Butyrate trials.
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).
Cappellini
M D
MD
Centro Anemie Congenite, Ospedale Maggiore Policlinico IRCCS, Università di Milano, Italy. Maria.Cappellini@unimi.it
Graziadei
G
G
Ciceri
L
L
Comino
A
A
Bianchi
P
P
Pomati
M
M
Fiorelli
G
G
D000328:Adult; D000577:Amides / Q000009:adverse effects / Q000627:therapeutic use*; D001803:Blood Transfusion; D004921:Erythropoietin / Q000097:blood; D005260:Female; D005319:Fetal Hemoglobin / Q000096:biosynthesis* / Q000235:genetics; D005914:Globins / Q000032:analysis / Q000096:biosynthesis*; D006801:Humans; D008297:Male; D011990:Receptors, Transferrin / Q000097:blood; D017086:beta-Thalassemia / Q000097:blood / Q000188:drug therapy*
D000577:Amides; D011990:Receptors, Transferrin; D004921:Erythropoietin; C030197:isobutyramide; D005914:Globins; D005319:Fetal Hemoglobin