title: Isobutyramide therapy in patients with sickle cell anemia.
abstract: We have administered Isobutyramide as a suspension over a period of 3 months, from a starting dose of 50 mg/kg/day up to 150 mg/kg/day, to four adult sickle cell (SS) anemia patients. The maximum dose was maintained for 3 weeks. The blood counts remained stable and the Hb F levels decreased slightly. The G gamma levels increased at the end of the trial, suggesting activation of the G gamma gene at the highest dose of Isobutyramide. Three patients showed a stable rate of hemolysis, while in one patient, an increase of lactate dehydrogenase occurred. None of the patients experienced pain crisis or organ-specific crisis, but all four complained about mild epigastric burning and a bitter taste. After the first month of treatment one patient complained about intolerable epigastric discomfort which was relieved by Omeprazole. Another patient complained about increasing dyspepsia in the 12th week leading to the termination of the trial. Oral Isobutyramide administration does not qualify as an effective treatment of SS patients.
authors.affiliation: Department of Internal Medicine, St. Elizabeth Hospital, Curaçao, The Netherlands Antilles.
authors.forename: A W
authors.initials: AW
authors.lastname: Saleh
authors.forename: A
authors.initials: A
authors.lastname: van Goethem
authors.forename: R
authors.initials: R
authors.lastname: Jansen
authors.forename: H J
authors.initials: HJ
authors.lastname: Velvis
authors.forename: L H
authors.initials: LH
authors.lastname: Gu
authors.forename: T H
authors.initials: TH
authors.lastname: Huisman
mesh_terms: D000328:Adult; D000577:Amides / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D000755:Anemia, Sickle Cell / Q000188:drug therapy*; D005260:Female; D005319:Fetal Hemoglobin / Q000378:metabolism; D005914:Globins / Q000235:genetics / Q000378:metabolism; D006801:Humans; D006918:Hydroxyurea / Q000627:therapeutic use; D007770:L-Lactate Dehydrogenase / Q000097:blood; D008297:Male; D008875:Middle Aged
chemical_list: D000577:Amides; C030197:isobutyramide; D005914:Globins; D005319:Fetal Hemoglobin; D007770:L-Lactate Dehydrogenase; D006918:Hydroxyurea