Young N, Griffith P, Brittain E, Elfenbein G, Gardner F, Huang A, Harmon D, Hewlett J, Fay J, Mangan K
Clinical Hematology Branch, NHLBI, Bethesda, MD 20892.
Blood. 1988 Dec;72(6):1861-9.
One hundred fifty patients with bone marrow failure were treated in three groups with antithymocyte globulin (ATG; Upjohn, Kalamazoo, MI) in a multicenter trial. Patients were assessed at 3, 6, and 12 months after initiation of treatment by three criteria: transfusion independence, clinical improvement, and blood counts. Group I consisted of 77 patients with acute severe aplastic anemia, randomized to receive either ten or 28 days of ATG. There was no significant difference between the two arms of this protocol: 47% of all patients were clinically improved and 31% were transfusion independent at 3 months. Of the severely affected patients, 27% died before 3 months; most deaths occurred early in treatment. Factors associated with survival in severely affected patients included male sex, age less than 40 years, absolute neutrophil count greater than 200/microL, and idiopathic etiology. Neutrophil counts generally increased by 8 weeks after treatment, but patients continued to show improvement to 1 year posttreatment. In Group II, 44 patients with moderate or chronic severe aplastic anemia were randomized to receive either ten days of ATG or 3 months of high-dose nandrolone decanoate. No patient initially treated with androgens recovered, but 28% of ATG-treated cases achieved transfusion independence at 3 months. Group III consisted of patients with a variety of bone marrow failure syndromes. Patients with pancytopenia and cellular bone marrow showed response rates similar to those of patients with chronic or moderate aplastic anemia.
在一项多中心试验中，150例骨髓衰竭患者被分为三组，接受抗胸腺细胞球蛋白（ATG；美国密歇根州卡拉马祖市普强公司生产）治疗。在开始治疗后的3个月、6个月和12个月，依据三项标准对患者进行评估：是否不再需要输血、临床症状是否改善以及血细胞计数情况。第一组有77例急性重型再生障碍性贫血患者，随机接受10天或28天的ATG治疗。该方案的两组之间没有显著差异：3个月时，所有患者中有47%临床症状改善，31%不再需要输血。在病情严重的患者中，27%在3个月前死亡；大多数死亡发生在治疗早期。病情严重患者生存相关的因素包括男性、年龄小于40岁、绝对中性粒细胞计数大于200/微升以及特发性病因。治疗后8周中性粒细胞计数一般会升高，但患者在治疗后1年仍持续改善。第二组有44例中度或慢性重型再生障碍性贫血患者，随机接受10天的ATG治疗或3个月的大剂量癸酸诺龙治疗。最初接受雄激素治疗的患者无一康复，但接受ATG治疗的患者中有28%在3个月时不再需要输血。第三组包括患有各种骨髓衰竭综合征的患者。全血细胞减少且骨髓细胞性的患者的缓解率与慢性或中度再生障碍性贫血患者相似。
