Evidence map›Paper›PMID 26547562›Full record

ArticleMolecular genetics and metabolism2015

Anaplerotic treatment of long-chain fat oxidation disorders with triheptanoin: Review of 15 years Experience.

Charles R Roe, Henri Brunengraber

Registry-linked trialOpen access · greenAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Molecular genetics and metabolism, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT04513002 (A Phase 2A/2B Placebo-controlled Randomised Clinical Trial to Test the Ability of Triheptanoin to Protect Primary Airway Epithelial Cells Obtained From Participants With Ataxia-telangiectasia Against Death Induced by Glucose Deprivation), which is not on this map. Cited by 47 papers, 3 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
47citing papers in PubMed, 3 pooled it
4.0field-weighted citation impact, top 6% 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.

NCT04513002 phase2completednot on this mapstarted 2022, after this paper: background citation

A Phase 2A/2B Placebo-controlled Randomised Clinical Trial to Test the Ability of Triheptanoin to Protect Primary Airway Epithelial Cells Obtained From Participants With Ataxia-telangiectasia Against Death Induced by Glucose Deprivation

TypeinterventionalSponsorThe University of QueenslandRan2022 to 2023Enrolled30ConditionsAtaxia TelangiectasiaArmsTriheptanoin
3 · Its place in the literature

Who cites it

47 citing papers in PubMed, 3 syntheses or guidelines pooled it, 90 citations in OpenAlex.

  1. Guideline
  2. Pooled it
  3. Pooled it
  4. Trial
  5. Trial
  6. Trial
  7. No effect of triheptanoin on exercise performance in McArdle disease.Annals of clinical and translational neurology · 2019
    Trial
  8. Trial
  9. Trial
  10. Review
  11. Article
  12. Article
  13. Article
  14. Article
  15. Review
  16. Article
  17. Article
  18. Review
  19. HomozygousMolecular genetics and metabolism reports · 2024
    Article
  20. Article
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 2 institutions in 1 country.

Charles R RoeDepartment of Neurology and Neurotherapeutics, UT Southwestern Medical Center, Dallas, TX 75390, USA; Investigations were performed at the Institute of Metabolic Disease, Baylor University Medical Center, Dallas, TX, USA. Electronic address: chasroe@earthlink.net.
Henri BrunengraberDepartments of Nutrition and Biochemistry, Case Western Reserve University School of Medicine, Cleveland, OH 44106, USA.
Baylor University Medical Center · USCase Western Reserve University · US

Funding

Nutritional Treatment of Fat Oxidation Defect in MiceR01DK069752 · NIDDK · CASE WESTERN RESERVE UNIVERSITY · PI BRUNENGRABER, HENRI · 2005 to 2009
$2.0M
KETONE BODY METABOLISM IN HEALTH AND DISEASER01DK035543 · NIDDK · NOTRE DAME HOSPITAL · PI BRUNENGRABER, HENRI · 1986 to 2004
$1.8M
NIDDK NIH HHS 1R01-DK035543NIDDK NIH HHS 1R01-DK069752NIDDK NIH HHS R01 DK035543NIDDK NIH HHS R01 DK069752
6 · The paper itself

Abstract

backgroundThe treatment of long-chain mitochondrial β-oxidation disorders (LC-FOD) with a low fat-high carbohydrate diet, a diet rich in medium-even-chain triglycerides (MCT), or a combination of both has been associated with high morbidity and mortality for decades. The pathological tableau appears to be caused by energy deficiency resulting from reduced availability of citric acid cycle (CAC) intermediates required for optimal oxidation of acetyl-CoA. This hypothesis was investigated by diet therapy with carnitine and anaplerotic triheptanoin (TH).

methodsFifty-two documented LC-FOD patients were studied in this investigation (age range: birth to 51 years). Safety monitoring included serial quantitative measurements of routine blood chemistries, blood levels of carnitine and acylcarnitines, and urinary organic acids.

resultsThe average frequency of serious clinical complications were reduced from ~60% with conventional diet therapy to 10% with TH and carnitine treatment and mortality decreased from ~65% with conventional diet therapy to 3.8%. Carnitine supplementation was uncomplicated.

conclusionThe energy deficiency in LC-FOD patients was corrected safely and more effectively with the triheptanoin diet and carnitine supplement than with conventional diet therapy. Safe intervention in neonates and infants will permit earlier intervention following pre-natal diagnosis or diagnosis by expanded newborn screening.

Indexed as

Administration, OralAdolescentAdultCarnitineChildChild, PreschoolCitric AcidFatty AcidsFemaleHumansInfantInfant, NewbornLactic AcidLipid Metabolism, Inborn ErrorsMalatesMaleacylcarnitineCarnitineCitric AcidFatty AcidsLactic AcidMalatesmalic acidSuccinic AcidTriglyceridestriheptanoinAnaplerosisFat oxidation disordersKetogenic diet therapyTriheptanoin

Identifiers

PMID26547562
PMCPMC4712637
OpenAlexW2123942174

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Registered trials

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.