{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,18]],"date-time":"2026-08-18T14:12:22Z","timestamp":1787062342729,"version":"build-2736575974"},"reference-count":44,"publisher":"Wiley","issue":"6","license":[{"start":{"date-parts":[[2018,8,29]],"date-time":"2018-08-29T00:00:00Z","timestamp":1535500800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"name":"University Medical Center Rotterdam"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["J of Inher Metab Disea"],"published-print":{"date-parts":[[2018,12]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Background<\/jats:title>\n                    <jats:p>There is increasing evidence that long\u2010term complications in organic acidemias are caused by impaired mitochondrial metabolism. Currently, there is no specific biomarker to monitor mitochondrial dysfunction in organic acidemias. Serum fibroblast growth factor 21 (FGF\u201021) is a biomarker for mitochondrial disease and could be a candidate to monitor mitochondrial function in the deleterious course of disease.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>Data of 17 patients with classical organic acidemias (11 propionic acidemia (PA), four methylmalonic acidemia (MMA) and two isovaleric acidemia (IVA) patients) were included. The clinical course was evaluated; metabolic decompensations and long\u2010term complications were correlated with plasma FGF\u201021 levels. Cardiomyopathy, prolonged QT interval, renal failure, and optic neuropathy were defined as long\u2010term complications.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>\n                      Patients ages ranged from 16 months up to 32 years. Serious long\u2010term complications occurred in eight patients (five PA and three MMA patients). In MMA and PA patients plasma FGF\u201021 levels during stable metabolic periods were significantly higher in patients with long\u2010term complications (\n                      <jats:italic>Mdn<\/jats:italic>\n                      \u2009=\u20092556.0 pg\/ml) compared to patients without (\n                      <jats:italic>Mdn<\/jats:italic>\n                      \u2009=\u2009287.0 pg\/ml). A median plasma FGF\u201021 level above 1500 pg\/ml during a stable metabolic period, measured before the occurrence of long\u2010term complications, had a positive predictive value of 0.83 and a negative predictive value of 1.00 on long\u2010term complications in MMA and PA patients.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusion<\/jats:title>\n                    <jats:p>This study demonstrates the potential role of FGF\u201021 as a biomarker for long\u2010term complications in classical organic acidemias, attributed to mitochondrial dysfunction.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1007\/s10545-018-0244-6","type":"journal-article","created":{"date-parts":[[2018,8,29]],"date-time":"2018-08-29T19:54:11Z","timestamp":1535572451000},"page":"1179-1187","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":31,"title":["Fibroblast growth factor 21 as a biomarker for long\u2010term complications in organic acidemias"],"prefix":"10.1002","volume":"41","author":[{"given":"F.","family":"Molema","sequence":"first","affiliation":[{"name":"Department of Pediatrics Sophia Children's Hospital, Center of Lysosomal and Metabolic Disorders Erasmus University Medical Center Rotterdam Postbus 2060 Rotterdam CB 3000 The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"E. H.","family":"Jacobs","sequence":"additional","affiliation":[{"name":"Department of Pediatrics Sophia Children's Hospital, Center of Lysosomal and Metabolic Disorders Erasmus University Medical Center Rotterdam Postbus 2060 Rotterdam CB 3000 The Netherlands"},{"name":"Department of Clinical Genetics Erasmus University Medical Center Rotterdam The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"W.","family":"Onkenhout","sequence":"additional","affiliation":[{"name":"Department of Pediatrics Sophia Children's Hospital, Center of Lysosomal and Metabolic Disorders Erasmus University Medical Center Rotterdam Postbus 2060 Rotterdam CB 3000 The Netherlands"},{"name":"Department of Clinical Genetics Erasmus University Medical Center Rotterdam The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"G. 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