Evidence map›Paper›PMID 32524048›Full record

ArticleEpilepsia open2020

Open-label long-term treatment of add-on triheptanoin in adults with drug-resistant epilepsy.

Karin Borges, Neha Kaul, Jack Germaine, Catalina Carrasco-Pozo, Patrick Kwan, Terence J O'Brien

Open access · goldAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Epilepsia open, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 8 papers.

0numbers the graph read from it
0cells of the map it votes in
8citing papers in PubMed
1.5field-weighted citation impact, top 18% 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

8 citing papers in PubMed, 11 citations in OpenAlex.

  1. Review
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  6. Non-pharmacological Interventions for Intractable Epilepsy.Saudi pharmaceutical journal : SPJ : the official publication of the Saudi Pharmaceutical Society · 2020
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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

6 authors at 4 institutions in 1 country.

Karin BorgesSchool of Biomedical Sciences Faculty of Medicine The University of Queensland St. Lucia QLD Australia.ORCID https://orcid.org/0000-0001-7448-0770
Neha KaulDepartment of Allied Health (Clinical Nutrition) Royal Melbourne Hospital University of Melbourne Parkville Vic Australia.
Jack GermaineDepartments of Neuroscience and Neurology The Central Clinical School Monash University and The Alfred Hospital Melbourne Vic Australia.
Catalina Carrasco-PozoDiscovery Biology Griffith Institute for Drug Discovery Griffith University Nathan QLD Australia.
Patrick KwanDepartments of Medicine and Neurology Royal Melbourne Hospital University of Melbourne Parkville Vic Australia.
Terence J O'BrienDepartments of Medicine and Neurology Royal Melbourne Hospital University of Melbourne Parkville Vic Australia.
The Royal Melbourne Hospital · AUGriffith University · AUThe Alfred Hospital · AUThe University of Queensland · AU

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

objectiveTo investigate feasibility, safety, and tolerability of long-term (48 weeks) add-on treatment with triheptanoin (UX007), the triglyceride of heptanoate, in adults with drug-resistant epilepsy.

methodsThis extension study was offered to adult participants with drug-resistant epilepsy who completed a 12-week randomized controlled trial of add-on medium-chain triglycerides (MCT) vs triheptanoin. Participants were asked to titrate triheptanoin to their maximum tolerated dose over 3 weeks, followed by 48-week maintenance before tapering or treatment extension. The primary aims were to assess retention and safety of the triheptanoin treatment, and secondary aims to assess the tolerated doses and changes in seizure frequency.

resultsEleven adults were enrolled and ten people were analyzed (because one patient was diagnosed as having nonepileptic seizures while on the study). Two adults finished the study and extended their treatment. Eight participants withdrew from the study, due to lack of efficacy (n = 3), unknown reasons (n = 2), belief of weight gain (n = 1), wanting to try a different treatment (n = 1), and a colonoscopy (n = 1). Diarrhea in two people and bloating in one person were deemed possibly related to treatment, but other adverse events were not. The duration of maintenance treatment dose was 27-513 days (median 247 days, range 27-513 days), and 0.49 -1.1 mL/kg triheptanoin was taken per day (0.77 ± 0.19 mL/kg, mean ± standard deviation, 40-100 mL/d). Two participants experienced >90% and three people >50% reduction in seizure frequency, and all had focal seizures. The median seizure reduction was 48% (average 38%). SIGNIFICANCE: Our results indicate antiseizure effects of triheptanoin on focal seizures in 5 out of 10 adults. However, only two people finished and extended the 48-week add-on treatment phase, despite lack of safety or tolerability issues.More studies focused on improved treatment formulations, the potential of lower dosages, and efficacy are needed. Trial registration number: ACTRN12615000406505.

Indexed as

anaplerosisfocal seizuremedium‐chain fatty acidtca cycle

Identifiers

PMID32524048
PMCPMC7278596
OpenAlexW3013440196

What OpenQuestion holds

Textmetadata
LicenceCC BY
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.