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Enzyme replacement therapy with galsulfase for mucopolysaccharidosis type VI

Overview of attention for article published in Cochrane database of systematic reviews, March 2016
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About this Attention Score

  • In the top 25% of all research outputs scored by Altmetric
  • High Attention Score compared to outputs of the same age (87th percentile)
  • Above-average Attention Score compared to outputs of the same age and source (58th percentile)

Mentioned by

blogs
1 blog
twitter
4 tweeters
facebook
1 Facebook page

Citations

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13 Dimensions

Readers on

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95 Mendeley
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Title
Enzyme replacement therapy with galsulfase for mucopolysaccharidosis type VI
Published in
Cochrane database of systematic reviews, March 2016
DOI 10.1002/14651858.cd009806.pub2
Pubmed ID
Authors

Marcela Junqueira Brunelli, Álvaro N Atallah, Edina MK da Silva

Abstract

Mucopolysaccharidosis type VI or Maroteaux-Lamy syndrome is a rare genetic disorder caused by the deficiency of arylsulphatase B. The resultant accumulation of dermatan sulphate causes lysosomal damage.The clinical symptoms are related to skeletal dysplasia (i.e. short stature and degenerative joint disease). Other manifestations include cardiac disease, impaired pulmonary function, ophthalmological complications, hepatosplenomegaly, sinusitis, otitis, hearing loss and sleep apnea. Intellectual impairment is generally absent. Clinical manifestation is typically by two or three years of age; however, slowly progressive cases may not present until adulthood.Enzyme replacement therapy with galsulfase is considered a new approach for treating mucopolysaccharidosis type VI. To evaluate the effectiveness and safety of treating mucopolysaccharidosis VI by enzyme replacement therapy with galsulfase compared to other interventions, placebo or no intervention. Eletronic searches were performed on the Cystic Fibrosis and Genetic Disorders Group's Inborn Errors of Metabolism Trials Register, in CENTRAL, MEDLINE, LILACS, the Journal of Inherited Metabolic Disease and ClinicalTrials.gov. Date of the last search of the Cystic Fibrosis and Genetic Disorders Group's Inborn Errors of Metabolism Trials Register: 05 February 2016. Randomized and quasi-randomized controlled clinical studies of enzyme replacement therapy with galsulfase compared to other interventions or placebo. Two authors independently screened the studies, assessed the risk of bias and extracted data. One study was included involving 39 participants who received either enzyme replacement therapy with galsulfase (recombinant human arylsulphatase B) or placebo. This small study was considered to be of overall unclear quality, since the authors did not report how both the allocation generation and concealment were performed.The key finding at 24 weeks in the 12-minute walk test was a statistically significant mean difference of 92.00 meters between the two groups in favour of the galsulfase group (95% confidence interval 11.00 to 172.00). While week 24 results for the three-minute stair climb demonstrated some improvement in the treatment group as compared to the placebo group, this was not significant, mean difference 5.70 (95% confidence interval -0.10 to 11.50).A significant decrease in the urinary glycosaminoglycan levels was observed in favour of the galsulfase group at 24 weeks, mean difference -227.00 (95% confidence interval -264.00 to -190.00).In general, the dose of galsulfase was well tolerated and there were no significant differences in relation to adverse events. These events include drug-related adverse events, serious and severe adverse events, those during infusion, drug-related adverse events during infusion, and deaths. More infusion-related reactions were observed in the galsulfase group and were managed with interruption or slowing of infusion rate or administration of antihistamines or corticosteroids drugs. No deaths occurred during the study. The results of one small study (based on 24-week randomised phase of the study and prior to the open-label extension) demonstrated that galsulfase is more effective than placebo in people with MPS VI, with significant improvements in the 12-minute walk test and a reduction in urinary glycosaminoglycans.There were no significant changes in cardiac or pulmonary functions, liver or spleen volume, overnight apnea-hypopnea, height and weight, quality of life and adverse effects.Further studies are needed to obtain more information on the long-term effectiveness and safety of enzyme replacement therapy with galsulfase.

Twitter Demographics

The data shown below were collected from the profiles of 4 tweeters 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 95 Mendeley readers of this research output. Click here to see the associated Mendeley record.

Geographical breakdown

Country Count As %
Ecuador 1 1%
Unknown 94 99%

Demographic breakdown

Readers by professional status Count As %
Student > Master 23 24%
Unspecified 12 13%
Student > Bachelor 12 13%
Researcher 11 12%
Student > Doctoral Student 8 8%
Other 28 29%
Unknown 1 1%
Readers by discipline Count As %
Medicine and Dentistry 41 43%
Unspecified 19 20%
Psychology 7 7%
Biochemistry, Genetics and Molecular Biology 5 5%
Agricultural and Biological Sciences 5 5%
Other 17 18%
Unknown 1 1%

Attention Score in Context

This research output has an Altmetric Attention Score of 13. 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 20 May 2018.
All research outputs
#1,111,310
of 12,968,481 outputs
Outputs from Cochrane database of systematic reviews
#3,443
of 10,419 outputs
Outputs of similar age
#32,229
of 267,915 outputs
Outputs of similar age from Cochrane database of systematic reviews
#79
of 192 outputs
Altmetric has tracked 12,968,481 research outputs across all sources so far. Compared to these this one has done particularly well and is in the 91st percentile: it's in the top 10% of all research outputs ever tracked by Altmetric.
So far Altmetric has tracked 10,419 research outputs from this source. They typically receive a lot more attention than average, with a mean Attention Score of 20.5. This one has gotten more attention than average, scoring higher than 66% 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 267,915 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 87% of its contemporaries.
We're also able to compare this research output to 192 others from the same source and published within six weeks on either side of this one. This one has gotten more attention than average, scoring higher than 58% of its contemporaries.