Decreased transfusion needs associated with hydroxyurea therapy in Algerian patients with thalassemia major or intermedia.

BACKGROUND: Studies of evolution of transfusion requirements in thalassemic patients treated with hydroxyurea have produced somewhat conflicting results, especially in patients with thalassemia major. Our aims were to determine the proportion of good responders to hydroxyurea in a population of transfusion-dependent thalassemic patients and to identify the factors associated with a decrease of transfusion needs. STUDY DESIGN AND METHODS: Hydroxyurea treatment was initiated in 9 patients with thalassemia intermedia (TI) and 45 with thalassemia major (TM). Patients received transfusions when their hemoglobin (Hb) levels dropped below 6 g per dL. A decrease in annual transfusion requirements greater than 70 percent defined a good response, between 40 and 70 percent a partial response, and smaller than 40 percent no response.
RESULTS: The response was good in 8 (90%) patients with TI and 20 (44.5%) with TM, partial in 9 (20%) patients with TM, and absent in 1 (10%) with TI and 16 (35.5%) with TM. In TM patients, transfusion needs decreased by 56 percent over the first year of hydroxyurea treatment. By univariate analysis, a better response to hydroxyurea was associated with older age at the first transfusion (p = 0.02), higher prehydroxyurea Hb (p = 0.0004), codon 6(-A) mutation (p = 0.002), TI (p = 0.03), and history of splenectomy (p = 0.05). Xmn1-/- was associated with a worse response (p = 0.0001). By multivariate analysis, a better response was associated with the Xmn1 polymorphism (p = 0.008).
CONCLUSION: Hydroxyurea may be an alternative to transfusions for TI patients as well as for TM patients in countries that have limited blood supplies.