{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,6,15]],"date-time":"2026-06-15T11:28:23Z","timestamp":1781522903428,"version":"3.54.1"},"reference-count":17,"publisher":"Wiley","issue":"5","license":[{"start":{"date-parts":[[2023,7,23]],"date-time":"2023-07-23T00:00:00Z","timestamp":1690070400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["JIMD Reports"],"published-print":{"date-parts":[[2023,9]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:p>In urea cycle disorders (UCDs) ammonia scavenger drugs, usually sodium\u2010based, have been the mainstay of treatment. Increasingly, glycerol phenylbutyrate (GPB, Ravicti\u00ae) is being used but scant real\u2010world data exist regarding clinical outcomes. A retrospective study of UCD patients initiated on or switched to GPB was performed at a UK centre. Data on population characteristics, treatment aspects, laboratory measurements, and clinical outcomes were collected before and after patients started GPB with a sub\u2010group analysis undertaken for patients with \u226512\u2009months of data before and after starting GPB. UCDs included arginosuccinate synthetase deficiency (<jats:italic>n<\/jats:italic>\u2009=\u20098), arginosuccinate lyase deficiency (<jats:italic>n<\/jats:italic>\u2009=\u20096), ornithine carbamoyltransferase deficiency (<jats:italic>n<\/jats:italic>\u2009=\u20093), and carbamoyl phosphate synthetase 1 deficiency (<jats:italic>n<\/jats:italic>\u2009=\u20093). In the sub\u2010group analysis (<jats:italic>n<\/jats:italic>\u2009=\u200911), GPB resulted in lower plasma ammonia (31 vs. 41\u2009\u03bcmol\/L, <jats:italic>p<\/jats:italic>\u2009=\u20090.037), glutamine (670 vs. 838\u2009\u03bcmol\/L, <jats:italic>p<\/jats:italic>\u2009=\u20090.002), annualised hyperammonaemic episodes (0.2 vs. 1.9, <jats:italic>p<\/jats:italic>\u2009=\u20090.020), hospitalisations (0.5 vs. 2.2, <jats:italic>p<\/jats:italic>\u2009=\u20090.010), and hyperammonaemic episodes resulting in hospitalisation (0.2 vs. 1.6, <jats:italic>p<\/jats:italic>\u2009=\u20090.035) reflecting changes seen in the whole group. Overall, patients exposed to sodium and propylene glycol levels above UK daily limits reduced by 78% and 83% respectively. Mean levels of branched chain amino acids, haemoglobin, and white cell count were unchanged. Two adverse drug reactions (pancytopenia, fatigue\/appetite loss) resolved without GPB discontinuation. Patients\/families preferred GPB for its lower volume, greater palatability and easier administration. GPB appeared to improve biochemical measures and clinical outcomes. The causes are multi\u2010factorial and are likely to include prolonged action of GPB and its good tolerability, even at higher doses, facilitating tighter control of ammonia.<\/jats:p>","DOI":"10.1002\/jmd2.12386","type":"journal-article","created":{"date-parts":[[2023,7,24]],"date-time":"2023-07-24T04:49:22Z","timestamp":1690174162000},"page":"317-326","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":6,"title":["Clinical experience with glycerol phenylbutyrate in 20 patients with urea cycle disorders at a <scp>UK<\/scp> paediatric centre"],"prefix":"10.1002","volume":"64","author":[{"given":"Mildrid","family":"Yeo","sequence":"first","affiliation":[{"name":"Department of Paediatric Inherited Metabolic Disease Great Ormond Street Hospital NHS Foundation Trust and Institute for Child Health  London UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Preeya","family":"Rehsi","sequence":"additional","affiliation":[{"name":"Department of Paediatric Inherited Metabolic Disease Great Ormond Street Hospital NHS Foundation Trust and Institute for Child Health  London UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Megan","family":"Dorman","sequence":"additional","affiliation":[{"name":"Department of Paediatric Inherited Metabolic Disease Great Ormond Street Hospital NHS Foundation Trust and Institute for Child Health  London UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Stephanie","family":"Grunewald","sequence":"additional","affiliation":[{"name":"Department of Paediatric Inherited Metabolic Disease Great Ormond Street Hospital NHS Foundation Trust and Institute for Child Health  London 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