Background Abnormalities in the number and function of plateletsmay contribute to thromboembolic complications in patients withessential thrombocythemia. We assessed whether maintaining theplatelet count below 600,000 per cubic millimeter with hydroxyureareduces the incidence of thrombosis in patients with essentialthrombocythemia and a high risk of thrombosis.
Methods A total of 114 patients with essential thrombocythemia(77 women and 37 men; median age, 68 years; range, 40 to 85)and a median platelet count of 788,000 per cubic millimeter(range, 533,000 to 1,240,000 per cubic millimeter) were randomlyassigned to receive hydroxyurea (56 patients) or no myelosuppressivetherapy (58 patients). Ninety-seven (85 percent) were over 60years old, and 52 (46 percent) had had thrombosis. The two groupswere matched for age, sex, and platelet count at randomization.Antiplatelet prophylaxis with aspirin or ticlopidine was notstopped. Follow-up lasted a median of 27 months in both groups.
Results Two patients (3.6 percent) treated with hydroxyureahad thrombotic episodes (one stroke and one myocardial infarction),whereas 14 patients (24 percent) in the control group had thromboticepisodes (one stroke, five transient ischemic attacks, fiveperipheral arterial occlusions, one deep-vein thrombosis, andtwo cases of superficial thrombophlebitis). The difference (20.4percentage points; 95 percent confidence interval, 8.5 to 32percent) was statistically significant (chi-square with Yates'correction, 8.3; 1 df; P = 0.003).
Conclusions Hydroxyurea is effective in preventing thrombosisin high-risk patients with essential thrombocythemia.
Source Information
From the Division of Hematology, Ospedali Riuniti di Bergamo, Bergamo (S.C., G.F., O.V., M.G., T.B.), and the Division of Hematology, Ospedale Civile S. Bortolo di Vicenza, Vicenza (M.R., F.R.) both in Italy.
Address reprint requests to Prof. Barbui at the Divisione di Ematologia, Ospedali Riuniti di Bergamo, Largo Barozzi 1, 24100 Bergamo, Italy.
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