| Title |
Comprehensive long‐term efficacy and safety of recombinant human alpha‐mannosidase (velmanase alfa) treatment in patients with alpha‐mannosidosis
|
|---|---|
| Published in |
Journal of Inherited Metabolic Disease, May 2018
|
| DOI | 10.1007/s10545-018-0175-2 |
| Pubmed ID | |
| Authors |
Allan M. Lund, Line Borgwardt, Federica Cattaneo, Diego Ardigò, Silvia Geraci, Mercedes Gil‐Campos, Linda De Meirleir, Cécile Laroche, Philippe Dolhem, Duncan Cole, Anna Tylki‐Szymanska, Monica Lopez‐Rodriguez, Encarna Guillén‐Navarro, Christine I. Dali, Bénédicte Héron, Jens Fogh, Nicole Muschol, Dawn Phillips, J. M. Hannerieke Van den Hout, Simon A. Jones, Yasmina Amraoui, Paul Harmatz, Nathalie Guffon |
| Abstract |
Long-term outcome data provide important insights into the clinical utility of enzyme replacement therapies. Such data are presented for velmanase alfa in the treatment of alpha-mannosidosis (AM). Patient data (n = 33; 14 adults, 19 paediatric) from the clinical development programme for velmanase alfa were integrated in this prospectively-designed analysis of long-term efficacy and safety. Patients who participated in the phase I/II or phase III trials and were continuing to receive treatment after completion of the trials were invited to participate in a comprehensive evaluation visit to assess long-term outcomes. Primary endpoints were changes in serum oligosaccharide and the 3-minute stair climb test (3MSCT). Mean (SD) treatment exposure was 29.3 (15.2) months. Serum oligosaccharide levels were significantly reduced in the overall population at 12 months (mean change: -72.7%, P < 0.001) and remained statistically significant at last observation (-62.8%, P < 0.001). A mean improvement of +9.3% in 3MSCT was observed at 12 months (P = 0.013), which also remained statistically significant at last observation (+13.8%, P = 0.004), with a more pronounced improvement detected in the paediatric subgroup. No treatment-emergent adverse events were reported leading to permanent treatment discontinuation. Patients treated with velmanase alfa experienced improvements in biochemical and functional measures that were maintained for up to 4 years. Long term follow-up is important and further supports the use of velmanase alfa as an effective and well-tolerated treatment for AM. Based on the currently available data set, no baseline characteristic can be predictive of treatment outcome. Early treatment during paediatric age showed better outcome in functional endpoints. |
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 States | 1 | 20% |
| Unknown | 3 | 60% |
Demographic breakdown
| Type | Count | As % |
|---|---|---|
| Members of the public | 3 | 60% |
| Science communicators (journalists, bloggers, editors) | 1 | 20% |
| Practitioners (doctors, other healthcare professionals) | 1 | 20% |
Mendeley readers
The data shown below were compiled from readership statistics for 67 Mendeley readers of this research output. Click here to see the associated Mendeley record.
Geographical breakdown
| Country | Count | As % |
|---|---|---|
| Unknown | 67 | 100% |
Demographic breakdown
| Readers by professional status | Count | As % |
|---|---|---|
| Student > Master | 9 | 13% |
| Researcher | 6 | 9% |
| Student > Postgraduate | 6 | 9% |
| Other | 4 | 6% |
| Student > Doctoral Student | 3 | 4% |
| Other | 7 | 10% |
| Unknown | 32 | 48% |
| Readers by discipline | Count | As % |
|---|---|---|
| Medicine and Dentistry | 18 | 27% |
| Nursing and Health Professions | 4 | 6% |
| Pharmacology, Toxicology and Pharmaceutical Science | 3 | 4% |
| Biochemistry, Genetics and Molecular Biology | 2 | 3% |
| Social Sciences | 2 | 3% |
| Other | 6 | 9% |
| Unknown | 32 | 48% |
Attention Score in Context
This research output has an Altmetric Attention Score of 2. 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 09 April 2020.
All research outputs
#15,102,803
of 24,417,958 outputs
Outputs from Journal of Inherited Metabolic Disease
#1,408
of 1,953 outputs
Outputs of similar age
#183,494
of 330,575 outputs
Outputs of similar age from Journal of Inherited Metabolic Disease
#22
of 43 outputs
Altmetric has tracked 24,417,958 research outputs across all sources so far. This one is in the 37th percentile – i.e., 37% of other outputs scored the same or lower than it.
So far Altmetric has tracked 1,953 research outputs from this source. They receive a mean Attention Score of 4.8. This one is in the 25th percentile – i.e., 25% 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 330,575 tracked outputs that were published within six weeks on either side of this one in any source. This one is in the 42nd percentile – i.e., 42% of its contemporaries scored the same or lower than it.
We're also able to compare this research output to 43 others from the same source and published within six weeks on either side of this one. This one is in the 44th percentile – i.e., 44% of its contemporaries scored the same or lower than it.