Induction of fetal hemoglobin in the treatment of sickle cell disease.
Reactivation of fetal hemoglobin (HbF) expression is an important therapeutic option in patients with hemoglobin disorders. In sickle cell disease (SCD), an increase in HbF inhibits the polymerization of sickle hemoglobin and the resulting pathophysiology. Hydroxyurea, an inducer of HbF, has already been approved for the treatment of patients with moderate and/or severe SCD. Recent clinical trials with other pharmacological inducers of HbF, such as butyrate and decitabine, have shown considerable promise. In this chapter, we highlight the important clinical trials with pharmacological inducers of HbF, discuss their mechanisms of action and speculate about the future of this therapeutic approach in the treatment of patients with SCD.
Fathallah
Hassana
H
Mount Sinai School of Medicine, Division of Hematology and Oncology, Box 1079, One Gustave L. Levy Place, New York, NY 10029, USA. hassana.fathallah@mssm.edu
Atweh
George F
GF
D000755:Anemia, Sickle Cell / Q000188:drug therapy*; D001374:Azacitidine / Q000031:analogs & derivatives / Q000627:therapeutic use; D002087:Butyrates / Q000627:therapeutic use; D000077209:Decitabine; D005319:Fetal Hemoglobin / Q000235:genetics*; D005786:Gene Expression Regulation / Q000187:drug effects; D006801:Humans; D006918:Hydroxyurea / Q000627:therapeutic use
D002087:Butyrates; D000077209:Decitabine; D005319:Fetal Hemoglobin; D001374:Azacitidine; D006918:Hydroxyurea