201. Heme arginate therapy for beta thalassemia: in vitro versus in vivo effects.
- Author
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Rund D, Fibach E, Goldfarb A, Friedberg A, and Rachmilewitz E
- Subjects
- Adult, Arginine therapeutic use, Cells, Cultured, Female, Heme therapeutic use, Humans, Infusions, Intravenous, Male, Treatment Outcome, beta-Thalassemia pathology, Arginine administration & dosage, Arginine pharmacology, Heme administration & dosage, Heme pharmacology, beta-Thalassemia drug therapy
- Abstract
Hemin has a profound effect on erythroid cell maturation and promotes fetal hemoglobin synthesis in vitro. In beta-thalassemia, increasing fetal hemoglobin levels can ameliorate the anemia. We administered heme arginate, a novel stable form of hemin, to 4 patients with thalassemia intermedia and studied the in vitro versus in vivo effects. In erythroid cultures, there was a marked rise in total hemoglobin and hemoglobin F. In vivo, 3 of 4 patients had a rise in hemoglobin levels (from 0.4 to 1.1 g%), which was statistically significant in 1 patient. There were no serious adverse effects. Heme arginate may be useful in the treatment of thalassemia intermedia.
- Published
- 1998
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