Effect of hydroxyurea in sickle cell anemia: A clinical trial in children and teenagers with severe sickle cell anemia and sickle cell beta-thalassemia

This study evaluated the efficacy of hydroxyurea treatment in the preventio n of vaso-occlusive crises among children and teenagers with severe sickle cell anemia and sickle cell beta-thalassemia. Nineteen children and young a dults with severe sickle cell disease were enrolled to the hydroxyurea trea tment trial. The incidence of vaso-occlusive crises, acute chest syndrome, hemolytic crises, splenic sequestration episodes, blood transfusions, and h ospital days in the 2 years before hydroxyurea (HU) treatment were compared with the same parameters in the first 2 years of treatment. The patients r eceived 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 I month in the Hgb, MCV, MCH, and MCHC levels and were more notable after 3 months. The increase in the Hgb F level became import ant after 3 months of HU therapy and was highly significant (P < .001) beyo nd 6 months. No differences were observed in the RDW, reticulocyte count, H gb S, and Hgb A(2). 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 demonstrate d during the HU treatment period compared to the same period before. The cl inical and laboratory response to HU was dramatic in severely affected sick le 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 n o severe adverse effects were observed.