Zheng Yizhou, Liu Yongze, Chu Yulin
Severe Aplastic Anemia Studying Program, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Science & Peking Union Medical College, Tianjin, People's Republic of China.
Exp Hematol. 2006 Jul;34(7):826-31. doi: 10.1016/j.exphem.2006.03.017.
This study was designed to investigate four different immunosuppressive therapy (IST) regimens as treatment of acquired severe aplastic anemia (SAA).
142 consecutive SAA patients were randomized to receive one of the following IST regimens: equine anti-human thymocyte immunoglobulin (E-ATG) alone (IST regimen I); E-ATG and cyclosporine A (CSA) (IST regimen II); E-ATG, CSA plus recombinant human granulocyte-macrophage colony-stimulating factor (rhuGM-CSF) and rhu erythropoietin (rhuEPO) (IST regimen III); or rabbit ATG (ATG-F), CSA, rhuGM-CSF, and rhuEPO (IST regimen IV). No repeated courses of E-ATG or ATG-F were given for nonresponders. All patients also received stanozolol or testosteron propionate.
The overall response rate to IST regimen I was 58%. The response to IST regimen II (79%) was significantly higher (p = 0.04), more rapid and complete than after IST regimen I. The response rate to IST regimen IV (53%) was significantly lower than that of IST regimen III (73%, p = 0.039). The additional use of growth factors did not reduce early deaths and did not accelerate hematopoietic recovery after IST. Of the 142 patients enrolled in this trial, 92 (65%) are alive at a median follow-up time of 102 months (range, 54-166 months). The 5-year actuarial survival for IST regimens I, II, III, and IV was 58%, 81%, 80%, and 66%, respectively.
The combination of E-ATG and CSA remains the best combination for the treatment of SAA patients, producing a survival advantage at 5 years. The addition of growth factors did not improve these results. Rabbit ATG-F appeared less effective than E-ATG.
本研究旨在调查四种不同的免疫抑制治疗（IST）方案用于治疗获得性重型再生障碍性贫血（SAA）的效果。
142例连续性SAA患者被随机分配接受以下IST方案之一：单独使用马抗人胸腺细胞免疫球蛋白（E-ATG）（IST方案I）；E-ATG和环孢素A（CSA）（IST方案II）；E-ATG、CSA加重组人粒细胞-巨噬细胞集落刺激因子（rhuGM-CSF）和重组人促红细胞生成素（rhuEPO）（IST方案III）；或兔抗胸腺细胞球蛋白（ATG-F）、CSA、rhuGM-CSF和rhuEPO（IST方案IV）。对无反应者不给予重复疗程的E-ATG或ATG-F。所有患者还接受司坦唑醇或丙酸睾酮治疗。
IST方案I的总体缓解率为58%。IST方案II的缓解率（79%）显著更高（p = 0.04），比IST方案I更快且更完全。IST方案IV的缓解率（53%）显著低于IST方案III（73%，p = 0.039）。生长因子的额外使用并未降低早期死亡率，也未加速IST后的造血恢复。在该试验纳入的142例患者中，92例（65%）在中位随访时间102个月（范围54 - 166个月）时存活。IST方案I、II、III和IV的5年精算生存率分别为58%、81%、80%和66%。
E-ATG和CSA联合使用仍然是治疗SAA患者的最佳组合，在5年时具有生存优势。生长因子的添加并未改善这些结果。兔ATG-F似乎比E-ATG效果更差。
