{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,6]],"date-time":"2026-10-06T22:53:59Z","timestamp":1791327239028,"version":"4.3.1"},"reference-count":43,"publisher":"Wiley","issue":"3","license":[{"start":{"date-parts":[[2020,10,29]],"date-time":"2020-10-29T00:00:00Z","timestamp":1603929600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100003061","name":"Erasmus Medisch Centrum","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100003061","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["J of Inher Metab Disea"],"published-print":{"date-parts":[[2021,5]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Background<\/jats:title>\n                    <jats:p>This study provides a general overview on liver and\/or kidney transplantation in patients with an amino and organic acid\u2010related disorder (AOA) with the aim to investigate patient characteristics and global outcome in Europe. This study was an initiative of the E\u2010IMD and the AOA subnetwork of MetabERN.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>A questionnaire was sent to all clinically active European Society for the Study of Inborn Errors of Metabolism (SSIEM) members. The questionnaire focused on transplanted individuals with methylmalonic acidemia (MMA), propionic acidemia (PA), maple syrup urine disease (MSUD), and urea\u2010cycle disorders (UCDs).<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>\n                      We identified 280 transplanted AOA patients (liver transplantation in 20\u2009MMA, 37 PA, 47 MSUD, and 111 UCD patients, kidney or combined liver and kidney transplantation in 57\u2009MMA patients and undefined transplantation type in 8\u2009MMA patients), followed by 51 metabolic centers. At a median follow\u2010up of 3.5\u2009years, posttransplant survival ranged between 78% and 100%, being the lowest in PA patients. Overall, the risk of mortality was highest within 14\u2009days posttransplantation. Neurological complications were mainly reported in Mut\n                      <jats:sup>0<\/jats:sup>\n                      type MMA (\n                      <jats:italic>n<\/jats:italic>\n                      \u00a0=\u20098). Nonneurological complications occurred in MMA (\n                      <jats:italic>n<\/jats:italic>\n                      \u00a0=\u200928), PA (\n                      <jats:italic>n<\/jats:italic>\n                      \u00a0=\u20097), and UCD (\n                      <jats:italic>n<\/jats:italic>\n                      \u00a0=\u200914) patients, while it was virtually absent in MSUD patients. Only 116\/280 patients were psychologically tested. In all, except MSUD patients, the intelligence quotient (IQ) remained unchanged in the majority (76\/94, 81%). Forty\u2010one percentage (9\/22) of MSUD patient showed improved IQ.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusion<\/jats:title>\n                    <jats:p>The survival in AOA individuals receiving liver and\/or kidney transplantation seems satisfactory. Evidence\u2010based guidelines, systematic data collection, and improved cooperation between transplantation centers and European Reference Networks are indispensable to improve patient care and outcomes.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1002\/jimd.12318","type":"journal-article","created":{"date-parts":[[2020,9,30]],"date-time":"2020-09-30T05:47:37Z","timestamp":1601444857000},"page":"593-605","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":56,"title":["Liver and\/or kidney transplantation in amino and organic acid\u2010related inborn errors of metabolism: An overview on European data"],"prefix":"10.1002","volume":"44","author":[{"given":"Femke","family":"Molema","sequence":"first","affiliation":[{"name":"Department of Pediatrics, Center for Lysosomal and Metabolic Diseases Erasmus MC University Medical Center, AOA subgroup MetabERN  Rotterdam The Netherlands"},{"name":"Subnetwork for Amino and Organic Acid\u2010Related Disorders (AOA) European Reference Network for Hereditary Metabolic Disorders (MetabERN)  Udine Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Diego","family":"Martinelli","sequence":"additional","affiliation":[{"name":"Subnetwork for Amino and Organic Acid\u2010Related Disorders (AOA) European Reference Network for Hereditary Metabolic Disorders (MetabERN)  Udine Italy"},{"name":"U.O.C. Patologia Metabolica Ospedale Pediatrico Bambino Ges\u00f9, AOA Subgroup MetabERN  Rome Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Friederike","family":"H\u00f6rster","sequence":"additional","affiliation":[{"name":"Subnetwork for Amino and Organic Acid\u2010Related Disorders (AOA) European Reference Network for Hereditary Metabolic Disorders (MetabERN)  Udine Italy"},{"name":"Centre for Child and Adolescent Medicine, Division of Neuropaediatrics and Metabolic Medicine University Hospital Heidelberg, AOA Subgroup MetabERN  Heidelberg Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Stefan","family":"K\u00f6lker","sequence":"additional","affiliation":[{"name":"Subnetwork for Amino and Organic Acid\u2010Related Disorders (AOA) European Reference Network for Hereditary Metabolic Disorders (MetabERN)  Udine Italy"},{"name":"Centre for Child and Adolescent Medicine, Division of Neuropaediatrics and Metabolic Medicine University Hospital Heidelberg, AOA Subgroup MetabERN  Heidelberg Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Trine","family":"Tangeraas","sequence":"additional","affiliation":[{"name":"Subnetwork for Amino and Organic Acid\u2010Related Disorders (AOA) European Reference Network for Hereditary Metabolic Disorders (MetabERN)  Udine Italy"},{"name":"Department of Paediatric and Adolescent Medicine, AOA subgroup MetabERN Oslo University Hospital Rikshospitalet  Oslo Norway"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Barbara","family":"de Koning","sequence":"additional","affiliation":[{"name":"Department of Paediatric Gastro\u2010Enterology Erasmus University Medical Center  Rotterdam The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Carlo","family":"Dionisi\u2010Vici","sequence":"additional","affiliation":[{"name":"Subnetwork for Amino and Organic Acid\u2010Related Disorders (AOA) European Reference Network for Hereditary Metabolic Disorders (MetabERN)  Udine Italy"},{"name":"U.O.C. Patologia Metabolica Ospedale Pediatrico Bambino Ges\u00f9, AOA Subgroup MetabERN  Rome Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Monique","family":"Williams","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Center for Lysosomal and Metabolic Diseases Erasmus MC University Medical Center, AOA subgroup MetabERN  Rotterdam The Netherlands"},{"name":"Subnetwork for Amino and Organic Acid\u2010Related Disorders (AOA) European Reference Network for Hereditary Metabolic Disorders (MetabERN)  Udine Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"name":"additional individual contributors of MetabERN","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2020,10,29]]},"reference":[{"key":"e_1_2_10_2_1","doi-asserted-by":"publisher","DOI":"10.1111\/petr.12746"},{"key":"e_1_2_10_3_1","doi-asserted-by":"publisher","DOI":"10.1007\/s10545-015-9839-3"},{"key":"e_1_2_10_4_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2014.01.006"},{"key":"e_1_2_10_5_1","doi-asserted-by":"publisher","DOI":"10.1186\/1750-1172-7-32"},{"key":"e_1_2_10_6_1","doi-asserted-by":"publisher","DOI":"10.1067\/mpd.2001.111838"},{"key":"e_1_2_10_7_1","doi-asserted-by":"publisher","DOI":"10.1023\/A:1005317909946"},{"key":"e_1_2_10_8_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2006.04.003"},{"issue":"116","key":"e_1_2_10_9_1","first-page":"e111","article-title":"Liver transplantation for classical maple syrup urine disease: long\u2010term follow\u2010up in 37 patients and comparative united network for organ sharing experience","volume":"160","author":"Mazariegos GV","year":"2012","journal-title":"J Pediatr"},{"key":"e_1_2_10_10_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0022-3476(89)80843-1"},{"key":"e_1_2_10_11_1","unstructured":"MurphyMSPM CollinsJ.Liver transplantation in a child with propionic acidaemia. 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