用去甲基化药物治疗骨髓增生异常综合征：阿扎胞苷。
Treatment of advanced myelodysplastic syndrome with demethylating agents: azacitidine.
机构信息
Hôpital Avicenne, Assistance Publique-Hôpitaux de Paris, Service d'hématologie clinique, Paris 13 university, Bobigny, France.
出版信息
Semin Hematol. 2012 Oct;49(4):323-9. doi: 10.1053/j.seminhematol.2012.09.002.
Azacitidine (AZA) improves long-term outcomes of higher-risk myelodysplastic syndrome (MDS) and is now the reference frontline therapy of higher-risk MDS not eligible for allogeneic stem cell transplant. Notably, in a phase III randomized trial, AZA significantly prolonged overall survival (OS) compared to conventional care regimens, in all cytogenetic subgroups. Nevertheless, further improvement of outcome for those patients is warranted, partly by researching for better prognostic factors of response to AZA, and also by developing new therapeutic strategies, in particular by combining AZA and other drugs known to have an effect in MDS. We review the prognostic factors of response and survival of patients treated with AZA, the combination of AZA with other drugs, and the use of AZA in specific situations, such as therapy-related MDS, chronic myelomonocytic leukemia (CMML), acute myeloid leukemia (AML), and in MDS/AML occurring in the course of myeloproliferative neoplasms (MPN).
阿扎胞苷（AZA）可改善高危骨髓增生异常综合征（MDS）患者的长期预后，现已成为不适合异体干细胞移植的高危 MDS 的一线参考治疗药物。值得注意的是，在一项 III 期随机试验中，与常规治疗方案相比，AZA 显著延长了所有细胞遗传学亚组患者的总生存期（OS）。然而，仍需要进一步改善这些患者的预后，部分方法是研究对 AZA 反应的更好的预后因素，以及开发新的治疗策略，特别是通过将 AZA 与其他已知对 MDS 有疗效的药物联合使用。我们回顾了接受 AZA 治疗的患者的反应和生存的预后因素、AZA 与其他药物的联合应用，以及 AZA 在特定情况下的应用，如治疗相关 MDS、慢性粒单核细胞白血病（CMML）、急性髓系白血病（AML），以及在骨髓增生性肿瘤（MPN）的病程中发生的 MDS/AML。
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