2013
DOI: 10.1016/j.ymgme.2013.09.017
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Elevated phenylacetic acid levels do not correlate with adverse events in patients with urea cycle disorders or hepatic encephalopathy and can be predicted based on the plasma PAA to PAGN ratio

Abstract: Background Phenylacetic acid (PAA) is the active moiety in sodium phenylbutyrate (NaPBA) and glycerol phenylbutyrate (GPB, HPN-100), both are approved for treatment of urea cycle disorders (UCDs) - rare genetic disorders characterized by hyperammonemia. PAA is conjugated with glutamine in the liver to form phenylacetyleglutamine (PAGN), which is excreted in urine. PAA plasma levels ≥500 μg/dL have been reported to be associated with reversible neurological adverse events (AEs) in cancer patients receiving PAA … Show more

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Cited by 32 publications
(27 citation statements)
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“…The formulation of glycerol PBA may have improved palatability and pharmacological properties (avoids sodium intake and is a tasteless liquid). The clinical trials on this product have shown equal effectiveness with sodium phenylbutyrate with no significant difference in adherence to either of two preparations. In terms of cost, glycerol PBA is still more expensive.…”
Section: New Developmentsmentioning
confidence: 96%
“…The formulation of glycerol PBA may have improved palatability and pharmacological properties (avoids sodium intake and is a tasteless liquid). The clinical trials on this product have shown equal effectiveness with sodium phenylbutyrate with no significant difference in adherence to either of two preparations. In terms of cost, glycerol PBA is still more expensive.…”
Section: New Developmentsmentioning
confidence: 96%
“…; Mokhtarani et al . ). Therefore, PB is accepted as safe and well‐tolerated agent with potential of clinical use in treatment of lethal illnesses that do not have any effective treatment (Iannitti & Palmieri ).…”
mentioning
confidence: 97%
“…First, the relative risk and frequency of recurrent HE episodes and HE hospitalizations increased significantly with fasting NH 3 levels >1.5 Â ULN. Second, although GPB metabolites phenylbutyrate and phenylacetate reportedly have biological effects other than removal of waste nitrogen, 9 the consistency of the relationship between NH 3 and HE episodes and hospitalizations in both treatment arms suggests that the observed GPB treatment effect is mediated by lowering of NH 3 . Note the disproportionately higher probability among patients whose baseline ammonia exceeded 1.5 Â ULN, which was approximately double that in patients with lower ammonia levels.…”
Section: Discussionmentioning
confidence: 97%