Wijermans P W, Krulder J W, Huijgens P C, Neve P
Department of Haematology, Leyenburg Hospital, The Hague, The Netherlands.
Leukemia. 1997 Jan;11(1):1-5. doi: 10.1038/sj.leu.2400526.
There is no standard therapy for elderly patients with high-risk myelodysplastic syndrome (MDS). The treatment options of low-dose Ara-C and haematopoietic growth factors are disappointing in regard to response rate or response duration. We tested the treatment with a 72-h continuous infusion of low-dose 5-Aza-2'-deoxycytidine (DAC) in a group of 29 elderly patients with high-risk MDS. In 15 patients (54%) we observed a response. Eight complete responses were reached, even among patients with bad prognostic cytogenetic findings. The actuarial median survival from the start of the therapy was 46 weeks. The only (and major) toxicity was myelosuppression, leading to a prolonged cytopenic period and thus leading to five toxic deaths (17%) in this high-risk patient group. We conclude that DAC is an effective drug in the treatment of MDS patients and that it probably works via its cytotoxic activity. Myelotoxicity is its major adverse effect.
对于老年高危骨髓增生异常综合征（MDS）患者，尚无标准治疗方法。低剂量阿糖胞苷和造血生长因子的治疗方案在缓解率或缓解持续时间方面令人失望。我们对一组29例老年高危MDS患者进行了低剂量5-氮杂-2'-脱氧胞苷（DAC）72小时持续输注治疗测试。15例患者（54%）出现缓解。即使在预后不良的细胞遗传学结果患者中，也有8例达到完全缓解。从治疗开始计算的精算中位生存期为46周。唯一（也是主要的）毒性是骨髓抑制，导致血细胞减少期延长，从而在这个高危患者组中导致5例毒性死亡（17%）。我们得出结论，DAC是治疗MDS患者的有效药物，其作用可能是通过细胞毒性活性发挥的。骨髓毒性是其主要不良反应。
