TY - JOUR
T1 - Butyrate trials
AU - Cappellini, Maria Domenica
AU - Graziadei, Giovanna
AU - Ciceri, Laura
AU - Comino, Alessia
AU - Bianchi, Paolo
AU - Pomati, Mauro
AU - Fiorelli, Gemino
PY - 1998
Y1 - 1998
N2 - 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).
AB - 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).
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U2 - 10.1111/j.1749-6632.1998.tb10468.x
DO - 10.1111/j.1749-6632.1998.tb10468.x
M3 - Article
C2 - 9668533
AN - SCOPUS:0031850654
VL - 850
SP - 110
EP - 119
JO - Annals of the New York Academy of Sciences
JF - Annals of the New York Academy of Sciences
SN - 0077-8923
ER -