2026-05-04T16:55:59Zhttps://repisalud.isciii.es/rest/oai/request

oai:repisalud.isciii.es:20.500.12105/187512024-02-27T15:10:28Zcom_20.500.12105_15322com_20.500.12105_2051col_20.500.12105_16938col_20.500.12105_16927col_20.500.12105_16966col_20.500.12105_16976

00925njm 22002777a 4500 dc Martín-Hernández, Elena author Quijada-Fraile, Pilar author Correcher, Patricia author Meavilla, Silvia author Sánchez-Pintos, Paula author de Las Heras Montero, Javier author Blasco-Alonso, Javier author Dougherty, Lucy author Marquez, Ana author Peña-Quintana, Luis author Cañedo, Elvira author García-Jimenez, María Concepción author Moreno Lozano, Pedro Juan author Murray Hurtado, Mercedes author Camprodon Gómez, María author Barrio-Carreras, Delia author de Los Santos, Mariela author Del Toro, Mireia author Couce, María L author Vitoria Miñana, Isidro author Morales Conejo, Montserrat author Bellusci, Marcello author 2022-08-28 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 ± 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 ± 102.1 mg/kg/day dose, compared to 262.6 ± 126.1 mg/kg/day of previous scavenger (46/48 Na-phenylbutyrate). At first follow-up (79 ± 59 days), a statistically significant reduction in ammonia (from 40.2 ± 17.3 to 32.6 ± 13.9 μmol/L, p 10.3390/jcm11175045 2077-0383 Journal of clinical medicine http://hdl.handle.net/10668/21339 36078975 http://hdl.handle.net/20.500.12105/18751 clinical practice glycerol phenylbutyrate (GPB) sodium benzoate (NaBZ) sodium phenylbutyrate (NaPB) urea cycle disorders (UCDs) Switching to Glycerol Phenylbutyrate in 48 Patients with Urea Cycle Disorders: Clinical Experience in Spain.