This study evaluated the efficacy of
hydroxyurea treatment in the prevention of vaso-occlusive crises among children and teenagers with severe
sickle cell anemia and sickle cell
beta-thalassemia. Nineteen children and young adults with severe
sickle cell disease were enrolled to the
hydroxyurea treatment trial. The incidence of vaso-occlusive crises,
acute chest syndrome, hemolytic crises, splenic sequestration episodes,
blood transfusions, and hospital days in the 2 years before
hydroxyurea (HU) treatment were compared with the same parameters in the first 2 years of treatment. The patients received a mean dose of 21.3 mg/kg/day daily and were treated during a mean period of 40.3 +/- 14 months (range 20 to 68 months). Significant increases were observed after 1 month in the Hgb, MCV, MCH, and MCHC levels and were more notable after 3 months. The increase in the Hgb F level became important after 3 months of HU
therapy and was highly significant (p < .001) beyond 6 months. No differences were observed in the RDW, reticulocyte count, Hgb S, and Hgb A2. Severe
neutropenia was observed in one case. A decrease in the frequency of vaso-occlusive crises,
acute chest syndrome, hemolytic crises,
blood transfusions, and days spent in the hospital was demonstrated during the HU treatment period compared to the same period before. The clinical and laboratory response to HU was dramatic in severely affected
sickle cell anemia (SCA) patients. The response to HU in children and teenagers with severe
sickle cell anemia is similar to the response in adults, and no severe adverse effects were observed.