Butyrate Trialsa

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-α/α globin chain ratio and percentage of HbF. Five patients experienced increases of non-α/α 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).