| Title |
Efficacy and safety of Velmanase alfa in the treatment of patients with alpha‐mannosidosis: results from the core and extension phase analysis of a phase III multicentre, double‐blind, randomised, placebo‐controlled trial
|
|---|---|
| Published in |
Journal of Inherited Metabolic Disease, May 2018
|
| DOI | 10.1007/s10545-018-0185-0 |
| Pubmed ID | |
| Authors |
Line Borgwardt, Nathalie Guffon, Yasmina Amraoui, Christine I. Dali, Linda De Meirleir, Mercedes Gil‐Campos, Bénédicte Heron, Silvia Geraci, Diego Ardigò, Federica Cattaneo, Jens Fogh, J. M. Hannerieke Van den Hout, Michael Beck, Simon A. Jones, Anna Tylki‐Szymanska, Ulla Haugsted, Allan M. Lund |
| Abstract |
This phase III, double-blind, randomised, placebo-controlled trial (and extension phase) was designed to assess the efficacy and safety of velmanase alfa (VA) in alpha-mannosidosis (AM) patients. Twenty-five patients were randomised to weekly 1 mg/kg VA or placebo for 52 weeks. At study conclusion, placebo patients switched to VA; 23 patients continued receiving VA in compassionate-use/follow-on studies and were evaluated in the extension phase [last observation (LO)]. Co-primary endpoints were changes in serum oligosaccharide (S-oligo) and in the 3-min stair-climb test (3MSCT). Mean relative change in S-oligo in the VA arm was -77.6% [95% confidence interval (CI) -81.6 to -72.8] at week 52 and -62.9% (95% CI -85.8 to -40.0) at LO; mean relative change in the placebo arm was -24.1% (95% CI -40.3 to -3.6) at week 52 and -55.7% (95% CI -76.4 to -34.9) at LO after switch to active treatment. Mean relative change in 3MSCT at week 52 was -1.1% (95% CI -9.0 to 7.6) and - % (95% CI -13.4 to 6.5) for VA and placebo, respectively. At LO, the mean relative change was 3.9% (95% CI -5.5 to 13.2) in the VA arm and 9.0% (95% CI -10.3 to 28.3) in placebo patients after switch to active treatment. Similar improvement pattern was observed in secondary parameters. A post hoc analysis investigated whether some factors at baseline could account for treatment outcome; none of those factors were predictive of the response to VA, besides age. These findings support the utility of VA for the treatment of AM, with more evident benefit over time and when treatment is started in the paediatric age. |
X Demographics
The data shown below were collected from the profiles of 5 X users who shared this research output. Click here to find out more about how the information was compiled.
Geographical breakdown
| Country | Count | As % |
|---|---|---|
| Spain | 1 | 20% |
| United Kingdom | 1 | 20% |
| Unknown | 3 | 60% |
Demographic breakdown
| Type | Count | As % |
|---|---|---|
| Practitioners (doctors, other healthcare professionals) | 3 | 60% |
| Science communicators (journalists, bloggers, editors) | 1 | 20% |
| Members of the public | 1 | 20% |
Mendeley readers
The data shown below were compiled from readership statistics for 51 Mendeley readers of this research output. Click here to see the associated Mendeley record.
Geographical breakdown
| Country | Count | As % |
|---|---|---|
| Unknown | 51 | 100% |
Demographic breakdown
| Readers by professional status | Count | As % |
|---|---|---|
| Student > Master | 6 | 12% |
| Researcher | 4 | 8% |
| Student > Ph. D. Student | 4 | 8% |
| Student > Doctoral Student | 3 | 6% |
| Other | 3 | 6% |
| Other | 8 | 16% |
| Unknown | 23 | 45% |
| Readers by discipline | Count | As % |
|---|---|---|
| Medicine and Dentistry | 13 | 25% |
| Biochemistry, Genetics and Molecular Biology | 4 | 8% |
| Nursing and Health Professions | 2 | 4% |
| Psychology | 2 | 4% |
| Pharmacology, Toxicology and Pharmaceutical Science | 1 | 2% |
| Other | 5 | 10% |
| Unknown | 24 | 47% |
Attention Score in Context
This research output has an Altmetric Attention Score of 22. 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 19 December 2023.
All research outputs
#1,653,012
of 25,022,483 outputs
Outputs from Journal of Inherited Metabolic Disease
#35
of 1,988 outputs
Outputs of similar age
#34,816
of 337,320 outputs
Outputs of similar age from Journal of Inherited Metabolic Disease
#2
of 34 outputs
Altmetric has tracked 25,022,483 research outputs across all sources so far. Compared to these this one has done particularly well and is in the 93rd percentile: it's in the top 10% of all research outputs ever tracked by Altmetric.
So far Altmetric has tracked 1,988 research outputs from this source. They receive a mean Attention Score of 4.8. This one has done particularly well, scoring higher than 98% of its peers.
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