Prophylaxis With Oral Penicillin in Children With Sickle Cell Anemia: A Randomized Trial

Children with sickle cell anemia have an increased susceptibility to bacterial infections, especially to those caused by Streptococcus pneumoniae. We therefore conducted a multicenter, randomized, double-blind, placebo-controlled clinical trial to test whether the regular, daily administration of oral penicillin would reduce the incidence of documented septicemia due to S pneumoniae in children with sickle cell anemia who were younger than 3 years of age at the time of entry. The children were randomly assigned to receive either 125 mg of penicillin potassium (105 children) or placebo (110 children) twice daily. The trial was terminated 8 months early, after an average of 15 months of follow-up, when an 84% reduction in the incidence of infection was observed in the group treated with penicillin, as compared with the group given placebo (13 of 110 patients v 2 of 105; P = .0025). There were no deaths from pneumococcal septicemia in the penicillin-treated group but three deaths from the infection occurred in the placebo group. On the basis of these results, we conclude that children should be screened in the neonatal period for sickle cell hemoglobinopathy and that those with sickle cell anemia should receive prophylactic therapy with oral penicillin by 4 months of age to decrease the morbidity and mortality [SEE FIGURE IN SOURCE PDF.] associated with pneumococcal septicemia. (Previously published in N Engl J Med 1986;314:1593-1599.