Article
Oral sodium phenylbutyrate therapy in homozygous beta thalassemia: a clinical trial.
Blood - 1 Jan 1995
Collins A F, Pearson H A, Giardina P, McDonagh K T, Brusilow S W, Dover G J
Abstract excerpt
Butyrate analogues have been shown to increase fetal hemoglobin (HbF) production in vitro and in vivo. Sodium phenylbutyrate (SPB), an oral agent used to treat individuals with urea-cycle disorders, has been shown to increase HbF in nonanemic individuals and in individuals with sickle cell disease. We have treated eleven patients with homozygous beta thalassemia (three transfusion dependent) and one...
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