2010
DOI: 10.1002/hep.23589
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Pharmacology and Safety of Glycerol Phenylbutyrate in Healthy Adults and Adults With Cirrhosis

Abstract: Phenylbutyric acid (PBA), which is approved for treatment of urea cycle disorders (UCDs) as sodium phenylbutyrate (NaPBA), mediates waste nitrogen excretion via combination of PBA-derived phenylacetic acid with glutamine to form phenylactylglutamine (PAGN) that is excreted in urine. Glycerol phenylbutyrate (GPB), a liquid triglyceride pro-drug of PBA, containing no sodium and having favorable palatability, is being studied for treatment of hepatic encephalopathy (HE). In vitro and clinical studies have been pe… Show more

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Cited by 65 publications
(63 citation statements)
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“…GPB has the same mechanism of action as sodium PB, but is a pre-pro drug consisting of phenylbutyric acid (PBA) linked to glycerol. Following hydrolysis by pancreatic lipases, PBA is released and converted to the active moiety, phenylacetic acid (Lee et al 2010;McGuire et al 2010;Monteleone et al 2013). In clinical trials, GPB has been shown to provide effective ammonia control in adult and pediatric UCD patients (Lichter-Konecki et al 2011;Smith et al 2013;Diaz et al 2013;Berry et al 2014).…”
Section: Introductionmentioning
confidence: 99%
“…GPB has the same mechanism of action as sodium PB, but is a pre-pro drug consisting of phenylbutyric acid (PBA) linked to glycerol. Following hydrolysis by pancreatic lipases, PBA is released and converted to the active moiety, phenylacetic acid (Lee et al 2010;McGuire et al 2010;Monteleone et al 2013). In clinical trials, GPB has been shown to provide effective ammonia control in adult and pediatric UCD patients (Lichter-Konecki et al 2011;Smith et al 2013;Diaz et al 2013;Berry et al 2014).…”
Section: Introductionmentioning
confidence: 99%
“…Patients had significantly improved mental status and cognitive function, along with a reduced length of hospitalization compared to those receiving placebo [21,47,48]. McGuire et al [52] recently studied the effects of glycerol phenylbutyrate (GPB), the prodrug of sodium phenylbutyric acid (NaPBA), which is used in the treatment of urea cycle disorders by mediating excretion of waste nitrogen. It was shown that GPB resulted in prolonged urinary excretion of phenylacetylglutamine (PAGN), with cirrhotic patients excreting the same amount of PAGN as controls.…”
Section: Treatmentmentioning
confidence: 98%
“…Studies have also shown a beneficial effect on lowering blood ammonia and HE events by administering the glutamine scavenger phenylacetate (as glycerol phenylbutyrate, pro-drug for phenylacetate) (McGuire et al 2010;Rockey et al 2014).…”
Section: Glutaminementioning
confidence: 98%