Shin Seung Hwan, Lee Sung Eun, Lee Jong Wook
Department of Hematology, Yeouido St. Mary's Hospital, College of Medicine, The Catholic University of Korea, Seoul, Korea.
Department of Hematology, Catholic Blood and Marrow Transplantation Center, Seoul St. Mary's Hospital, College of Medicine, The Catholic University of Korea, Seoul, Korea.
Korean J Intern Med. 2014 Nov;29(6):713-26. doi: 10.3904/kjim.2014.29.6.713. Epub 2014 Oct 31.
Recent advances in the treatment of aplastic anemia (AA) made most of patients to expect to achieve a long-term survival. Allogeneic stem cell transplantation (SCT) from HLA-matched sibling donor (MSD-SCT) is a preferred first-line treatment option for younger patients with severe or very severe AA, whereas immunosuppressive treatment (IST) is an alternative option for others. Horse anti-thymocyte globuline (ATG) with cyclosporin A (CsA) had been a standard IST regimen with acceptable response rate. Recently, horse ATG had been not available and replaced with rabbit ATG in most countries. Subsequently, recent comparative studies showed that the outcomes of patients who received rabbit ATG/CsA were similar or inferior compared to those who received horse ATG/CsA. Therefore, further studies to improve the outcomes of IST, including additional eltrombopag, are necessary. On the other hand, the upper age limit of patients who are able to receive MSD-SCT as first-line treatment is a current issue because of favorable outcomes of MSD-SCT of older patients using fludarabine-based conditioning. In addition, further studies to improve the outcomes of patients who receive allogeneic SCT from alternative donors are needed. In this review, current issues and the newly emerging trends that may improve their outcomes in near futures will be discussed focusing the management of patients with AA.
再生障碍性贫血（AA）治疗的最新进展使大多数患者期望实现长期生存。来自人类白细胞抗原（HLA）匹配同胞供者的异基因干细胞移植（SCT）（MSD-SCT）是重度或极重度AA年轻患者首选的一线治疗方案，而免疫抑制治疗（IST）是其他患者的替代方案。马抗胸腺细胞球蛋白（ATG）联合环孢素A（CsA）曾是一种具有可接受缓解率的标准IST方案。最近，在大多数国家马ATG已无法获得并被兔ATG所取代。随后，近期的比较研究表明，接受兔ATG/CsA治疗的患者的结局与接受马ATG/CsA治疗的患者相似或更差。因此，有必要开展进一步研究以改善IST的结局，包括加用艾曲泊帕。另一方面，由于使用含氟达拉滨预处理方案的老年患者进行MSD-SCT的结局良好，能够接受MSD-SCT作为一线治疗的患者的年龄上限是当前的一个问题。此外，需要开展进一步研究以改善接受来自替代供者的异基因SCT患者的结局。在本综述中，将围绕AA患者的管理，讨论当前问题以及可能在不久的将来改善其结局的新出现趋势。
