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Review

Triheptanoin

No authors listed
In: LiverTox: Clinical and Research Information on Drug-Induced Liver Injury [Internet]. Bethesda (MD): National Institute of Diabetes and Digestive and Kidney Diseases; 2012.
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Review

Triheptanoin

No authors listed.
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Excerpt

Triheptanoin is synthetic, medium-chain triglyceride that was developed for nutritional support of patients with long-chain fatty acid oxidation disorders. Triheptanoin is given orally by feeding tube titrated to provide approximately 30% of calories. Triheptanoin therapy has not been associated with elevations in serum aminotransferase or bilirubin levels or to instances of clinically apparent liver injury.

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References

    1. FDA. Integrated Review. 2020.
    1. https://www.accessdata.fda.gov/drugsatfda_docs/nda/2020/213687Orig1s000I... [ (FDA website with integrated review of data on triheptanoin safety and efficacy submitted in support of the application for approval as therapy of long-chain fatty acid oxidation disorders (LC-FAOD), mentions laboratory abnormalities found during therapy include elevations in CPK [71%], ALT [72%], and AST [67%] which are greater than 5 times the ULN in 47%, 24%, and 15%, the elevations however, were attributed to the underlying disease not to triheptanoin). ]
    1. Vockley J, Marsden D, McCracken E, DeWard S, Barone A, Hsu K, Kakkis E. Long-term major clinical outcomes in patients with long chain fatty acid oxidation disorders before and after transition to triheptanoin treatment--A retrospective chart review. Mol Genet Metab. 2015;116(1-2):53-60.(In a retrospective analysis of 29 patients with LC-FAOD treated with triheptanoin [providing 28% of daily caloric intake] for 78 weeks, the annualized rate of major clinical events [rhabdomyolysis, hypoglycemia, myocardiopathy] decreased by 48% compared to previous therapy with standard MCT [providing 17% of daily caloric intake]; no mention of adverse events). - PMC - PubMed
    1. Gillingham MB, Heitner SB, Martin J, Rose S, Goldstein A, El-Gharbawy AH, Deward S, et al. Triheptanoin versus trioctanoin for long-chain fatty acid oxidation disorders: a double blinded, randomized controlled trial. J Inherit Metab Dis. 2017;40:831-843.(Among 32 patients with LC-FAOD treated with either triheptanoin or trioctanoin for 4 months, slight improvements in cardiac function was found with the 7 carbon but not the 8 carbon triglyceride, while adverse events were similar with the two products; no mention of ALT elevations or hepatotoxicity). - PMC - PubMed
    1. Hainque E, Caillet S, Leroy S, Flamand-Roze C, Adanyeguh I, Charbonnier-Beaupel F, Retail M, et al. A randomized, controlled, double-blind, crossover trial of triheptanoin in alternating hemiplegia of childhood. Orphanet J Rare Dis. 2017;12:160.(Among 10 patients with alternating hemiplegia of childhood treated with triheptanoin or placebo in a cross-over design, there were no differences in paroxysmal events between the treatment and placebo periods and adverse event rates were similar [50% vs 70%] and triheptanoin was considered “well tolerated”; no mention of ALT elevations or hepatotoxicity). - PMC - PubMed

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