2026-05-04T16:55:59Zhttps://repisalud.isciii.es/rest/oai/requestoai: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
Repisalud
author
Martín-Hernández, Elena
author
Quijada-Fraile, Pilar
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Correcher, Patricia
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Meavilla, Silvia
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Sánchez-Pintos, Paula
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de Las Heras Montero, Javier
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Blasco-Alonso, Javier
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Dougherty, Lucy
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Marquez, Ana
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Peña-Quintana, Luis
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Cañedo, Elvira
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García-Jimenez, María Concepción
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Moreno Lozano, Pedro Juan
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Murray Hurtado, Mercedes
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Camprodon Gómez, María
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Barrio-Carreras, Delia
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de Los Santos, Mariela
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Del Toro, Mireia
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Couce, María L
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Vitoria Miñana, Isidro
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Morales Conejo, Montserrat
author
Bellusci, Marcello
2024-02-27T15:10:28Z
2024-02-27T15:10:28Z
2022-08-28
10.3390/jcm11175045
2077-0383
Journal of clinical medicine
http://hdl.handle.net/10668/21339
36078975
http://hdl.handle.net/20.500.12105/18751
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
eng
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.
research article