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Pharmacokinetics of arginine butyrate in patients with hemoglobinopathy.

Abstract
Recent reports have demonstrated improvement in the clinical status and hemoglobin levels with use of intravenous arginine butyrate in patients with homozygous β-thalassemia and sickle cell disease. To allow optimalization of therapy, we conducted pharmacokinetic studies in nine patients, five with sickle cell disease and four with β-thalassemia, treated with continuous intravenous infusion of arginine butyrate. The disappearance of the drug after discontinuation was characterized by a biphasic elimination with an initial rapid phase followed by a slower phase. Redistribution was noted in five of the patients after 11.2 ± 4.0 min. The short half life was the result of both rapid clearance rate of 93.6 ± 31.9 ml/kg/min and small Vc (0.21 ± 0.26 l/kg) and Vss (0.31 ± 0.37 l/kg). While preliminary results of the effectiveness of arginine butyrate are encouraging with a rise of γ-globin mRNA and F reticulocytes in some patients, the rapid elimination of this agent will probably limit its current use to administration by continuous infusion.
AuthorsM Berkovitch, G Sher, R McCleland, D Matsui, G Hadzialic, N F Olivieri, G Koren
JournalEnvironmental toxicology and pharmacology (Environ Toxicol Pharmacol) Vol. 2 Issue 4 Pg. 403-5 (Dec 20 1996) ISSN: 1382-6689 [Print] Netherlands
PMID21781749 (Publication Type: Journal Article)

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