Gaston M H, Verter J I, Woods G, Pegelow C, Kelleher J, Presbury G, Zarkowsky H, Vichinsky E, Iyer R, Lobel J S
N Engl J Med. 1986 Jun 19;314(25):1593-9. doi: 10.1056/NEJM198606193142501.
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 under the age of three years at the time of entry. The children were randomly assigned to receive either 125 mg of penicillin V 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 percent 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 vs. 2 of 105; P = 0.0025), with no deaths from pneumococcal septicemia occurring in the penicillin group but three deaths from the infection occurring 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 four months of age to decrease the morbidity and mortality associated with pneumococcal septicemia.
镰状细胞贫血患儿更容易受到细菌感染，尤其是由肺炎链球菌引起的感染。因此，我们开展了一项多中心、随机、双盲、安慰剂对照的临床试验，以测试对于入组时年龄在三岁以下的镰状细胞贫血患儿，每日规律口服青霉素是否会降低由肺炎链球菌引起的确诊败血症的发生率。这些儿童被随机分配，分别每日两次接受125毫克青霉素V钾（105名儿童）或安慰剂（110名儿童）。在平均随访15个月后，试验提前8个月终止，此时观察到青霉素治疗组的感染发生率相比安慰剂组降低了84%（110名患者中有13例，105名患者中有2例；P = 0.0025），青霉素组无肺炎球菌败血症死亡病例，而安慰剂组有3例因感染死亡。基于这些结果，我们得出结论，新生儿期应筛查镰状细胞血红蛋白病，患有镰状细胞贫血的儿童应在四个月大时接受口服青霉素预防性治疗，以降低与肺炎球菌败血症相关的发病率和死亡率。
