{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,7,24]],"date-time":"2026-07-24T09:01:45Z","timestamp":1784883705956,"version":"3.55.0"},"reference-count":33,"publisher":"Wiley","issue":"2","license":[{"start":{"date-parts":[[2026,3,13]],"date-time":"2026-03-13T00:00:00Z","timestamp":1773360000000},"content-version":"vor","delay-in-days":12,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"},{"start":{"date-parts":[[2026,3,1]],"date-time":"2026-03-01T00:00:00Z","timestamp":1772323200000},"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":["J of Inher Metab Disea"],"published-print":{"date-parts":[[2026,3]]},"abstract":"<jats:title>ABSTRACT<\/jats:title>\n                  <jats:p>Transplantation is an effective therapeutic option to improve quality of life in patients with severe methylmalonic acidemia (MMA). However, data regarding neurological complications following transplantation remain limited. A retrospective, single\u2010center study was conducted at Necker Hospital (France), including MMA patients who underwent kidney (KT), liver (LT), or liver\u2010kidney transplantation (LKT) between 2007 and 2022. Our aim was to evaluate acute neurological complications occurring after transplantation, focusing on clinical features, laboratory parameters and neuroimaging. Sensorineural complications were also assessed. Thirty\u2010five patients were included, 21 had undergone LKT, 10 LT and 4 KT. The median age at transplantation was 10.1\u2009years, with a median follow\u2010up of 5\u2009years. Tacrolimus was used in 91% of patients. Acute neurological complications likely related to MMA occurred in 17% of patients. They include Leigh syndrome with identifiable triggers, observed in 4 patients (2 early and 2 late\u2010post\u2010transplantation), leading to one early\u2010 and one late\u2010onset death. Stroke\u2010like episodes occurred in 2 patients. Non\u2010epileptic myoclonus, likely related to calcineurin inhibitor (CNI), were reported in 31% of patients. Pre\u2010transplant brain MRI showed nonspecific abnormalities in 31% of patients and remained stable afterwards. Post\u2010transplant ophthalmological data available for 17 patients showed 3 optic atrophies. No acute vision or hearing loss was reported. Although transplantation improves metabolic control in MMA, acute neurological complications can still occur following a triggering event, even years after transplantation. The risk may arise from sensitivity to CNI\u2010induced neurotoxicity. Pre\u2010 and post\u2010transplant neuroimaging, emergency metabolic protocols, and tailored immunosuppression are essential for long\u2010term management.<\/jats:p>","DOI":"10.1002\/jimd.70165","type":"journal-article","created":{"date-parts":[[2026,3,13]],"date-time":"2026-03-13T13:33:55Z","timestamp":1773408835000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":1,"title":["Acute Neurological Complications After Transplantation in Methylmalonic Acidemia: A 35\u2010Patient French Cohort"],"prefix":"10.1002","volume":"49","author":[{"given":"Ad\u00e9la\u00efde","family":"Vissac","sequence":"first","affiliation":[{"name":"Reference Center for Inborn Error of Metabolism, H\u00f4pital Necker\u2010Enfants Malades, APHP and Universit\u00e9 Paris Cit\u00e9, Fili\u00e8re G2m, MetabERN  Paris 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France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Chlo\u00e9","family":"de Puyraimond","sequence":"additional","affiliation":[{"name":"Reference Center for Inborn Error of Metabolism, H\u00f4pital Necker\u2010Enfants Malades, APHP and Universit\u00e9 Paris Cit\u00e9, Fili\u00e8re G2m, MetabERN  Paris France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Margaux","family":"Gaschignard","sequence":"additional","affiliation":[{"name":"Reference Center for Inborn Error of Metabolism, H\u00f4pital Necker\u2010Enfants Malades, APHP and Universit\u00e9 Paris Cit\u00e9, Fili\u00e8re G2m, MetabERN  Paris France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Tristan","family":"Mekdade","sequence":"additional","affiliation":[{"name":"Reference Center for Inborn Error of Metabolism, H\u00f4pital Necker\u2010Enfants Malades, APHP and Universit\u00e9 Paris Cit\u00e9, Fili\u00e8re G2m, MetabERN  Paris 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