{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,6]],"date-time":"2026-10-06T08:52:24Z","timestamp":1791276744456,"version":"4.1.0"},"reference-count":18,"publisher":"Wiley","license":[{"start":{"date-parts":[[2026,10,6]],"date-time":"2026-10-06T00:00:00Z","timestamp":1791244800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"},{"start":{"date-parts":[[2026,10,6]],"date-time":"2026-10-06T00:00:00Z","timestamp":1791244800000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/doi.wiley.com\/10.1002\/tdm_license_1.1"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Acta Paediatrica"],"abstract":"ABSTRACT<\/jats:title>\n \n Aim<\/jats:title>\n Over the past four decades, liver transplantation has been performed worldwide for metabolic diseases, mainly to ameliorate the metabolic defect. However, little has been described regarding the indications and outcomes of the procedure.<\/jats:p>\n <\/jats:sec>\n \n Methods<\/jats:title>\n Data were collected retrospectively from medical records of 28 paediatric patients with metabolic diseases who underwent liver transplantation in Israel since 2008, considering both metabolic and liver transplant outcomes.<\/jats:p>\n <\/jats:sec>\n \n Results<\/jats:title>\n \n The underlying diagnoses for liver transplantation included urea cycle defects, organic acidemias, aminoacidopathies, glycogen storage disease, congenital disorder of glycosylation and a mitochondrial disorder. The mean age at transplantation was 49.5\u2009\u00b1\u200949.8\u2009months. The mean follow\u2010up time was 53.1\u2009\u00b1\u200946.8\u2009months. Metabolic crises per year decreased, from a mean 3.8\u2009\u00b1\u20093.9 pre\u2010transplant to none post\u2010transplant (\n p<\/jats:italic>\n \u2009<\u20090.001), and hospitalization frequency decreased from 7.1\u2009\u00b1\u20095.4 to 1.6\u2009\u00b1\u20091.7 (\n p<\/jats:italic>\n \u2009<\u20090.001). Both surgical and late\u2010term complications were mostly similar to those of nonmetabolic paediatric liver transplantations. Only three patients exhibited new neurological issues after transplant, two of which were likely attributed to the neurotoxic effects of immunosuppression.\n <\/jats:p>\n <\/jats:sec>\n \n Conclusions<\/jats:title>\n Liver transplantation is a promising alternative for the treatment of certain metabolic diseases, offering potential benefits in metabolic crises and hospitalization frequency, dietary restrictions, medication regimens and neurodevelopmental status.<\/jats:p>\n <\/jats:sec>","DOI":"10.1111\/apa.70792","type":"journal-article","created":{"date-parts":[[2026,10,6]],"date-time":"2026-10-06T08:18:06Z","timestamp":1791274686000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["Liver Transplantation in Inborn Errors of Metabolism: A Retrospective Study of a National Experience"],"prefix":"10.1111","author":[{"ORCID":"https:\/\/orcid.org\/0009-0008-5089-5128","authenticated-orcid":false,"given":"Adi","family":"Efron\u2010Srour","sequence":"first","affiliation":[{"name":"Pediatric Department A Schneider Children's Medical Center of Israel Petah\u2010Tikva Israel"},{"name":"Gray Faculty of Medical and Health Sciences Tel\u2010Aviv University Tel\u2010Aviv Israel"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Eyal","family":"Reller","sequence":"additional","affiliation":[{"name":"Nutrition and Dietary Unit Schneider Children's Medical Center of Israel Petah\u2010Tikva Israel"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michal","family":"Gafner","sequence":"additional","affiliation":[{"name":"Pediatric Department B Schneider Children's Medical Center of Israel Petah\u2010Tikva Israel"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Danit","family":"Javasky","sequence":"additional","affiliation":[{"name":"Nutrition and Dietary Unit Schneider Children's Medical Center of Israel Petah\u2010Tikva Israel"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Chen Barazani","family":"Chayat","sequence":"additional","affiliation":[{"name":"Metabolic Laboratory Schneider Children's Medical Center of Israel Petah\u2010Tikva Israel"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Galit","family":"Tal","sequence":"additional","affiliation":[{"name":"Metabolic Clinic Ruth Rappaport Children's Hospital, Rambam Health Care Campus Haifa Israel"},{"name":"Rappaport Faculty of Medicine Technion\u2010Israel Institute of Technology Haifa Israel"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6975-4909","authenticated-orcid":false,"given":"Orna","family":"Staretz\u2010Chacham","sequence":"additional","affiliation":[{"name":"Metabolic Clinic, Pediatric Division, Soroka University Medical Center Ben Gurion University Beer Sheva Israel"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ronen","family":"Spiegel","sequence":"additional","affiliation":[{"name":"Department of Pediatrics B, Metabolic Service Emek Medical Center Afula Israel"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yael","family":"Mozer\u2010Glassberg","sequence":"additional","affiliation":[{"name":"Gray Faculty of Medical and Health Sciences Tel\u2010Aviv University Tel\u2010Aviv Israel"},{"name":"Gastroenterology, Nutrition and Liver Diseases Schneider Children's Medical Center of Israel Petah\u2010Tikva Israel"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michal","family":"Rozenfeld Bar\u2010Lev","sequence":"additional","affiliation":[{"name":"Gray Faculty of Medical and Health Sciences Tel\u2010Aviv University Tel\u2010Aviv Israel"},{"name":"Gastroenterology, Nutrition and Liver Diseases Schneider Children's Medical Center of Israel Petah\u2010Tikva Israel"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michael","family":"Gurevich","sequence":"additional","affiliation":[{"name":"The Organ Transplantation Division Schneider Children's Medical Center of Israel Petah\u2010Tikva Israel"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0410-175X","authenticated-orcid":false,"given":"Orith","family":"Waisbourd\u2010Zinman","sequence":"additional","affiliation":[{"name":"Gray Faculty of Medical and Health Sciences Tel\u2010Aviv University Tel\u2010Aviv Israel"},{"name":"Gastroenterology, Nutrition and Liver Diseases Schneider Children's Medical Center of Israel Petah\u2010Tikva Israel"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-4420-1480","authenticated-orcid":false,"given":"Yuval E.","family":"Landau","sequence":"additional","affiliation":[{"name":"Gray Faculty of Medical and Health Sciences Tel\u2010Aviv University Tel\u2010Aviv Israel"},{"name":"Metabolic Diseases Clinic Schneider Children's Medical Center of Israel Petah\u2010Tikva Israel"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2026,10,6]]},"reference":[{"key":"e_1_2_12_2_1","doi-asserted-by":"crossref","first-page":"14","DOI":"10.1007\/978-3-662-63123-2","volume-title":"Inborn Metabolic Diseases: Diagnosis and Treatment","author":"Saudubray J. M.","year":"2022"},{"key":"e_1_2_12_3_1","doi-asserted-by":"crossref","first-page":"756","DOI":"10.1111\/petr.12741","article-title":"Liver Transplantation for Pediatric Inherited Metabolic Disorders: Considerations for Indications, Complications, and Perioperative Management","volume":"20","author":"Oishi K.","year":"2016","journal-title":"Pediatric Transplantation"},{"key":"e_1_2_12_4_1","doi-asserted-by":"crossref","first-page":"911","DOI":"10.1002\/lt.25433","article-title":"Evolving Trends in Liver Transplant for Metabolic Liver Disease in the United States","volume":"25","author":"McKiernan P. J.","year":"2019","journal-title":"Liver Transplantation: Official Publication of the American Association for the Study of Liver Diseases and the International Liver Transplantation Society"},{"key":"e_1_2_12_5_1","doi-asserted-by":"crossref","first-page":"88","DOI":"10.1002\/jmd2.12219","article-title":"Liver Transplantation in Children With Inborn Errors of Metabolism: 30\u2009Years Experience in NSW, Australia","volume":"60","author":"Elserafy N.","year":"2021","journal-title":"JIMD Reports"},{"key":"e_1_2_12_6_1","doi-asserted-by":"crossref","first-page":"593","DOI":"10.1002\/jimd.12318","article-title":"Liver and\/or Kidney Transplantation in Amino and Organic Acid\u2010Related Inborn Errors of Metabolism: An Overview on European Data","volume":"44","author":"Molema F.","year":"2021","journal-title":"Journal of Inherited Metabolic Disease"},{"issue":"10","key":"e_1_2_12_7_1","doi-asserted-by":"crossref","first-page":"1351","DOI":"10.4254\/wjh.v13.i10.1351","article-title":"Liver Transplantation for Pediatric Inherited Metabolic Liver Diseases","volume":"13","author":"Vimalesvaran S.","year":"2021","journal-title":"World Journal of Hepatology"},{"key":"e_1_2_12_8_1","doi-asserted-by":"crossref","first-page":"6","DOI":"10.1111\/petr.12196","article-title":"Living Donor Liver Transplantation for Pediatric Patients With Metabolic Disorders: The Japanese Multicenter Registry","volume":"18","author":"Kasahara M.","year":"2014","journal-title":"Pediatric Transplantation"},{"issue":"6","key":"e_1_2_12_9_1","doi-asserted-by":"crossref","first-page":"161","DOI":"10.5500\/wjt.v11.i6.161","article-title":"Pediatric Metabolic Liver Diseases: Evolving Role of Liver Transplantation","volume":"11","author":"Menon L.","year":"2021","journal-title":"World Journal of Transplantation"},{"key":"e_1_2_12_10_1","doi-asserted-by":"crossref","first-page":"87","DOI":"10.1097\/TP.0b013e31818bc0c4","article-title":"Pediatric Liver Transplantation for Metabolic Liver Disease: Experience at King's College Hospital","volume":"87","author":"Sze Y. K.","year":"2009","journal-title":"Transplantation"},{"key":"e_1_2_12_11_1","doi-asserted-by":"crossref","DOI":"10.1002\/jimd.12785","article-title":"Long\u2010Term Neurodevelopmental Outcomes Following Liver Transplantation for Metabolic Disease\u2010a Single Center Experience","volume":"48","author":"Patterson C.","year":"2024","journal-title":"Journal of Inherited Metabolic Disease"},{"key":"e_1_2_12_12_1","article-title":"Solid Organ Transplantation in Methylmalonic Academia and Propionic Academia: A Points to Consider Statement of the American College of Medical Genetic and Genomics (ACMG)","volume":"1098","author":"Sen K.","year":"2022","journal-title":"Genetics in Medicine"},{"key":"e_1_2_12_13_1","doi-asserted-by":"crossref","first-page":"62","DOI":"10.1186\/s13023-022-02233-9","article-title":"Therapeutic Potential of Living Donor Liver Transplantation From Heterozygous Carrier Donors in Children With Propionic Acidemia","volume":"17","author":"Zeng Z.\u2010G.","year":"2022","journal-title":"Orphanet Journal of Rare Diseases"},{"key":"e_1_2_12_14_1","doi-asserted-by":"crossref","first-page":"1311","DOI":"10.1002\/jimd.12415","article-title":"Role of Liver Transplantation in Urea Cycle Disorders: Report From a Nationwide Study in Japan","volume":"44","author":"Kido J.","year":"2021","journal-title":"Journal of Inherited Metabolic Disease"},{"issue":"3","key":"e_1_2_12_15_1","doi-asserted-by":"crossref","DOI":"10.1111\/petr.14727","article-title":"Biliary Strictures Post Pediatric Liver Transplantation\u2010Incidence and Risk Factors in a Single Tertiary Referral Transplant Center","volume":"28","author":"Vingrovich O.","year":"2024","journal-title":"Pediatric Transplantation"},{"issue":"7","key":"e_1_2_12_16_1","doi-asserted-by":"crossref","DOI":"10.1111\/petr.14872","article-title":"Perioperative Complications After Pediatric Liver Transplantation\u2010A Retrospective Analysis of 421 Cases","volume":"28","author":"Weigle C. A.","year":"2024","journal-title":"Pediatric Transplantation"},{"issue":"1","key":"e_1_2_12_17_1","doi-asserted-by":"crossref","first-page":"11","DOI":"10.1097\/MPG.0000000000002952","article-title":"Living Related Liver Transplantation for Metabolic Liver Diseases in Children","volume":"72","author":"Sood V.","year":"2021","journal-title":"Journal of Pediatric Gastroenterology and Nutrition"},{"key":"e_1_2_12_18_1","doi-asserted-by":"crossref","first-page":"336","DOI":"10.1016\/j.ymgme.2016.01.005","article-title":"Living Related Versus Deceased Donor Liver Transplantation for Maple Syrup Urine Disease","volume":"117","author":"Feier F.","year":"2016","journal-title":"Molecular Genetics and Metabolism"},{"key":"e_1_2_12_19_1","doi-asserted-by":"crossref","first-page":"178","DOI":"10.1016\/j.ymgme.2016.04.009","article-title":"Solid Organ Transplantation in Mitochondrial Disease: Proceed With Caution","volume":"118","author":"Parikh S.","year":"2016","journal-title":"Molecular Genetics and Metabolism"}],"container-title":["Acta Paediatrica"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1111\/apa.70792","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/full-xml\/10.1111\/apa.70792","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1111\/apa.70792","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,10,6]],"date-time":"2026-10-06T08:18:09Z","timestamp":1791274689000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1111\/apa.70792"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2026,10,6]]},"references-count":18,"alternative-id":["10.1111\/apa.70792"],"URL":"https:\/\/doi.org\/10.1111\/apa.70792","archive":["Portico"],"relation":{},"ISSN":["0803-5253","1651-2227"],"issn-type":[{"value":"0803-5253","type":"print"},{"value":"1651-2227","type":"electronic"}],"subject":[],"published":{"date-parts":[[2026,10,6]]},"assertion":[{"value":"2026-06-25","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2026-09-30","order":2,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2026-10-06","order":3,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}],"article-number":"apa.70792"}}