{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,6,9]],"date-time":"2026-06-09T01:54:44Z","timestamp":1780970084151,"version":"3.54.1"},"reference-count":6,"publisher":"Ovid Technologies (Wolters Kluwer Health)","issue":"4","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2018,8]]},"abstract":"<jats:title>Abstract:<\/jats:title>\n          <jats:p>Eltrombopag is an oral thrombopoietin receptor agonist approved for the treatment of patients with chronic idiopathic thrombocytopenic purpura (ITP), who are more than 1 year old, and show poor response to first-line therapy. ITP is a hematological disorder characterized by isolated thrombocytopenia in the absence of secondary causes or disorders. Eltrombopag is generally well tolerated in the pediatric population; therefore, therapeutic drug monitoring (TDM) is not usually performed in clinical practice.<\/jats:p>\n          <jats:p>We presented the case study of a 3-year-old girl with chronic ITP. She arrived in the pediatric intensive care unit with acute liver failure due to eltrombopag toxicity despite taking the standard drug dosage. A very high eltrombopag plasma concentration, indicating drug toxicity, was found through TDM. The patient also carried the allelic variations that are involved in drug metabolism [CYP2C8 and UDP glucuronosyltransferase (UGT) 1A1 (UGT1A1)] and drug cellular transportation [ABCG2 (ATP-binding cassette G2)]. This observation highlights the importance of using TDM and pharmacogenetic approaches to manage patients' unusual complications associated with standard pharmacological treatment regimens.<\/jats:p>","DOI":"10.1097\/ftd.0000000000000522","type":"journal-article","created":{"date-parts":[[2018,4,20]],"date-time":"2018-04-20T22:09:47Z","timestamp":1524262187000},"page":"386-388","source":"Crossref","is-referenced-by-count":22,"title":["Eltrombopag-Induced Acute Liver Failure in a Pediatric Patient: A Pharmacokinetic and Pharmacogenetic Analysis"],"prefix":"10.1097","volume":"40","author":[{"given":"Marco","family":"Marano","sequence":"first","affiliation":[{"name":"Pediatric Intensive Care Unit, Bambino Ges\u00f9 Children Hospital IRCSS;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jessica","family":"Serafinelli","sequence":"additional","affiliation":[{"name":"Division of Immunology and Infectious Diseases, University-Hospital Pediatric Department (DPUO), Bambino Ges\u00f9 Children Hospital IRCSS, \u201cTor Vergata University\u201d;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sara","family":"Cairoli","sequence":"additional","affiliation":[{"name":"Laboratory of Analytical Biochemistry, Bambino Ges\u00f9 Children Hospital IRCCS;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Diego","family":"Martinelli","sequence":"additional","affiliation":[{"name":"Division of Metabolism, Bambino Ges\u00f9 Children Hospital IRCCS;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mara","family":"Pisani","sequence":"additional","affiliation":[{"name":"DEA, IRCCS Bambino Ges\u00f9 Children Hospital IRCSS;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Giuseppe","family":"Palumbo","sequence":"additional","affiliation":[{"name":"Department of Pediatric Hematology\/Oncology, Bambino Ges\u00f9 Children Hospital IRCCS, Rome, Italy; and"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Maria G.","family":"Cefalo","sequence":"additional","affiliation":[{"name":"Department of Pediatric Hematology\/Oncology, Bambino Ges\u00f9 Children Hospital IRCCS, Rome, Italy; and"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Corrado","family":"Cecchetti","sequence":"additional","affiliation":[{"name":"Pediatric Intensive Care Unit, Bambino Ges\u00f9 Children Hospital IRCSS;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Matteo","family":"Di Nardo","sequence":"additional","affiliation":[{"name":"Pediatric Intensive Care Unit, Bambino Ges\u00f9 Children Hospital IRCSS;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Felicia S.","family":"Falvella","sequence":"additional","affiliation":[{"name":"Unit of Clinical Pharmacology, Department of Laboratory Medicine, ASST Fatebenefratelli-Sacco, Milan, Italy."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Bianca M.","family":"Goffredo","sequence":"additional","affiliation":[{"name":"Laboratory of Analytical Biochemistry, Bambino Ges\u00f9 Children Hospital IRCCS;"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"276","reference":[{"key":"R1-20230814","doi-asserted-by":"crossref","first-page":"2386","DOI":"10.1182\/blood-2008-07-162503","article-title":"Standardization of terminology, definitions and outcome criteria in immune thrombocytopenic purpura of adults and children: report from an international working group","volume":"113","author":"Rodeghiero","year":"2009","journal-title":"Blood"},{"key":"R2-20230814","doi-asserted-by":"crossref","first-page":"739","DOI":"10.1177\/0091270010372106","article-title":"Effect of hepatic or renal impairment on eltrombopag pharmacokinetics","volume":"51","author":"Bauman","year":"2011","journal-title":"J Clin Pharmacol."},{"key":"R3-20230814","doi-asserted-by":"crossref","first-page":"869","DOI":"10.1007\/s40265-016-0581-4","article-title":"Eltrombopag: a review in paediatric chronic immune thrombocytopenia","volume":"76","author":"Burness","year":"2016","journal-title":"Drugs"},{"key":"R5-20230814","doi-asserted-by":"crossref","first-page":"68","DOI":"10.1016\/j.jpba.2014.04.028","article-title":"Liquid chromatography-tandem mass spectrometric assay for eltrombopag in 50\u03bcL of human plasma: a pharmacokinetic study","volume":"98","author":"Maddela","year":"2014","journal-title":"J Pharm Biomed Anal."},{"key":"R6-20230814","doi-asserted-by":"crossref","first-page":"652","DOI":"10.1186\/s40064-015-1446-0","article-title":"Development and validation of a LC-MS\/MS method for quantification of hetrombopag for pharmacokinetics study","volume":"4","author":"Chen","year":"2015","journal-title":"Springerplus"},{"key":"R7-20230814","doi-asserted-by":"crossref","first-page":"1649","DOI":"10.1016\/S0140-6736(15)61107-2","article-title":"Eltrombopag for children with chronic immune thrombocytopenia (PETIT2): a randomised, multicentre, placebo-controlled trial","volume":"386","author":"Grainger","year":"2015","journal-title":"Lancet"}],"container-title":["Therapeutic Drug Monitoring"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/journals.lww.com\/00007691-201808000-00002","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,8,14]],"date-time":"2023-08-14T22:15:10Z","timestamp":1692051310000},"score":1,"resource":{"primary":{"URL":"https:\/\/journals.lww.com\/00007691-201808000-00002"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2018,8]]},"references-count":6,"journal-issue":{"issue":"4","published-print":{"date-parts":[[2018]]}},"URL":"https:\/\/doi.org\/10.1097\/ftd.0000000000000522","relation":{},"ISSN":["0163-4356"],"issn-type":[{"value":"0163-4356","type":"print"}],"subject":[],"published":{"date-parts":[[2018,8]]}}}