Article
Oral isobutyramide therapy in patients with thalassemia intermedia: results of a phase II open study.
Blood cells, molecules & diseases - 1 Feb 2000
Domenica Cappellini M, Graziadei G, Ciceri L, Comino A, Bianchi P, Porcella A, Fiorelli G
Abstract excerpt
A pilot phase II open study on 12 patients with thalassemia intermedia (7 men, 5 women; age 31 +/- 2.0 years SE) treated with oral isobutyramide, a derivative of butyric acid (150 mg/kg body wt/day), was performed in order to evaluate the effect of this compound in stimulating hemoglobin F (HbF) production. No patient underwent blood transfusion in the 1-year time frame prior to the study. Nine patients were...
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