{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,14]],"date-time":"2026-10-14T22:12:08Z","timestamp":1792015928722,"version":"4.4.0"},"reference-count":16,"publisher":"Springer Science and Business Media LLC","issue":"6","license":[{"start":{"date-parts":[[2025,5,31]],"date-time":"2025-05-31T00:00:00Z","timestamp":1748649600000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0"},{"start":{"date-parts":[[2025,5,31]],"date-time":"2025-05-31T00:00:00Z","timestamp":1748649600000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0"}],"funder":[{"name":"Social Science University of Ankara"}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["Eur J Pediatr"],"abstract":"<jats:title>Abstract<\/jats:title>\n          <jats:p>Long-chain fatty acid oxidation disorders (LC-FAOD) are a rare metabolic condition that results in impaired fatty acid utilization, leading to metabolic crises, hospitalization, and reduced quality of life. Despite dietary management, many patients experience ongoing complications. Triheptanoin, a seven-carbon triglyceride, has emerged as a therapeutic alternative by providing an energy source and supporting metabolic stability. This study aims to evaluate the clinical outcomes of LC-FAOD patients receiving triheptanoin therapy in T\u00fcrkiye. A retrospective nationwide study was conducted to analyze 14 patients with LC-FAOD who received oral triheptanoin as part of a compassionate use program in T\u00fcrkiye. The study collected data on emergency department visits, hospitalizations, metabolic decompensation episodes, creatine kinase (CK) levels, hypoglycemia, and cardiac function. Additionally, patient-reported outcomes were assessed through surveys. The findings of the study demonstrated that triheptanoin treatment led to a significant reduction in the number of emergency service applications and hospitalizations per month (<jats:italic>p<\/jats:italic>\u2009&lt;\u20090.01). A notable decrease in the frequency of myalgia attacks was observed, while the decline in rhabdomyolysis episodes did not reach statistical significance. Furthermore, creatine kinase levels during metabolic crises exhibited a substantial decrease following triheptanoin therapy (<jats:italic>p<\/jats:italic>\u2009&lt;\u20090.0001).Among patients with cardiomyopathy, cardiac function showed improvement in four out of seven patients. Survey data indicated an improvement in appetite, physical performance, and overall quality of life. <\/jats:p>\n          <jats:p>\n            <jats:italic>Conclusion<\/jats:italic>: Triheptanoin treatment has been demonstrated to be associated with significant clinical improvements in patients diagnosed with LC-FAOD, including a reduction in the frequency of emergency department visits, hospitalizations, and metabolic crises. These findings provide support for the utilization of triheptanoin as a therapeutic approach that holds promise in the management of LC-FAOD.<\/jats:p>\n          <jats:p>\n            <jats:table-wrap>\n              <jats:table>\n                <jats:tbody>\n                  <jats:tr>\n                    <jats:td align=\"left\">\n                      <jats:bold>What is Known:<\/jats:bold>\n                    <\/jats:td>\n                  <\/jats:tr>\n                  <jats:tr>\n                    <jats:td align=\"left\">\u2022\u00a0<jats:italic>Long-chain fatty acid oxidation disorders (LC-FAOD) are associated with significant morbidity due to metabolic crises, despite conventional dietary treatment including medium-chain triglycerides (MCT).<\/jats:italic>\n                    <\/jats:td>\n                  <\/jats:tr>\n                  <jats:tr>\n                    <jats:td align=\"left\">\n                      <jats:bold>What is New:<\/jats:bold>\n                    <\/jats:td>\n                  <\/jats:tr>\n                  <jats:tr>\n                    <jats:td align=\"left\">\u2022\u00a0<jats:italic>This nationwide study demonstrates that triheptanoin therapy significantly reduces emergency visits, hospitalizations, and creatine kinase levels during crises, and improves patient-reported outcomes, including physical activity and quality of life, in LC-FAOD patients in T\u00fcrkiye.<\/jats:italic>\n                    <\/jats:td>\n                  <\/jats:tr>\n                <\/jats:tbody>\n              <\/jats:table>\n            <\/jats:table-wrap>\n          <\/jats:p>","DOI":"10.1007\/s00431-025-06216-3","type":"journal-article","created":{"date-parts":[[2025,5,31]],"date-time":"2025-05-31T07:32:13Z","timestamp":1748676733000},"update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":5,"title":["The effect of triheptanoin treatment on clinical and laboratory outcomes in patients with long-chain fatty acid oxidation disorder"],"prefix":"10.1007","volume":"184","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-7238-2894","authenticated-orcid":false,"given":"Engin","family":"K\u00f6se","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Asl\u0131","family":"\u0130nci","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Havva","family":"Yaz\u0131c\u0131","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Tanyel","family":"Z\u00fcbario\u011flu","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sebile","family":"K\u0131lavuz","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"K\u0131smet","family":"\u00c7\u0131k\u0131","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Esra","family":"Er","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mehmet Cihan","family":"Balc\u0131","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ay\u00e7a Burcu","family":"Kahraman","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Neslihan","family":"\u00d6nenli Mungan","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"\u00c7i\u011fdem","family":"Aktu\u011flu Zeybek","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sema","family":"Kalkan U\u00e7ar","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Leyla","family":"T\u00fcmer","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Fatma Tuba","family":"Emino\u011flu","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2025,5,31]]},"reference":[{"key":"6216_CR1","doi-asserted-by":"publisher","unstructured":"Yang EC, Fang Z, Tan R, Guo Y, He S, Kruger E, Nedzesky J, Marsden D (2024) Clinical manifestations, healthcare resource utilization, and costs among patients with long-chain fatty acid oxidation disorders: a retrospective claims database analysis. 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