Potential therapeutics forγ-globin gene induction in the β-thalassemia syndromes.
Patient Characteristics . | Therapeutic Classes . |
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Abbreviations: EPO, erythropoietin; HDACi, histone deacetylase inhibitor | |
Thalassemia intermedia Low HbF (< 30%) and Low EPO (< 100 mU/mL) Total Hb > 5 g/dL | • EPO • Fetal globin gene activator |
Thalassemia intermedia/major High HbF (> 60%) and High EPO (> 160 mU/mL) Total Hb > 4–5 g/dL | • Fetal globin gene activator or • Hydroxyurea |
Thalassemia major Total Hb > 4–5 g/dL | • EPO • Fetal globin gene activator • Hypomethylating agent/HDACi (?) |
Patient Characteristics . | Therapeutic Classes . |
---|---|
Abbreviations: EPO, erythropoietin; HDACi, histone deacetylase inhibitor | |
Thalassemia intermedia Low HbF (< 30%) and Low EPO (< 100 mU/mL) Total Hb > 5 g/dL | • EPO • Fetal globin gene activator |
Thalassemia intermedia/major High HbF (> 60%) and High EPO (> 160 mU/mL) Total Hb > 4–5 g/dL | • Fetal globin gene activator or • Hydroxyurea |
Thalassemia major Total Hb > 4–5 g/dL | • EPO • Fetal globin gene activator • Hypomethylating agent/HDACi (?) |