MSH
Trial question
What is the role of hydroxyurea in adult patients with sickle cell anemia?
Study design
Multi-center
Double blinded
RCT
Population
Characteristics of study participants
51.0% female
49.0% male
N = 299
299 patients (151 female, 148 male).
Inclusion criteria: adult patients with sickle cell anemia.
Key exclusion criteria: sickle cell-beta(+)-thalassemia and sickle cell-beta(0)-thalassemia, pregnancy, narcotic addiction, or regular consumption of > 30 oxycodone capsules (or the equivalent) every two weeks; indication of marrow depression; or prior hydroxyurea therapy;.
Interventions
N=152 hydroxyurea (initial dose of 15 mg/kg body weight and adjusted according to marrow depression, platelet count, and hemoglobin level).
N=147 placebo (Starch 1500, with the same adjustments of the dose).
Primary outcome
Painful crises
2.5%
4.5%
4.5 %
3.4 %
2.3 %
1.1 %
0.0 %
Hydroxyurea
Placebo
Significant
decrease ▼
NNT = 50
Significant decrease in painful crises (2.5% vs. 4.5%; RR 0.56, 95% CI 0.23 to 0.89).
Secondary outcomes
Significant decrease in chest syndrome (25 vs. 51; RR 0.49, 95% CI 0.2 to 0.78).
Significant decrease in underwent transfusions (48 vs. 73; RR 0.66, 95% CI 0.27 to 1.05).
Conclusion
In adult patients with sickle cell anemia, hydroxyurea was superior to placebo with respect to painful crises.
Reference
Charache S, Terrin ML, Moore RD et al. Effect of hydroxyurea on the frequency of painful crises in sickle cell anemia. Investigators of the Multicenter Study of Hydroxyurea in Sickle Cell Anemia. N Engl J Med. 1995 May 18;332(20):1317-22.
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