Triheptanoin improves left ventricular function in patients with long chain fatty acid oxidation disorders (LCFAODs) who have a left ventricular ejection fraction (LVEF) of 55% or less, according to a study presented at the 2022 lecture series of the International Network for Fatty Acid Oxidation Research and Management (INFORM) by Kathryn Ray, PhD, a biostatistician at Ultragenyx Pharmaceutical.

It is known that LCFAOD can cause cardiomyopathic events that are unpredictable and that may be life-threatening. Previous research has shown that triheptanoin can increase LVEF. The treatment is approved by the US Food and Drug Administration to treat children and adults with the disease and is sold under the brand name Dojolvi®.

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The aim of the present study was to compare the effect of triheptanoin treatment in patients with different LVEFs. Data from 4 clinical trials were included in the study and patients were divided into 3 groups. The first group included patients who had an LVEF of 55% or less, those in the second group included patients with an LVEF in the normal range, ie between 55% and 70%, and those in the third group included patients with an LVEF of more than 70%.

The results showed that triheptanoin treatment led to improvements in LVEF in patients with LCFAOD who had a baseline LVEF of 55% or less.

In patients with a normal range of LVEF at baseline, there still was an increase in LVEF following triheptanoin treatment while triheptanoin treatment led to a decrease in LVEF down to the normal range in patients who had a baseline level of more than 70%.

“Our overall conclusion from this is that we don’t believe it’s just an increase in LVEF from triheptanoin in the normal range,” Dr. Ray said. She added that they also think that patients who are not in the normal range and have low LVEF can benefit from triheptanoin treatment.

Reference

Ray K, Vockley J, Ramirez AN, Lu X, Gillingham MB. Improvement in left ventricular ejection fraction in patients treated with triheptanoin for long-chain fatty acid oxidation disorders (LC-FAOD). Lecture presented at: International Network for Fatty Acid Oxidation Research and Management (INFORM) 2022 lecture series: May 23, 2022; Virtual.