Butyrate trials

被引：12

作者：

Cappellini, MD

Graziadei, G

Ciceri, L

Comino, A

Bianchi, P

Pomati, M

Fiorelli, G

机构：

[1] Univ Milan, Osped Maggiore Policlin, IRCCS,Ist Med Interna & Fisiopatol Med, Ctr Anemie Congenite, I-20122 Milan, Italy

[2] IRCCS, Osped Maggiore Policlin, Serv Ematol, Milan, Italy

来源：

COOLEYS ANEMIA: SEVENTH SYMPOSIUM | 1998年 / 850卷

关键词：

D O I：

10.1111/j.1749-6632.1998.tb10468.x

中图分类号：

Q3 [遗传学];

学科分类号：

071007 ; 090102 ;

摘要：

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).

引用

页码：110 / 119

页数：10

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