Clinical response and adverse events in young patients with sickle cell disease treated with hydroxyurea.

The authors studied the long-term clinical and hematological response to hydroxyurea (HU) therapy in young patients, with either S/beta-thalassemia (beta(thal)) (8 patients) or SS (6 patients). All patients with S/beta(thal) responded well to treatment. Longitudinal evaluation of Hb, HbF, and MCV showed a significant increase compared to baseline levels, but the pattern of HbF changes varied among patients.