Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins University, Baltimore, MD 21231, USA.
Hematology Am Soc Hematol Educ Program. 2011;2011:550-5. doi: 10.1182/asheducation-2011.1.550.
Ongoing analysis of the seminal AZA-001 study has taught many important lessons in the use of DNA methyltransferase (DNMT) inhibitors. The data emphasize the importance of patience in the use of these drugs, with several cycles required for the manifestations of hematologic responses. Improved survival in patients with high-risk myelodysplastic syndrome (MDS) treated with azacitidine extends to patients with any International Working Group-defined hematologic response; however, the benefit to patients with stable disease is less clear. A great deal remains to be learned about the optimal dosing and scheduling of the DNMT inhibitors, alone and in combination. New information on the impact of DNMT inhibitors on the immune system and on stem cells will likely lead to novel uses of these drugs in MDS and other hematologic and nonhematologic malignancies.
正在对 AZA-001 研究进行持续分析，该研究为 DNA 甲基转移酶（DNMT）抑制剂的应用提供了许多重要的经验教训。这些数据强调了在使用这些药物时要有耐心，因为需要几个周期才能出现血液学反应。用阿扎胞苷治疗高危骨髓增生异常综合征（MDS）患者的生存率提高适用于任何国际工作组定义的血液学反应的患者；然而，对疾病稳定患者的益处则不太明确。关于 DNMT 抑制剂的最佳剂量和方案，无论是单独使用还是联合使用，还有很多需要了解。DNMT 抑制剂对免疫系统和干细胞的影响的新信息可能会导致这些药物在 MDS 和其他血液系统和非血液系统恶性肿瘤中的新用途。
