Manson Martijn L, Derissen Ellen J B, Wijermans Pierre W, Schellens Jan H M, Beijnen Jos H
Universiteit Utrecht, dept. Farmaceutische Wetenschappen, Utrecht, the Netherlands.
Ned Tijdschr Geneeskd. 2012;156(4):A3167.
Myelodysplastic syndrome, a disorder of haematopoiesis, is associated with anaemia and an increased risk of infections, bleeding and the development of acute myeloid leukaemia. The disorder occurs mainly in later life. Until recently the only therapy that could induce sustained remission was allogeneic stem cell transplantation. However in elderly patients caution is needed with this therapy. Increasing awareness of the role of epigenetic changes in cancer development has led to the rediscovery of the cytidine analogues azacitidine and decitabine. At low doses these drugs inhibit DNA methylation. The efficacy of these drugs was demonstrated in the treatment of patients with myelodysplastic syndrome. These drugs showed low toxicity and were relatively well-tolerated in elderly patients. The results with azacitidine and decitabine have demonstrated that manipulation of the epigenetic process offers new antineoplastic treatment options.
骨髓增生异常综合征是一种造血系统疾病，与贫血以及感染、出血风险增加和急性髓系白血病的发生有关。该疾病主要发生在晚年。直到最近，唯一能诱导持续缓解的治疗方法是异基因干细胞移植。然而，对于老年患者，这种治疗需要谨慎。对表观遗传变化在癌症发展中作用的认识不断提高，导致胞苷类似物阿扎胞苷和地西他滨被重新发现。低剂量时，这些药物可抑制DNA甲基化。这些药物在治疗骨髓增生异常综合征患者方面的疗效得到了证实。这些药物毒性低，在老年患者中耐受性相对较好。阿扎胞苷和地西他滨的研究结果表明，对表观遗传过程的调控提供了新的抗肿瘤治疗选择。
