Champlin R, Ho W, Gale R P
N Engl J Med. 1983 Jan 20;308(3):113-8. doi: 10.1056/NEJM198301203080301.
We evaluated the efficacy of antithymocyte globulin for the treatment of moderate to severe aplastic anemia in a randomized controlled study. Eleven of 21 patients initially randomized to receive antithymocyte globulin (given intravenously on eight consecutive days) had sustained improvement in hematopoiesis within three months of treatment; none of 21 control patients who received supportive care alone improved (P = 0.0005). Six of 12 control patients who subsequently received antithymocyte globulin improved. Responders had gradual improvement in hematopoiesis, but none recovered completely normal peripheral-blood counts. The severity of bone-marrow failure, age, cause of aplastic anemia, and transfusion history had no apparent bearing on treatment outcome. The interval from diagnosis to antithymocyte globulin treatment correlated inversely with the chance of a treatment response, although this correlation was not statistically significant. These data indicate that antithymocyte globulin is effective in improving hematopoiesis in some patients with aplastic anemia.
我们在一项随机对照研究中评估了抗胸腺细胞球蛋白治疗中重度再生障碍性贫血的疗效。最初随机接受抗胸腺细胞球蛋白治疗（连续八天静脉给药）的21例患者中有11例在治疗三个月内造血功能持续改善；而仅接受支持治疗的21例对照患者均无改善（P = 0.0005）。随后接受抗胸腺细胞球蛋白治疗的12例对照患者中有6例病情改善。有反应者的造血功能逐渐改善，但外周血计数均未完全恢复正常。骨髓衰竭的严重程度、年龄、再生障碍性贫血的病因及输血史对治疗结果无明显影响。从诊断到抗胸腺细胞球蛋白治疗的间隔时间与治疗反应的可能性呈负相关，尽管这种相关性无统计学意义。这些数据表明，抗胸腺细胞球蛋白对部分再生障碍性贫血患者改善造血功能有效。
