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Correspondence: Hassana Fathallah, PhD, Assistant Professor; Phone 212-241-7931; Fax 212-426-4390; Email hassana.fathallah{at}mssm.edu, George F Atweh, MD, Professor; Phone 212-241-8109; Fax 212-369-8375; Email george.atweh{at}mssm.edu
Abstract
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.
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