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A randomized, controlled, double-blind, crossover trial of triheptanoin in alternating hemiplegia of childhood

Overview of attention for article published in Orphanet Journal of Rare Diseases, October 2017
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Title
A randomized, controlled, double-blind, crossover trial of triheptanoin in alternating hemiplegia of childhood
Published in
Orphanet Journal of Rare Diseases, October 2017
DOI 10.1186/s13023-017-0713-2
Pubmed ID
Authors

Elodie Hainque, Samantha Caillet, Sandrine Leroy, Constance Flamand-Roze, Isaac Adanyeguh, Fanny Charbonnier-Beaupel, Maryvonne Retail, Benjamin Le Toullec, Mariana Atencio, Sophie Rivaud-Péchoux, Vanessa Brochard, Florence Habarou, Chris Ottolenghi, Florence Cormier, Aurélie Méneret, Marta Ruiz, Mohamed Doulazmi, Anne Roubergue, Jean-Christophe Corvol, Marie Vidailhet, Fanny Mochel, Emmanuel Roze

Abstract

Based on the hypothesis of a brain energy deficit, we investigated the safety and efficacy of triheptanoin on paroxysmal episodes in patients with alternating hemiplegia of childhood due to ATP1A3 mutations. We conducted a randomized, double-blind, placebo-controlled crossover study of triheptanoin, at a target dose corresponding to 30% of daily calorie intake, in ten patients with alternating hemiplegia of childhood due to ATP1A3 mutations. Each treatment period consisted of a 12-week fixed-dose phase, separated by a 4-week washout period. The primary outcome was the total number of paroxysmal events. Secondary outcomes included the number of paroxysmal motor-epileptic events; a composite score taking into account the number, severity and duration of paroxysmal events; interictal neurological manifestations; the clinical global impression-improvement scale (CGI-I); and safety parameters. The paired non-parametric Wilcoxon test was used to analyze treatment effects. In an intention-to-treat analysis, triheptanoin failed to reduce the total number of paroxysmal events (p = 0.646), including motor-epileptic events (p = 0.585), or the composite score (p = 0.059). CGI-I score did not differ between triheptanoin and placebo periods. Triheptanoin was well tolerated. Triheptanoin does not prevent paroxysmal events in Alternating hemiplegia of childhood. We show the feasibility of a randomized placebo-controlled trial in this setting. The study has been registered with clinicaltrials.gov ( NCT002408354 ) the 03/24/2015.

Mendeley readers

The data shown below were compiled from readership statistics for 42 Mendeley readers of this research output. Click here to see the associated Mendeley record.

Geographical breakdown

Country Count As %
Unknown 42 100%

Demographic breakdown

Readers by professional status Count As %
Student > Master 7 17%
Student > Bachelor 6 14%
Other 3 7%
Researcher 3 7%
Student > Ph. D. Student 3 7%
Other 5 12%
Unknown 15 36%
Readers by discipline Count As %
Medicine and Dentistry 9 21%
Nursing and Health Professions 5 12%
Biochemistry, Genetics and Molecular Biology 2 5%
Neuroscience 2 5%
Psychology 2 5%
Other 3 7%
Unknown 19 45%

Attention Score in Context

This research output has an Altmetric Attention Score of 1. 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 04 October 2017.
All research outputs
#9,479,999
of 11,868,523 outputs
Outputs from Orphanet Journal of Rare Diseases
#1,090
of 1,295 outputs
Outputs of similar age
#199,322
of 272,685 outputs
Outputs of similar age from Orphanet Journal of Rare Diseases
#16
of 19 outputs
Altmetric has tracked 11,868,523 research outputs across all sources so far. This one is in the 11th percentile – i.e., 11% of other outputs scored the same or lower than it.
So far Altmetric has tracked 1,295 research outputs from this source. They typically receive a little more attention than average, with a mean Attention Score of 6.8. This one is in the 8th percentile – i.e., 8% 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 272,685 tracked outputs that were published within six weeks on either side of this one in any source. This one is in the 15th percentile – i.e., 15% of its contemporaries scored the same or lower than it.
We're also able to compare this research output to 19 others from the same source and published within six weeks on either side of this one. This one is in the 5th percentile – i.e., 5% of its contemporaries scored the same or lower than it.