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Switching from imiglucerase to miglustat for the treatment of French patients with Gaucher disease type 1: a case series

Overview of attention for article published in Journal of Medical Case Reports, June 2015
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Title
Switching from imiglucerase to miglustat for the treatment of French patients with Gaucher disease type 1: a case series
Published in
Journal of Medical Case Reports, June 2015
DOI 10.1186/s13256-015-0617-5
Pubmed ID
Authors

Christine Serratrice, Laure Swiader, Jacques Serratrice

Abstract

Gaucher disease is caused by a deficiency of the enzyme β-glucocerebrosidase. Treatment with enzyme replacement therapy has been available for the past two decades but, although effective, enzyme replacement therapy can be delivered only by intravenous infusion every other week. The oral substrate reduction therapy miglustat (Zavesca®) has been available in Europe since 2002 for the treatment of patients with mild or moderate Gaucher disease type 1 for whom enzyme replacement therapy is unsuitable or not a therapeutic option. There are few published real-world data on the use of miglustat as a maintenance therapy in Gaucher disease type 1 patients switched from previous enzyme replacement therapy. We report a case series of three patients who were switched from long-term enzyme replacement therapy to miglustat for various reasons. All three patients were Caucasian and had confirmed Gaucher disease type 1. An 80-year-old man requested a switch to oral miglustat therapy in preference to ongoing intravenous enzyme replacement therapy, a 57-year-old woman was commenced on miglustat due to a shortage of imiglucerase, and a 56-year-old woman was switched from previous enzyme replacement therapy due to allergic reactions to intravenous infusions. Haematological disease parameters were stable in each patient on previous enzyme replacement therapy. Two patients continue to be treated with miglustat, having shown good tolerability and stable core disease parameters for approximately 4 years. One patient, who was also stable during 7 years of therapy, eventually discontinued miglustat as a precaution because he developed peripheral neuropathy of as yet unknown origin. Overall, our experience indicates that miglustat can be used as maintenance therapy for Gaucher disease type 1 after initial enzyme replacement therapy, but the selection of patients to whom this approach should be applied should be made after careful consideration of all disease parameters.

Twitter Demographics

The data shown below were collected from the profile of 1 tweeter who shared this research output. Click here to find out more about how the information was compiled.

Mendeley readers

The data shown below were compiled from readership statistics for 16 Mendeley readers of this research output. Click here to see the associated Mendeley record.

Geographical breakdown

Country Count As %
Unknown 16 100%

Demographic breakdown

Readers by professional status Count As %
Student > Bachelor 6 38%
Student > Ph. D. Student 2 13%
Other 1 6%
Student > Doctoral Student 1 6%
Lecturer 1 6%
Other 2 13%
Unknown 3 19%
Readers by discipline Count As %
Medicine and Dentistry 5 31%
Pharmacology, Toxicology and Pharmaceutical Science 2 13%
Biochemistry, Genetics and Molecular Biology 2 13%
Neuroscience 2 13%
Nursing and Health Professions 1 6%
Other 1 6%
Unknown 3 19%

Attention Score in Context

This research output has an Altmetric Attention Score of 1. This is our high-level measure of the quality and quantity of online attention that it has received. This Attention Score, as well as the ranking and number of research outputs shown below, was calculated when the research output was last mentioned on 26 March 2016.
All research outputs
#5,631,357
of 7,441,664 outputs
Outputs from Journal of Medical Case Reports
#764
of 1,374 outputs
Outputs of similar age
#194,342
of 276,433 outputs
Outputs of similar age from Journal of Medical Case Reports
#26
of 44 outputs
Altmetric has tracked 7,441,664 research outputs across all sources so far. This one is in the 13th percentile – i.e., 13% of other outputs scored the same or lower than it.
So far Altmetric has tracked 1,374 research outputs from this source. They receive a mean Attention Score of 3.3. This one is in the 27th percentile – i.e., 27% of its peers scored the same or lower than it.
Older research outputs will score higher simply because they've had more time to accumulate mentions. To account for age we can compare this Altmetric Attention Score to the 276,433 tracked outputs that were published within six weeks on either side of this one in any source. This one is in the 15th percentile – i.e., 15% of its contemporaries scored the same or lower than it.
We're also able to compare this research output to 44 others from the same source and published within six weeks on either side of this one. This one is in the 25th percentile – i.e., 25% of its contemporaries scored the same or lower than it.