Department of Hematology, Seoul St. Mary's Hospital, College of Medicine, The Catholic University of Korea, 505 Banpo-Dong, Seocho-Gu, Seoul, 137-701, Republic of Korea.
Ann Hematol. 2013 Jun;92(6):817-24. doi: 10.1007/s00277-013-1674-8. Epub 2013 Jan 15.
Antithymocyte globulin (ATG) is the drug of choice for immunosuppressive therapy (IST) in patients with severe aplastic anemia (SAA) ineligible for allogeneic stem cell transplantation. Recently, rabbit ATG with cyclosporine A has been used as a first-line IST regimen in patients with SAA because of unavailability of horse ATG. We retrospectively analyzed adult SAA patients who were treated with horse ATG (n=46) or rabbit ATG (n=53) between Feb 2001 and May 2010 to compare hematologic response and survival. Overall response rates at 3, 6, 12, and 18 months were similar in both the horse and rabbit ATG groups: 28.3 versus 35.8 % (P=0.421), 39.1 versus 45.3 % (P=0.537), 45.7 versus 49.1 % (P=0.735), and 47.8 versus 50.9 % (P=0.757), respectively. The complete response (CR) rate at 6 months in the horse ATG was significantly superior in comparison with the rabbit ATG (13.0 versus 1.9 %, P=0.031). But CR rates became similar in both groups after 6 months: 17.4 versus 11.3 % (P=0.387) at 12 months and 21.7 versus 22.6 % (P=0.914) at 18 months. Lymphocyte depletion after ATG was more profound and protracted in the rabbit ATG group compared to the horse ATG group. Overall survival (P=0.460) and failure-free survival (P=0.911) were not significantly different between the two groups. Our retrospective study demonstrated that the efficacy of first-line IST with rabbit ATG is similar to that with horse ATG. However, the time from treatment to CR was longer with rabbit ATG than with horse ATG, partly due to more profound and protracted lymphocyte depletion.
抗胸腺细胞球蛋白（ATG）是不合宜进行异基因干细胞移植的严重再生障碍性贫血（SAA）患者免疫抑制治疗（IST）的首选药物。由于马源性 ATG 无法获得，最近，兔源性 ATG 联合环孢素 A 已被用作 SAA 患者的一线 IST 方案。我们回顾性分析了 2001 年 2 月至 2010 年 5 月期间接受马源性 ATG（n=46）或兔源性 ATG（n=53）治疗的成年 SAA 患者，比较其血液学反应和生存情况。两组患者在 3、6、12 和 18 个月的总反应率相似：28.3%比 35.8%（P=0.421），39.1%比 45.3%（P=0.537），45.7%比 49.1%（P=0.735）和 47.8%比 50.9%（P=0.757）。马源性 ATG 在 6 个月时的完全缓解（CR）率明显优于兔源性 ATG（13.0%比 1.9%，P=0.031）。但 6 个月后两组 CR 率相似：12 个月时为 17.4%比 11.3%（P=0.387），18 个月时为 21.7%比 22.6%（P=0.914）。与马源性 ATG 相比，兔源性 ATG 后淋巴细胞耗竭更明显且持续时间更长。两组患者的总生存率（P=0.460）和无失败生存率（P=0.911）无显著差异。我们的回顾性研究表明，一线 IST 采用兔源性 ATG 的疗效与马源性 ATG 相似。然而，与马源性 ATG 相比，兔源性 ATG 从治疗到 CR 的时间更长，这在一定程度上是由于淋巴细胞耗竭更明显且持续时间更长。
