Evidence map›Paper›PMID 27459034›Full record

Trial reportEpilepsy & behavior : E&B2016

Efficacy and tolerability of perampanel in ten patients with Lafora disease.

Danielle Goldsmith, Berge A Minassian

Abstract readClinical TrialMulticenter Study
PubMed Full text at PMC Publisher
In one paragraph

Trial report in Epilepsy & behavior : E&B, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 27 papers, 3 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
27citing papers in PubMed, 3 pooled it
10.8field-weighted citation impact, top 1% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

27 citing papers in PubMed, 3 syntheses or guidelines pooled it, 80 citations in OpenAlex.

  1. Pooled it
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  15. Perampanel in lissencephaly-associated epilepsy.Epilepsy & behavior case reports · 2019
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4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

2 authors at 1 institution in 1 country.

Danielle GoldsmithChelsea's Hope: Lafora Children Research Fund, Danville, CA, USA; Program in Genetics and Genome Biology, The Hospital for Sick Children, Toronto, ON, Canada.
Berge A MinassianProgram in Genetics and Genome Biology, The Hospital for Sick Children, Toronto, ON, Canada; The Institute of Medical Science and The Department of Paediatrics (Neurology), University of Toronto, ON, Canada. Electronic address: berge.minassian@sickkids.ca.
Hospital for Sick Children · CA

Funding

Suppressing glycogen storage with small molecule inhibitors as a therapeutic approach to Lafora DiseaseP01NS097197 · NINDS · UNIVERSITY OF KENTUCKY · PI GAO, TIANYAN · 2016 to 2021
$9.0M
NINDS NIH HHS P01 NS097197
6 · The paper itself

Abstract

Lafora disease (LD) is a fatal intractable adolescence-onset progressive myoclonus epilepsy. Recently, two single-case studies reported drastic reductions in seizures and myoclonus with the AMPA antagonist perampanel and improved activities of daily living. We proceeded to study the effect of perampanel on 10 patients with genetically confirmed LD with disease duration ranging from 2 to 27years. Open-label perampanel was added to ongoing medications to a mean dose of 6.7mg/day. Seizures, myoclonus, functional disability, and cognition scores were measured for the third and ninth months following initiation and compared with those of the month prior to the start of therapy. Three patients withdrew because of inefficacy or side effects. Four had significant reduction in seizures of greater than 74% from baseline. Seven had major improvement in myoclonus with group-adjusted sum score of myoclonus intensity reduced from 7.01 at baseline to 5.67 and 5.18 at 3 and 9months, respectively. There was no significant improvement in disability and cognition. While not universal, perampanel adjunctive therapy appears to confer particular benefit not commonly seen with other antiepileptic drugs on seizures and myoclonus in LD. Improvement in the extremely disabling myoclonus of LD is a major benefit in this devastating disease.

Indexed as

Activities of Daily LivingAdolescentAdultAnticonvulsantsFemaleHumansLafora DiseaseMaleMyoclonusNitrilesPyridonesYoung AdultAnticonvulsantsNitrilesperampanelPyridonesAntiepilepticLafora diseasePerampanelProgressive myoclonus epilepsy

Identifiers

PMID27459034
PMCPMC5691360
OpenAlexW2479317925

What OpenQuestion holds

Textmetadata
LicenceTDM
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.