Article
Oral isobutyramide reduces transfusion requirements in some patients with homozygous beta-thalassemia.
Blood - 15 Nov 2000
Reich S, Bührer C, Henze G, Ohlendorf D, Mesche M, Sinha P, Kage A, Müller C, Vetter B, Kulozik A E
Abstract excerpt
The butyrate derivative isobutyramide (IBT) increases fetal hemoglobin (HbF) in patients with beta-hemoglobinopathies, but little is known about its usefulness for prolonged therapeutic use. We treated 8 patients with transfusion-dependent beta-thalassemia with 350 mg/kg of body weight per day of oral IBT for 126 to 384 days. During the trial period, the hemoglobin level was maintained between 85 g/L (range 82-87...
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