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Efficacy and safety of patisiran for familial amyloidotic polyneuropathy: a phase II multi-dose study

Overview of attention for article published in Orphanet Journal of Rare Diseases, September 2015
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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 (90th percentile)
  • High Attention Score compared to outputs of the same age and source (90th percentile)

Mentioned by

news
1 news outlet
twitter
6 X users
patent
1 patent
wikipedia
1 Wikipedia page

Citations

dimensions_citation
253 Dimensions

Readers on

mendeley
281 Mendeley
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Title
Efficacy and safety of patisiran for familial amyloidotic polyneuropathy: a phase II multi-dose study
Published in
Orphanet Journal of Rare Diseases, September 2015
DOI 10.1186/s13023-015-0326-6
Pubmed ID
Authors

Ole B Suhr, Teresa Coelho, Juan Buades, Jean Pouget, Isabel Conceicao, John Berk, Hartmut Schmidt, Márcia Waddington-Cruz, Josep M. Campistol, Brian R. Bettencourt, Akshay Vaishnaw, Jared Gollob, David Adams

Abstract

Transthyretin-mediated amyloidosis is an inherited, progressively debilitating disease caused by mutations in the transthyretin gene. This study evaluated the safety, tolerability, pharmacokinetics, and pharmacodynamics of multiple doses of patisiran (ALN-TTR02), a small interfering RNA encapsulated within lipid nanoparticles, in patients with transthyretin-mediated familial amyloid polyneuropathy (FAP). In this phase II study, patients with FAP were administered 2 intravenous infusions of patisiran at one of the following doses: 0.01 (n = 4), 0.05 (n = 3), 0.15 (n = 3), or 0.3 (n = 7) mg/kg every 4 weeks (Q4W), or 0.3 mg/kg (n = 12) every 3 weeks (Q3W). Of 29 patients in the intent-to-treat population, 26 completed the study. Administration of patisiran led to rapid, dose-dependent, and durable knockdown of transthyretin, with the maximum effect seen with patisiran 0.3 mg/kg; levels of mutant and wild-type transthyretin were reduced to a similar extent in Val30Met patients. A mean level of knockdown exceeding 85 % after the second dose, with maximum knockdown of 96 %, was observed for the Q3W dose. The most common treatment-related adverse event (AE) was mild-to-moderate infusion-related reactions in 10.3 % of patients. Four serious AEs (SAEs) were reported in 1 patient administered 0.3 mg/kg Q3W (urinary tract infection, sepsis, nausea, vomiting), and 1 patient administered 0.3 mg/kg Q4W had 1 SAE (extravasation-related cellulitis). Patisiran was generally well tolerated and resulted in significant dose-dependent knockdown of transthyretin protein in patients with FAP. Patisiran 0.3 mg/kg Q3W is currently in phase III development. NCT01617967 .

X Demographics

X Demographics

The data shown below were collected from the profiles of 6 X users who shared this research output. Click here to find out more about how the information was compiled.
Mendeley readers

Mendeley readers

The data shown below were compiled from readership statistics for 281 Mendeley readers of this research output. Click here to see the associated Mendeley record.

Geographical breakdown

Country Count As %
Brazil 1 <1%
Unknown 280 100%

Demographic breakdown

Readers by professional status Count As %
Researcher 39 14%
Student > Ph. D. Student 38 14%
Student > Bachelor 35 12%
Student > Master 34 12%
Other 26 9%
Other 45 16%
Unknown 64 23%
Readers by discipline Count As %
Medicine and Dentistry 44 16%
Biochemistry, Genetics and Molecular Biology 38 14%
Pharmacology, Toxicology and Pharmaceutical Science 32 11%
Agricultural and Biological Sciences 25 9%
Chemistry 18 6%
Other 46 16%
Unknown 78 28%
Attention Score in Context

Attention Score in Context

This research output has an Altmetric Attention Score of 19. 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 07 September 2021.
All research outputs
#1,858,115
of 24,649,404 outputs
Outputs from Orphanet Journal of Rare Diseases
#200
of 2,950 outputs
Outputs of similar age
#24,806
of 272,356 outputs
Outputs of similar age from Orphanet Journal of Rare Diseases
#5
of 40 outputs
Altmetric has tracked 24,649,404 research outputs across all sources so far. Compared to these this one has done particularly well and is in the 92nd percentile: it's in the top 10% of all research outputs ever tracked by Altmetric.
So far Altmetric has tracked 2,950 research outputs from this source. They typically receive more attention than average, with a mean Attention Score of 8.1. This one has done particularly well, scoring higher than 93% 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 272,356 tracked outputs that were published within six weeks on either side of this one in any source. This one has done particularly well, scoring higher than 90% of its contemporaries.
We're also able to compare this research output to 40 others from the same source and published within six weeks on either side of this one. This one has done particularly well, scoring higher than 90% of its contemporaries.