Pharmacological modification of hemoglobin F expression in sickle cell anemia: an update on hydroxyurea studies.
The most studied pharmacological intervention in sickle cell anemia aiming at elevating HbF expression is the use of hydroxyurea. At the present time the experience has been that after 1 year of treatment with maximum tolerated doses (MTD) all patients showed increases of percent HbF, with a mean of 15% HbF, without apparent side effects besides the reversible ones observed during the process of attaining the MTD. The question of efficacy is presently being investigated by a multicenter placebo controlled double blind clinical trial that involves more than 20 sites. The goal of the study is to determine if hydroxyurea can decrease the incidence of painful crises by 50%. Results of this study are not expected before the end of 1993.
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Hematology Laboratories, Johns Hopkins University, School of Medicine, Baltimore, Maryland 21205.
D000755:Anemia, Sickle Cell / Q000188:drug therapy*; D000818:Animals; D001374:Azacitidine / Q000494:pharmacology; D004912:Erythrocytes / Q000378:metabolism; D005319:Fetal Hemoglobin / Q000502:physiology*; D015870:Gene Expression / Q000187:drug effects; D006801:Humans; D006918:Hydroxyurea / Q000009:adverse effects / Q000493:pharmacokinetics / Q000627:therapeutic use*
D005319:Fetal Hemoglobin; D001374:Azacitidine; D006918:Hydroxyurea