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  • Title: Butyrate trials.
    Author: Cappellini MD, Graziadei G, Ciceri L, Comino A, Bianchi P, Pomati M, Fiorelli G.
    Journal: Ann N Y Acad Sci; 1998 Jun 30; 850():110-9. PubMed ID: 9668533.
    Abstract:
    The aims of this study were to ascertain tolerability, safety and efficacy of oral isobutyramide (150 mg/kg bw/day) in stimulating fetal hemoglobin production in twelve thalassemia intermedia patients. Patients were treated for 28 days and followed for a further 28 days. Efficacy was monitored by non-alpha/alpha globin chain ratio and percentage of HbF. Five patients experienced increases of non-alpha/alpha ratio ranging between 5.3 and 100% at the end of treatment. Five patients show an increase of HbF ranging between 4.4 and 26%. Their HbF% continues to increase during follow-up period. The analysis of variance for HbF showed a time effect close to significance both in treatment period (p = 0.06) and in follow-up period (p = 0.08). Moreover, to evaluate a possible erythropoietic modification, serum Erythropoietin (sEpo) and serum Transferrin Receptor (sTfR) were evaluated. Serum Epo and sTfR levels were significantly increased during treatment (p < 0.05 vs baseline).
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