{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,18]],"date-time":"2026-09-18T10:45:04Z","timestamp":1789728304623,"version":"4.0.1"},"reference-count":35,"publisher":"MDPI AG","issue":"17","license":[{"start":{"date-parts":[[2022,8,28]],"date-time":"2022-08-28T00:00:00Z","timestamp":1661644800000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"name":"AECOM (Spanish Association for the Study of Inborn Errors of Metabolism)"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["JCM"],"abstract":"<jats:p>Background and objectives: Glycerol phenylbutyrate (GPB) has demonstrated safety and efficacy in patients with urea cycle disorders (UCDs) by means of its clinical trial program, but there are limited data in clinical practice. In order to analyze the efficacy and safety of GPB in clinical practice, here we present a national Spanish experience after direct switching from another nitrogen scavenger to GPB. Methods: This observational, retrospective, multicenter study was performed in 48 UCD patients (age 11.7 \u00b1 8.2 years) switching to GPB in 13 centers from nine Spanish regions. Clinical, biochemical, and nutritional data were collected at three different times: prior to GPB introduction, at first follow-up assessment, and after one year of GPB treatment. Number of related adverse effects and hyperammonemic crisis 12 months before and after GPB introduction were recorded. Results: GPB was administered at a 247.8 \u00b1 102.1 mg\/kg\/day dose, compared to 262.6 \u00b1 126.1 mg\/kg\/day of previous scavenger (46\/48 Na-phenylbutyrate). At first follow-up (79 \u00b1 59 days), a statistically significant reduction in ammonia (from 40.2 \u00b1 17.3 to 32.6 \u00b1 13.9 \u03bcmol\/L, p &lt; 0.001) and glutamine levels (from 791.4 \u00b1 289.8 to 648.6 \u00b1 247.41 \u03bcmol\/L, p &lt; 0.001) was observed. After one year of GPB treatment (411 \u00b1 92 days), we observed an improved metabolic control (maintenance of ammonia and glutamine reduction, with improved branched chain amino acids profile), and a reduction in hyperammonemic crisis rate (from 0.3 \u00b1 0.7 to less than 0.1 \u00b1 0.3 crisis\/patients\/year, p = 0.02) and related adverse effects (RAE, from 0.5 to less than 0.1 RAEs\/patients\/year p &lt; 0.001). Conclusions: This study demonstrates the safety of direct switching from other nitrogen scavengers to GPB in clinical practice, which improves efficacy, metabolic control, and RAE compared to previous treatments.<\/jats:p>","DOI":"10.3390\/jcm11175045","type":"journal-article","created":{"date-parts":[[2022,8,29]],"date-time":"2022-08-29T03:34:56Z","timestamp":1661744096000},"page":"5045","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":12,"title":["Switching to Glycerol Phenylbutyrate in 48 Patients with Urea Cycle Disorders: Clinical Experience in Spain"],"prefix":"10.3390","volume":"11","author":[{"given":"Elena","family":"Mart\u00edn-Hern\u00e1ndez","sequence":"first","affiliation":[{"name":"Centro de Referencia Nacional (CSUR) y Europeo (MetabERN) en Enfermedades Metab\u00f3licas, Hospital Universitario 12 de Octubre, Instituto de Investigaci\u00f3n i+12, CIBERER, 28041 Madrid, Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Pilar","family":"Quijada-Fraile","sequence":"additional","affiliation":[{"name":"Centro de Referencia Nacional (CSUR) y Europeo (MetabERN) en Enfermedades Metab\u00f3licas, Hospital Universitario 12 de Octubre, Instituto de Investigaci\u00f3n i+12, CIBERER, 28041 Madrid, Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9704-9534","authenticated-orcid":false,"given":"Patricia","family":"Correcher","sequence":"additional","affiliation":[{"name":"Centro de Referencia Nacional de Enfermedades Metab\u00f3licas (CSUR), Hospital La F\u00e9 de Valencia, 46026 Valencia, Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Silvia","family":"Meavilla","sequence":"additional","affiliation":[{"name":"Centro de Referencia Nacional (CSUR) y Europeo (MetabERN) de Enfermedades Metab\u00f3licas, Hospital San Joan de Deu Barcelona, 08950 Esplugues de Llobregat, Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-4992-1305","authenticated-orcid":false,"given":"Paula","family":"S\u00e1nchez-Pintos","sequence":"additional","affiliation":[{"name":"Centro de Referencia Nacional (CSUR) y Europeo (MetabERN) de Enfermedades Metab\u00f3licas, Hospital Cl\u00ednico Universitario de Santiago de Compostela, IDIS, CIBERER, 15706 Santiago de Compostela, Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-5663-537X","authenticated-orcid":false,"given":"Javier","family":"de las Heras Montero","sequence":"additional","affiliation":[{"name":"Division of Pediatric Metabolism, CIBERER, MetabERN, Cruces University Hospital, University of the Basque Country (UPV\/EHU) and Biocruces-Bizkaia Health Research Institute, 48903 Barakaldo, Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-4008-3747","authenticated-orcid":false,"given":"Javier","family":"Blasco-Alonso","sequence":"additional","affiliation":[{"name":"Secci\u00f3n de Gastroenterolog\u00eda y Nutrici\u00f3n Infantil, Unidad de Enfermedades Metab\u00f3licas Hereditarias, Grupo IBIMA Multidisciplinar Pedi\u00e1trico, Hospital Regional Universitario de M\u00e1laga, 29010 M\u00e1laga, Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-8865-2438","authenticated-orcid":false,"given":"Lucy","family":"Dougherty","sequence":"additional","affiliation":[{"name":"Centro de Referencia Nacional (CSUR) y Europeo (MetabERN) de Enfermedades Metab\u00f3licas, Hospital Vall D\u2019Hebr\u00f3n, 08035 Barcelona, Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ana","family":"Marquez","sequence":"additional","affiliation":[{"name":"Unidad de Gastroenterolog\u00eda y Enfermedades Metab\u00f3licas, Hospital de Badajoz, 06002 Badajoz, Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6052-5894","authenticated-orcid":false,"given":"Luis","family":"Pe\u00f1a-Quintana","sequence":"additional","affiliation":[{"name":"Unidad de Gastroenterolog\u00eda y Nutrici\u00f3n Pedi\u00e1trica, Complejo Hospitalario Universitario Insular Materno-Infantil de Las Palmas, CIBEROBN, ISCIII, ACIP, Universidad de Las Palmas de Gran Canaria, 35016 Las Palmas de Gran Canaria, Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-6610-5644","authenticated-orcid":false,"given":"Elvira","family":"Ca\u00f1edo","sequence":"additional","affiliation":[{"name":"Unidad de Gastroenterolog\u00eda y Nutrici\u00f3n, Hospital del Ni\u00f1o Jes\u00fas, 28009 Madrid, Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mar\u00eda Concepci\u00f3n","family":"Garc\u00eda-Jimenez","sequence":"additional","affiliation":[{"name":"Unidad de Metabolismo, Hospital Miguel Servet, 50009 Zaragoza, Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-5501-5272","authenticated-orcid":false,"given":"Pedro Juan","family":"Moreno Lozano","sequence":"additional","affiliation":[{"name":"Unidad de Enfermedades Musculares y Metab\u00f3licas Hereditarias, Departamento de Medicina Interna, Hospital Clinic, 08036 Barcelona, Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mercedes","family":"Murray Hurtado","sequence":"additional","affiliation":[{"name":"Pediatr\u00eda, Secci\u00f3n de Nutrici\u00f3n y Errores Innatos del Metabolismo, Complejo Hospitalario Universitario de Canarias, 38320 San Crist\u00f3bal de La Laguna, Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mar\u00eda","family":"Camprodon G\u00f3mez","sequence":"additional","affiliation":[{"name":"Centro de Referencia Nacional (CSUR) y Europeo (MetabERN) de Enfermedades Metab\u00f3licas, Hospital Vall D\u2019Hebr\u00f3n, 08035 Barcelona, Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0171-3830","authenticated-orcid":false,"given":"Delia","family":"Barrio-Carreras","sequence":"additional","affiliation":[{"name":"Centro de Referencia Nacional (CSUR) y Europeo (MetabERN) en Enfermedades Metab\u00f3licas, Hospital Universitario 12 de Octubre, Instituto de Investigaci\u00f3n i+12, CIBERER, 28041 Madrid, Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mariela","family":"de los Santos","sequence":"additional","affiliation":[{"name":"Centro de Referencia Nacional (CSUR) y Europeo (MetabERN) de Enfermedades Metab\u00f3licas, Hospital San Joan de Deu Barcelona, 08950 Esplugues de Llobregat, Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mireia","family":"del Toro","sequence":"additional","affiliation":[{"name":"Centro de Referencia Nacional (CSUR) y Europeo (MetabERN) de Enfermedades Metab\u00f3licas, Hospital Vall D\u2019Hebr\u00f3n, 08035 Barcelona, Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-4861-9905","authenticated-orcid":false,"given":"Mar\u00eda L.","family":"Couce","sequence":"additional","affiliation":[{"name":"Centro de Referencia Nacional (CSUR) y Europeo (MetabERN) de Enfermedades Metab\u00f3licas, Hospital Cl\u00ednico Universitario de Santiago de Compostela, IDIS, CIBERER, 15706 Santiago de Compostela, Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-4450-1213","authenticated-orcid":false,"given":"Isidro","family":"Vitoria Mi\u00f1ana","sequence":"additional","affiliation":[{"name":"Centro de Referencia Nacional de Enfermedades Metab\u00f3licas (CSUR), Hospital La F\u00e9 de Valencia, 46026 Valencia, Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Montserrat","family":"Morales Conejo","sequence":"additional","affiliation":[{"name":"Centro de Referencia Nacional (CSUR) y Europeo (MetabERN) en Enfermedades Metab\u00f3licas, Hospital Universitario 12 de Octubre, Instituto de Investigaci\u00f3n i+12, CIBERER, 28041 Madrid, Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marcello","family":"Bellusci","sequence":"additional","affiliation":[{"name":"Centro de Referencia Nacional (CSUR) y Europeo (MetabERN) en Enfermedades Metab\u00f3licas, Hospital Universitario 12 de Octubre, Instituto de Investigaci\u00f3n i+12, CIBERER, 28041 Madrid, Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1968","published-online":{"date-parts":[[2022,8,28]]},"reference":[{"key":"ref_1","unstructured":"Pagon, R.A., Adam, M.P., Ardinger, H.H., Wallace, S.E., 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