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The role of hydroxyurea in sickle cell disease
Author(s) -
Halsey Christina,
Roberts Irene A. G.
Publication year - 2003
Publication title -
british journal of haematology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.907
H-Index - 186
eISSN - 1365-2141
pISSN - 0007-1048
DOI - 10.1046/j.1365-2141.2003.03849.x
Subject(s) - medicine , hydroxycarbamide , disease , acute chest syndrome , clinical trial , intensive care medicine , sickle cell anemia , pediatrics
Although the molecular basis for the sickling disorders was identified more than 50 years ago (reviewed in Weatherall 2001), progress towards definitive therapy for sickle cell disease (SCD) has been frustratingly slow. Until the mid1990s, treatment was almost entirely supportive with no clinically useful drugs available to prevent or reverse the polymerization of HbS. However, a number of key observations about the role of HbF (Stevens et al, 1981; de Simone et al, 1982, Platt et al, 1984, 1991; Veith et al, 1985; Noguchi et al, 1993) led, in 1995, to the publication of a crucially important study in which a new therapeutic agent, hydroxyurea, was heralded as a major breakthrough in the management of SCD (Charache et al, 1995; Schechter & Rodgers, 1995). The initial clinical trials of hydroxyurea in SCD were stopped early as a result of outstanding results in the treatment arm and the drug was rapidly licensed in the USA for the treatment of patients with severe SCD. Since then, hydroxyurea has been used successfully in a variety of situations in SCD, including new studies in very young children (Wang et al, 2001). However, doubts remain both about its efficacy and safety in the long term. This review aims to outline current information about the mechanisms of action of hydroxyurea in SCD, its clinical efficacy in sickling disorders, the approaches to monitoring treatment and to update the evidence about its short-term and long-term toxicity.