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Butyrate trials

M D Cappellini1, G Graziadei, L Ciceri

  • 1Centro Anemie Congenite, Ospedale Maggiore Policlinico IRCCS, Università di Milano, Italy. Maria.Cappellini@unimi.it

Annals of the New York Academy of Sciences
|July 21, 1998
PubMed
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Oral isobutyramide shows promise in increasing fetal hemoglobin (HbF) for thalassemia intermedia patients. This study found that isobutyramide stimulated HbF production and increased erythropoietic markers over 28 days.

Area of Science:

  • Hematology
  • Pharmacology
  • Genetics

Background:

  • Thalassemia intermedia is a severe inherited blood disorder.
  • Current treatments for thalassemia intermedia have limitations.
  • Stimulating fetal hemoglobin (HbF) production is a therapeutic strategy.

Purpose of the Study:

  • To evaluate the tolerability, safety, and efficacy of oral isobutyramide in stimulating fetal hemoglobin production.
  • To assess the impact of isobutyramide on erythropoietic markers in thalassemia intermedia patients.

Main Methods:

  • A 28-day open-label study involving twelve thalassemia intermedia patients.
  • Isobutyramide administered orally at 150 mg/kg body weight/day.
  • Efficacy assessed by non-alpha/alpha globin chain ratio, HbF percentage, serum erythropoietin (sEpo), and serum transferrin receptor (sTfR).

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Main Results:

  • Five patients showed increased non-alpha/alpha globin chain ratio (5.3-100%) and HbF percentage (4.4-26%) by end of treatment.
  • HbF levels continued to rise during the 28-day follow-up period.
  • Serum Epo and sTfR levels significantly increased during treatment (p < 0.05), indicating erythropoietic stimulation.

Conclusions:

  • Oral isobutyramide appears to be tolerable and safe for short-term use in thalassemia intermedia.
  • Isobutyramide effectively stimulates fetal hemoglobin production and erythropoiesis in these patients.
  • Further research is warranted to explore long-term efficacy and safety.