Hydroxyurea in ET
Trial question
What is the role of hydroxyurea in patients with essential thrombocythemia and high risk of thrombosis?
Study design
Multi-center
Open label
RCT
Population
Characteristics of study participants
68.0% female
32.0% male
N = 114
114 patients (77 female, 37 male).
Inclusion criteria: patients with essential thrombocythemia and a median platelet count of 788,000/mm³.
Key exclusion criteria: platelet count > 1,500,000/mm³.
Interventions
N=56 hydroxyurea (a starting dose of 15 mg/kg/day, followed by a maintenance dose to maintain platelet count < 600,000/mm³).
N=58 no myelosuppressive therapy (no hydroxyurea therapy).
Primary outcome
Thrombotic events
3.6%
24%
24.0 %
18.0 %
12.0 %
6.0 %
0.0 %
Hydroxyurea
No myelosuppressive
therapy
Significant
decrease ▼
NNT = 4
Significant decrease in thrombotic events (3.6% vs. 24%; ARD -20.4, 95% CI -32 to -8.5).
Safety outcomes
No significant difference in hemorrhage.
Conclusion
In patients with essential thrombocythemia and a median platelet count of 788,000/mm³, hydroxyurea was superior to no myelosuppressive therapy with respect to thrombotic events.
Reference
S Cortelazzo, G Finazzi, M Ruggeri et al. Hydroxyurea for patients with essential thrombocythemia and a high risk of thrombosis. N Engl J Med. 1995 Apr 27;332(17):1132-6.
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