{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,26]],"date-time":"2026-09-26T11:32:22Z","timestamp":1790422342160,"version":"4.1.0"},"reference-count":11,"publisher":"Wiley","issue":"2","license":[{"start":{"date-parts":[[2020,11,11]],"date-time":"2020-11-11T00:00:00Z","timestamp":1605052800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":["aspenjournals.onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["J Parenter Enteral Nutr"],"published-print":{"date-parts":[[2021,2]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Background<\/jats:title><jats:p>Patients with severe long\u2010chain fatty acid oxidation disorders (LC\u2010FAODs) experience serious morbidity and mortality despite traditional dietary management including medium\u2010chain triglyceride (MCT)\u2013supplemented, low\u2010fat diets. Triheptanoin is a triglyceride oil that is broken down to acetyl\u2013coenzyme A (CoA) and propionyl\u2010CoA, which replenishes deficient tricarboxylic acid cycle intermediates. We report the complex medical and nutrition management of triheptanoin therapy initiated emergently for 3 patients with LC\u2010FAOD.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>Triheptanoin (Ultragenyx Pharmaceutical, Inc, Novato, CA, USA) was administered to 3 patients with LC\u2010FAOD on a compassionate\u2010use basis. Triheptanoin was mixed with non\u2013MCT\u2010containing low\u2010fat formula. Patients were closely followed with regular cardiac and laboratory monitoring.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Cardiac ejection fraction normalized after triheptanoin initiation. Patients experienced fewer hospitalizations related to metabolic crises while on triheptanoin. Patient 1 has tolerated oral administration without difficulty since birth. Patients 2 and 3 experienced increased diarrhea. Recurrent breakdown of the silicone gastrostomy tube occurred in patient 3, whereas the polyurethane nasogastric tube for patient 2 remained intact. Patient 3 experiences recurrent episodes of elevated creatine kinase levels and muscle weakness associated with illness. Patient 3 had chronically elevated C10\u2010acylcarnitines while on MCT supplementation, which normalized after initiation of triheptanoin and discontinuation of MCT oil.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions<\/jats:title><jats:p>Triheptanoin can ameliorate acute cardiomyopathy and increase survival in patients with severe LC\u2010FAOD. Substituting triheptanoin for traditional MCT\u2010based treatment improves clinical outcomes. MCT oil might be less effective in carnitine\u2010acylcarnitine translocase deficiency patients compared with other FAODs and needs further investigation.<\/jats:p><\/jats:sec>","DOI":"10.1002\/jpen.2034","type":"journal-article","created":{"date-parts":[[2020,10,21]],"date-time":"2020-10-21T12:16:11Z","timestamp":1603282571000},"page":"230-238","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":16,"title":["Tutorial: Triheptanoin and Nutrition Management for Treatment of Long\u2010Chain Fatty Acid Oxidation Disorders"],"prefix":"10.1002","volume":"45","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-2856-775X","authenticated-orcid":false,"given":"Marie K.","family":"Norris","sequence":"first","affiliation":[{"name":"Department of Nutrition and Integrative Physiology University of Utah  Salt Lake City Utah USA"},{"name":"Department of Nutrition, Biochemical Genetics Seattle Children's Hospital  Seattle Washington USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-6975-8552","authenticated-orcid":false,"given":"Anna I.","family":"Scott","sequence":"additional","affiliation":[{"name":"Department of Laboratories, Biochemical Genetics Seattle Children's Hospital  Seattle Washington USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-2694-2958","authenticated-orcid":false,"given":"Sarah","family":"Sullivan","sequence":"additional","affiliation":[{"name":"Department of Nutrition, Biochemical Genetics Seattle Children's Hospital  Seattle Washington USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Irene J.","family":"Chang","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Biochemical Genetics University of Washington, Seattle Children's Hospital  Seattle Washington USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-4705-6434","authenticated-orcid":false,"given":"Christina","family":"Lam","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Biochemical Genetics University of Washington, Seattle Children's Hospital  Seattle Washington USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Angela","family":"Sun","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Biochemical Genetics University of Washington, Seattle Children's Hospital  Seattle Washington USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sihoun","family":"Hahn","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Biochemical Genetics University of Washington, Seattle Children's Hospital  Seattle Washington USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jenny M.","family":"Thies","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Biochemical Genetics University of Washington, Seattle Children's Hospital  Seattle Washington USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Melissa","family":"Gunnarson","sequence":"additional","affiliation":[{"name":"Department of Nutrition, Biochemical Genetics Seattle Children's Hospital  Seattle Washington USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kelly N.","family":"McKean","sequence":"additional","affiliation":[{"name":"Department of Nutrition, Biochemical Genetics Seattle Children's Hospital  Seattle Washington USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"suffix":"II","given":"J. Lawrence","family":"Merritt","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Biochemical Genetics University of Washington, Seattle Children's Hospital  Seattle Washington USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2020,11,11]]},"reference":[{"key":"e_1_2_7_2_1","doi-asserted-by":"publisher","DOI":"10.1007\/8904_2014_382"},{"key":"e_1_2_7_3_1","doi-asserted-by":"publisher","DOI":"10.1007\/s10545-012-9542-6"},{"key":"e_1_2_7_4_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2016.08.008"},{"key":"e_1_2_7_5_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2015.10.005"},{"key":"e_1_2_7_6_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2015.06.006"},{"key":"e_1_2_7_7_1","doi-asserted-by":"publisher","DOI":"10.1002\/jimd.12038"},{"key":"e_1_2_7_8_1","doi-asserted-by":"publisher","DOI":"10.1007\/s10545-017-0085-8"},{"key":"e_1_2_7_9_1","doi-asserted-by":"publisher","DOI":"10.1023\/A:1005548201355"},{"key":"e_1_2_7_10_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0022-3476(97)70157-4"},{"key":"e_1_2_7_11_1","doi-asserted-by":"publisher","DOI":"10.1038\/gim.2017.194"},{"key":"e_1_2_7_12_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.amjcard.2009.03.058"}],"container-title":["Journal of Parenteral and Enteral Nutrition"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/jpen.2034","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/full-xml\/10.1002\/jpen.2034","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/aspenjournals.onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/jpen.2034","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,10,19]],"date-time":"2025-10-19T10:47:38Z","timestamp":1760870858000},"score":1,"resource":{"primary":{"URL":"https:\/\/aspenjournals.onlinelibrary.wiley.com\/doi\/10.1002\/jpen.2034"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2020,11,11]]},"references-count":11,"journal-issue":{"issue":"2","published-print":{"date-parts":[[2021,2]]}},"alternative-id":["10.1002\/jpen.2034"],"URL":"https:\/\/doi.org\/10.1002\/jpen.2034","archive":["Portico"],"relation":{},"ISSN":["0148-6071","1941-2444"],"issn-type":[{"value":"0148-6071","type":"print"},{"value":"1941-2444","type":"electronic"}],"subject":[],"published":{"date-parts":[[2020,11,11]]},"assertion":[{"value":"2020-07-09","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2020-10-14","order":2,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2020-11-11","order":3,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}]}}