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Miglustat therapy in type 1 Gaucher disease: Clinical and safety outcomes in a multicenter retrospective cohort study

Overview of attention for article published in Blood Cells, Molecules & Diseases, May 2013
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  • In the top 25% of all research outputs scored by Altmetric
  • Good Attention Score compared to outputs of the same age (76th percentile)

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1 policy source
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2 patents

Citations

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61 Dimensions

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47 Mendeley
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Title
Miglustat therapy in type 1 Gaucher disease: Clinical and safety outcomes in a multicenter retrospective cohort study
Published in
Blood Cells, Molecules & Diseases, May 2013
DOI 10.1016/j.bcmd.2013.04.005
Pubmed ID
Authors

David J. Kuter, Atul Mehta, Carla E.M. Hollak, Pilar Giraldo, Derralynn Hughes, Nadia Belmatoug, Monika Brand, Audrey Muller, Berthold Schaaf, Ruben Giorgino, Ari Zimran

Abstract

We evaluated clinical and safety outcomes in adult patients with type 1 Gaucher disease receiving miglustat in clinical practice settings. An observational, retrospective cohort study was conducted in centers across the EU and the USA. Medical chart data were collected from consecutive patients between the 20th November 2002 and 31st December 2008. A total of 115 patients were included; 34 (30%) were enzyme replacement therapy-naïve ('naïve') and 81 (70%) were enzyme pretreated ('pretreated'). Median (range) miglustat exposures in these groups were 15.1 (0.6-52.9)months and 15.2 (0.3-62.1)months, respectively. Low numbers of patients were anemic (10/101) or thrombocytopenic (21/101) at initiation of miglustat therapy. The median (range) hemoglobin concentration at miglustat initiation was 12.8 (10.2-16.4)g/dl in naïve patients and 13.6 (7.3-17.4)g/dl in pretreated patients; median (range) changes in hemoglobin were 0.3 (-2.5-3.6) and -0.3 (-4-4.6)g/dl, respectively. The median (range) platelet counts at miglustat initiation were 101 (37-730)×10(9)/l in naïve patients and 173 (43-382)×10(9)/l in pretreated patients; median (range) changes in platelet count were 8 (-77-145)×10(9)/l and -10 (-144-434)×10(9)/l, respectively. Plasma chitotriosidase was substantially reduced in naïve but not in pretreated patients. Organ volumes were not routinely monitored. Forty-nine (43%) patients discontinued miglustat; most due to gastrointestinal manifestations and some due to tremor. Overall, hemoglobin and platelet counts tended to increase in naïve patients treated with miglustat, and to remain stable or decrease slightly in pretreated patients. The profile of safety and tolerability observed with miglustat in the current study is similar to previous studies.

Mendeley readers

Mendeley readers

The data shown below were compiled from readership statistics for 47 Mendeley readers of this research output. Click here to see the associated Mendeley record.

Geographical breakdown

Country Count As %
Unknown 47 100%

Demographic breakdown

Readers by professional status Count As %
Other 7 15%
Student > Ph. D. Student 6 13%
Researcher 6 13%
Student > Postgraduate 5 11%
Student > Doctoral Student 4 9%
Other 8 17%
Unknown 11 23%
Readers by discipline Count As %
Medicine and Dentistry 13 28%
Agricultural and Biological Sciences 9 19%
Biochemistry, Genetics and Molecular Biology 4 9%
Pharmacology, Toxicology and Pharmaceutical Science 3 6%
Social Sciences 1 2%
Other 3 6%
Unknown 14 30%
Attention Score in Context

Attention Score in Context

This research output has an Altmetric Attention Score of 6. 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 01 March 2022.
All research outputs
#5,446,210
of 25,371,288 outputs
Outputs from Blood Cells, Molecules & Diseases
#102
of 821 outputs
Outputs of similar age
#44,403
of 205,979 outputs
Outputs of similar age from Blood Cells, Molecules & Diseases
#1
of 4 outputs
Altmetric has tracked 25,371,288 research outputs across all sources so far. Compared to these this one has done well and is in the 75th percentile: it's in the top 25% of all research outputs ever tracked by Altmetric.
So far Altmetric has tracked 821 research outputs from this source. They receive a mean Attention Score of 3.9. This one has done well, scoring higher than 77% of its peers.
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 205,979 tracked outputs that were published within six weeks on either side of this one in any source. This one has done well, scoring higher than 76% of its contemporaries.
We're also able to compare this research output to 4 others from the same source and published within six weeks on either side of this one. This one has scored higher than all of them