Risitano Antonio M, Perna Fabiana
Department of Biochemistry and Medical Biotechnologies, Federico II University of Naples, Naples, Italy.
Pediatr Rep. 2011 Jun 22;3 Suppl 2(Suppl 2):e7. doi: 10.4081/pr.2011.s2.e7.
Acquired aplastic anemia (AA) is the typical bone marrow failure syndrome characterized by an empty bone marrow; an immune-mediated pathophysiology has been demonstrated by experimental works as well as by clinical observations. Immunusuppressive therapy (IST) is a key treatment strategy for aplastic anemia; since 20 years the standard IST for AA patients has been anti-thymocyte globuline (ATG) plus cyclosporine A (CyA), which results in response rates ranging between 50% and 70%, and even higher overall survival. However, primary and secondary failures after IST remain frequent, and to date all attempts aiming to overcome this problem have been unfruitful. Here we review the state of the art of IST for AA in 2010, focusing on possible strategies to improve current treatments. We also discuss very recent data which question the equality of different ATG preparations, leading to a possible reconsideration of the current standards of care for AA patients.
获得性再生障碍性贫血（AA）是典型的骨髓衰竭综合征，其特征为骨髓空虚；实验研究以及临床观察均已证实其病理生理机制为免疫介导。免疫抑制治疗（IST）是再生障碍性贫血的关键治疗策略；20年来，AA患者的标准IST方案一直是抗胸腺细胞球蛋白（ATG）加环孢素A（CyA），该方案的缓解率在50%至70%之间，总体生存率甚至更高。然而，IST后的原发性和继发性失败仍然很常见，迄今为止，所有旨在克服这一问题的尝试均未成功。在此，我们回顾2010年AA的IST现状，重点关注改善当前治疗的可能策略。我们还讨论了最近的一些数据，这些数据对不同ATG制剂的等效性提出了质疑，可能会促使人们重新考虑AA患者当前的护理标准。
