Heme arginate therapy for beta thalassemia: in vitro versus in vivo effects.
Hemin has a profound effect on erythroid cell maturation and promotes fetal hemoglobin synthesis in vitro. In beta-thalassemia, increasing fetal hemoglobin levels can ameliorate the anemia. We administered heme arginate, a novel stable form of hemin, to 4 patients with thalassemia intermedia and studied the in vitro versus in vivo effects. In erythroid cultures, there was a marked rise in total hemoglobin and hemoglobin F. In vivo, 3 of 4 patients had a rise in hemoglobin levels (from 0.4 to 1.1 g%), which was statistically significant in 1 patient. There were no serious adverse effects. Heme arginate may be useful in the treatment of thalassemia intermedia.
Rund
D
D
Hematology Department, Hadassah University Hospital, Ein Kerem, Jerusalem, Israel. rund@cc.huji.ac.il
Fibach
E
E
Goldfarb
A
A
Friedberg
A
A
Rachmilewitz
E
E
D000328:Adult; D001120:Arginine / Q000008:administration & dosage* / Q000494:pharmacology* / Q000627:therapeutic use; D002478:Cells, Cultured; D005260:Female; D006418:Heme / Q000008:administration & dosage* / Q000494:pharmacology* / Q000627:therapeutic use; D006801:Humans; D007262:Infusions, Intravenous; D008297:Male; D016896:Treatment Outcome; D017086:beta-Thalassemia / Q000188:drug therapy* / Q000473:pathology
D006418:Heme; D001120:Arginine; C048849:heme arginate