Hydroxyurea for the treatment of sickle cell disease.

Jodi Segal, John Strouse, Mary Catherine Beach, Carlton Haywood, Catherine Witkop, Haeseong Park, Renee Wilson, Eric B Bass, Sophie Lanzkron

Research output: Contribution to journalArticle


OBJECTIVES: To synthesize the published literature on the efficacy, effectiveness, and toxicity of hydroxyurea (HU) when used for treatment of sickle cell disease (SCD); and to review the evidence regarding barriers to its use. DATA SOURCES: Articles cited in MEDLlNE, EMBASE, TOXLine, and CINAHL through June 30, 2007. REVIEW METHODS: Paired reviewers reviewed each title, abstract, and article to assess eligibility. They abstracted data sequentially and then independently graded the evidence. RESULTS: In one small, randomized trial of HU in children with SCD; the yearly hospitalization rate was lower with HU than placebo (1.1 versus 2.8, p=0.002). The absolute increase in fetal hemoglobin (Hb F%) was 10.7 percent. Twenty observational studies of HU in children reported similar increases in Hb F%, while hemoglobin concentration increased by roughly 1 g/dl. One large randomized trial tested the efficacy of HU in adults with SCD and found that after 2 years of treatment, Hb F% increased by 3.2 percent and hemoglobin increased by 0.6 g/dl, The median number of painful crises was 44 percent (p

Original languageEnglish (US)
Pages (from-to)1-95
Number of pages95
JournalEvidence report/technology assessment
Issue number165
Publication statusPublished - Mar 2008
Externally publishedYes


ASJC Scopus subject areas

  • Medicine(all)

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