Doney K C, Weiden P L, Buckner C D, Storb R, Thomas E D
Exp Hematol. 1981 Sep;9(8):829-34.
Nineteen patients with severe aplastic anemia were treated with a 4-day course of horse-anti-human thymocyte globulin (ATG). Thirteen of these patients also received an infusion of HLA one haplotype-identical bone marrow. Toxicity of ATG included fever, chills, rash, arthralgias and elevated liver function tests. Platelet transfusion requirements increased during therapy. Eleven patients died 0.2-9.4 months after beginning ATG therapy. None of the 11 patients had any improvement in hematologic status prior to death. The eight surviving patients have been followed for at least 24 months. Six had evidence of hematologic improvement within 6-8 weeks after ATG therapy and are transfusion-independent. The other two patients improved more than one year after treatment. Survival after ATG therapy did not correlate with the presumed etiology of aplasia, duration of aplasia, patient age or sex, prior therapy, or admission granulocyte count. Addition of bone marrow infusion to ATG treatment also did not affect survival. This study demonstrated the necessity for a randomized trial of ATG versus supportive care alone for the treatment of severe aplastic anemia.
19例重型再生障碍性贫血患者接受了为期4天的马抗人胸腺细胞球蛋白（ATG）治疗。其中13例患者还输注了一个单倍型相合的HLA骨髓。ATG的毒性包括发热、寒战、皮疹、关节痛和肝功能检查异常。治疗期间血小板输注需求增加。11例患者在开始ATG治疗后0.2至9.4个月死亡。这11例患者在死亡前血液学状态均无改善。8例存活患者已随访至少24个月。6例患者在ATG治疗后6至8周有血液学改善证据，且不再依赖输血。另外2例患者在治疗一年多后有所改善。ATG治疗后的生存率与再生障碍性贫血的推测病因、再生障碍性贫血持续时间、患者年龄或性别、既往治疗或入院时粒细胞计数均无相关性。在ATG治疗中加用骨髓输注也不影响生存率。本研究表明有必要进行一项关于ATG与单纯支持治疗对比治疗重型再生障碍性贫血的随机试验。
