
Keyword(s):  
Bone Marrow ◽  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
Gaucher Disease ◽  
Bone Marrow Involvement ◽  
Long Term Evolution ◽  
Enzyme Replacement ◽  
Term Evolution

Retrospective Analysis of Whole-Body Magnetic Resonance Imaging of Bone Manifestations in Long-Term Treated Patients with Gaucher Disease Type 1

2018 ◽  
Vol 231 (02) ◽  
pp. 52-59
Author(s):  
André Lollert ◽  
Katharina Laudemann ◽  
Eugen Mengel ◽  
Christian Hoffmann ◽  
Larissa Moos ◽  
...  
Keyword(s):  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
Gaucher Disease ◽  
Disease Type ◽  
Whole Body ◽  
Enzyme Replacement ◽  
Gaucher Disease Type 1 ◽  
Body Magnetic Resonance Imaging

Abstract Purpose We retrospectively assessed bone and visceral manifestations in patients with Gaucher disease type 1 (GD1) with whole-body magnetic resonance imaging (WB-MRI) to determine the effects of different timing in initiating long-term enzyme replacement therapy. Materials and Methods In 17 patients with GD1, we performed 2 WB-MRI examinations at a median interval of 13 months. Patients had received enzyme replacement therapy with alglucerase/imiglucerase for a median of 13 years prior to the first examination. MRI results were retrospectively stratified based on treatment initiation into 2 groups: “early” (age ≤12 years, median 5 years) and “late” (during adulthood, median 32 years). We evaluated occurrence of irreversible avascular necroses (AVN) and applied several semi-quantitative scores, including the Bone-Marrow-Burden (BMB) score, the Düsseldorf-Gaucher score (DGS), the Vertebra-Disc-Ratio (VDR), and the Gaucher disease type 1 Severity Scoring System (GD-DS3). Results MRI assessments showed no AVN in the “early” group. AVN were observed in 2 patients of the “late” group; one also had a splenic Gaucheroma. The follow-up examinations showed slight improvements in the BMB-score, DGS, and VDR, with similar tendencies in both treatment groups. The GD-DS3 score only improved in “late” group. Conclusion This retrospective study supported the ongoing clinical value of enzyme replacement therapy with alglucerase/imiglucerase, as WB-MRI-based scores stayed constant or slightly improved even after long-term treatment. Secondary complications were only observed in the late treatment group. Our results suggest that “early initiation” of enzyme replacement therapy may protect the bone.

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