Chemotherapy to increase fetal hemoglobin in patients with sickle cell anemia.
The obvious beneficial effects of hemoglobin F on sickling have motivated numerous investigators to increase this type of hemoglobin artificially in patients with sickle cell anemia. Various chemotherapeutic agents including 5-azacytidine, hydroxyurea, and cytosine arabinoside, have been used successfully in patients. All of these drugs can increase the level of hemoglobin F in sickle cell anemia (SS) patients, but the kinetics and magnitude of the responses are highly individual and variable. The mechanism or mechanisms responsible for the increased synthesis of hemoglobin F remain unknown. Further controlled studies in a limited number of patients with severe sickle cell disease will be necessary in order to work out a rational, safe treatment program suitable for wider use.
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D000755:Anemia, Sickle Cell / Q000097:blood / Q000188:drug therapy*; D001374:Azacitidine / Q000627:therapeutic use; D003561:Cytarabine / Q000627:therapeutic use; D005319:Fetal Hemoglobin / Q000096:biosynthesis*; D006801:Humans; D006918:Hydroxyurea / Q000627:therapeutic use; D007700:Kinetics; D013789:Thalassemia / Q000097:blood / Q000188:drug therapy
D003561:Cytarabine; D005319:Fetal Hemoglobin; D001374:Azacitidine; D006918:Hydroxyurea