Please use this identifier to cite or link to this item:http://hdl.handle.net/20.500.12105/18751
Title
Switching to Glycerol Phenylbutyrate in 48 Patients with Urea Cycle Disorders: Clinical Experience in Spain.
Author(s)
Martín-Hernández, Elena | Quijada-Fraile, Pilar | Correcher, Patricia | Meavilla, Silvia | Sánchez-Pintos, Paula | de Las Heras Montero, Javier | Blasco-Alonso, Javier | Dougherty, Lucy | Marquez, Ana | Peña-Quintana, Luis | Cañedo, Elvira | García-Jimenez, María Concepción | Moreno Lozano, Pedro Juan | Murray Hurtado, Mercedes | Camprodon Gómez, María | Barrio-Carreras, Delia | de Los Santos, Mariela | Del Toro, Mireia | Couce, María L | Vitoria Miñana, Isidro | Morales Conejo, Montserrat | Bellusci, Marcello
Date issued
2022-08-28
Language
Inglés
Document type
research article
Abstract
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
Subject
clinical practice | glycerol phenylbutyrate (GPB) | sodium benzoate (NaBZ) | sodium phenylbutyrate (NaPB) | urea cycle disorders (UCDs)
DOI
Collections
- Investigación > IIS > IIS BioBizkaia - Asociación Instituto de Investigación Sanitaria BioBizkaia (País Vasco)
- Investigación > IIS > IBIMA-Plataforma BIONAND - Instituto de Investigación Biomédica de Málaga y Plataforma en Nanomedicina (Andalucía)
- Investigación > IIS > i+12 - Instituto de Investigación Hospital 12 de Octubre (Madrid)
- Investigación > IIS > IDIS - Instituto de Investigación Sanitaria de Santiago de Compostela (Galicia)
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