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Cochrane Database of Systematic Reviews

Enzyme replacement therapy with laronidase (Aldurazyme®) for treating mucopolysaccharidosis type I

Overview of attention for article published in Cochrane database of systematic reviews, April 2016
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  • In the top 25% of all research outputs scored by Altmetric
  • High Attention Score compared to outputs of the same age (83rd percentile)
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Mentioned by

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1 policy source
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6 X users
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2 Facebook pages
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1 Wikipedia page

Citations

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35 Dimensions

Readers on

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114 Mendeley
Title
Enzyme replacement therapy with laronidase (Aldurazyme<sup>®</sup>) for treating mucopolysaccharidosis type I
Published in
Cochrane database of systematic reviews, April 2016
DOI 10.1002/14651858.cd009354.pub4
Pubmed ID
Authors

Elisabeth Jameson, Simon Jones, Tracey Remmington

Abstract

Mucopolysaccharidosis type I can be classified as three clinical sub-types; Hurler syndrome, Hurler-Scheie syndrome and Scheie syndrome, with the scale of severity being such that Hurler syndrome is the most severe and Scheie syndrome the least severe. It is a rare, autosomal recessive disorder caused by a deficiency of alpha-L-iduronidase. Deficiency of this enzyme results in the accumulation of glycosaminoglycans within the tissues. The clinical manifestations are facial dysmorphism, hepatosplenomegaly, upper airway obstruction, skeletal deformity and cardiomyopathy. If Hurler syndrome is left untreated, death ensues by adolescence. There are more attenuated variants termed Hurler-Scheie or Scheie syndrome, with those affected potentially not presenting until adulthood. Enzyme replacement therapy has been used for a number of years in the treatment of Hurler syndrome, although the current gold standard would be a haemopoietic stem cell transplant in those diagnosed by 2.5 years of age. This is an updated version of the original Cochrane review published in 2013. To evaluate the effectiveness and safety of treating mucopolysaccharidosis type I with laronidase enzyme replacement therapy as compared to placebo. We searched the Cochrane Cystic Fibrosis and Genetic Disorders Group's Inborn Errors of Metabolism Trials Register, MEDLINE via OVID and Embase.Date of most recent search: 05 October 2015. Randomised and quasi-randomised controlled studies of laronidase enzyme replacement therapy compared to placebo. Two authors independently screened the identified studies. The authors then appraised and extracted data. One study of 45 patients met the inclusion criteria. This double-blind, placebo-controlled, randomised, multinational study looked at laronidase at a dose of 0.58 mg/kg/week versus placebo in patients with mucopolysaccharidosis type I. All primary outcomes listed in this review were studied in this study. The laronidase group achieved statistically significant improvements in per cent predicted forced vital capacity compared to placebo, MD 5.60 (95% confidence intervals 1.24 to 9.96) and in the six-minute-walk test (mean improvement of 38.1 metres in the laronidase group; P = 0.039, when using a prospectively planned analysis of covariance). The levels of urinary glycoaminoglycans were also significantly reduced. In addition, there were improvements in hepatomegaly, sleep apnoea and hypopnoea. Laronidase antibodies were detected in nearly all patients in the treatment group with no apparent clinical effect and titres were reducing by the end of the study. Infusion-related adverse reactions occurred in both groups but all were mild and none necessitated medical intervention or infusion cessation. The current evidence demonstrates that laronidase is effective when compared to placebo in the treatment of mucopolysaccharidosis type I. The included study was comprehensive and of good quality, although there were few participants. The study included all of the key outcome measures we wished to look at. It demonstrated that laronidase is efficacious in relation to reducing biochemical parameters (reduced urine glycosaminoglycan excretion) and improved functional capacity as assessed by forced vital capacity and the six-minute-walk test. In addition glycosaminoglycan storage was reduced as ascertained by a reduction in liver volume. Laronidase appeared to be safe and, while antibodies were generated, these titres were reducing by the end of the study. More studies are required to determine long-term effectiveness and safety and to assess the impact upon quality of life. Enzyme replacement therapy with laronidase can be used pre- and peri-haemopoietic stem cell transplant, which is now the gold standard treatment in those patients diagnosed under 2.5 years of age.

X Demographics

X Demographics

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Mendeley readers

Mendeley readers

The data shown below were compiled from readership statistics for 114 Mendeley readers of this research output. Click here to see the associated Mendeley record.

Geographical breakdown

Country Count As %
South Africa 1 <1%
Unknown 113 99%

Demographic breakdown

Readers by professional status Count As %
Student > Master 21 18%
Researcher 14 12%
Student > Ph. D. Student 14 12%
Student > Bachelor 11 10%
Other 10 9%
Other 17 15%
Unknown 27 24%
Readers by discipline Count As %
Medicine and Dentistry 22 19%
Nursing and Health Professions 14 12%
Psychology 12 11%
Biochemistry, Genetics and Molecular Biology 11 10%
Pharmacology, Toxicology and Pharmaceutical Science 6 5%
Other 18 16%
Unknown 31 27%
Attention Score in Context

Attention Score in Context

This research output has an Altmetric Attention Score of 11. 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 21 July 2023.
All research outputs
#3,337,376
of 25,457,297 outputs
Outputs from Cochrane database of systematic reviews
#5,947
of 11,499 outputs
Outputs of similar age
#51,550
of 314,928 outputs
Outputs of similar age from Cochrane database of systematic reviews
#147
of 237 outputs
Altmetric has tracked 25,457,297 research outputs across all sources so far. Compared to these this one has done well and is in the 86th percentile: it's in the top 25% of all research outputs ever tracked by Altmetric.
So far Altmetric has tracked 11,499 research outputs from this source. They typically receive a lot more attention than average, with a mean Attention Score of 40.0. This one is in the 48th percentile – i.e., 48% 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 314,928 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 83% of its contemporaries.
We're also able to compare this research output to 237 others from the same source and published within six weeks on either side of this one. This one is in the 37th percentile – i.e., 37% of its contemporaries scored the same or lower than it.