A pilot study of add-on oral triheptanoin treatment for children with medically refractory epilepsy

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Calvert, Sophie
Barwick, Katie
Par, Melody
Tan, Kah Ni
Borges, Karin
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2018
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Abstract

Aim: Despite antiepileptic medication and dietary treatment options available about 45% of children with epilepsy still suffer from uncontrolled seizures. Triheptanoin is an anaplerotic treatment designed to improve energy generation via the Krebs cycle.

Method: For the first time, we evaluated the feasibility, tolerability and efficacy of add-on triheptanoin in 12 patients with medically refractory epilepsy (seven males, five females; min–max: 3–18yr, median 13.5 yr).

Results: Eight out of a total of 12 children (67%), who tested the treatment, finished the trial and tolerated between 30 and 100 ml of triheptanoin per day for >12 weeks (median 55 ml, 20.5% caloric intake). The most common adverse effects were diarrhea and other gastro-intestinal effects in seven kids. One child experienced leaking and another child had an infected percutaneous endoscopic gastrostomy button. Five children (62.5%), who all had been on the ketogenic diet previously, showed sustained >50% reductions in seizure frequency, including one patient who became seizure free for 30 weeks. Four patients extended their treatment to a total of 201–909 days, until seizure frequency or severity increased.

Interpretation: In this small trial, triheptanoin was safe and tolerable in children with epilepsy. As some children showed reductions in seizure numbers and/or severity, larger randomized controlled studies are now needed for further evaluation of safety and efficacy.

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European Journal of Paediatric Neurology

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22

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6

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© 2018 European Paediatric Neurology Society. Published by Elsevier Ltd. Licensed under the Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International Licence (http://creativecommons.org/licenses/by-nc-nd/4.0/) which permits unrestricted, non-commercial use, distribution and reproduction in any medium, providing that the work is properly cited.

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Clinical sciences

Neurosciences

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Calvert, S; Barwick, K; Par, M; Ni Tan, K; Borges, K, A pilot study of add-on oral triheptanoin treatment for children with medically refractory epilepsy, European Journal of Paediatric Neurology, 2018, 22 (6), pp. 1074-1080

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