Department of Hematology, Asan Medical Center, University of Ulsan College of Medicine, Seoul, South Korea.
Ann Hematol. 2010 Jan;89(1):15-23. doi: 10.1007/s00277-009-0771-1. Epub 2009 Jun 19.
This study was performed to identify whether cytogenetics, International Prognostic Scoring System (IPSS), or World Health Organization Classification-Based Prognostic Scoring System are predictive of the efficacy of azacitidine in patients with myelodysplastic syndrome (MDS). We retrospectively reviewed the clinical records of 113 patients with MDS treated with azacitidine. The "response alternating disease natural history," "cytogenetic response," and "hematologic improvement" were assessed by serial bone marrow biopsy, cytogenetic study, and hemogram analyses. The complete and partial remission rates were 17.6% and 3.9% in 51 evaluable patients. There were no significant differences in response rate in the different cytogenetic/IPSS/WPSS groups. The overall hematologic response (HR) rate was 49.6%, and the HR rate was significantly greater in patients classed as "very high" risk according to the WPSS compared with other patient groups. The 1-year overall survival (OS) rate was higher among patients with HR compared with those without HR (80.9% vs 63.3%, p = 0.046), and the 1-year OS rate among patients classed as being at high risk by each criteria was similar to that of patients classed as being at low risk. The hazard ratio of death among patients with HR compared with those without HR was 0.17 (95% CI 0.04-0.69) for high + very high risk group based on WPSS. Patients in the WPSS high-risk group had an increased HR rate compared with other patient groups, and the achievement of HR was associated with a significant increase in OS. Azacitidine showed similar efficacy in all patient groups, even in patients with poor cytogenetics and in high-risk groups.
这项研究旨在确定细胞遗传学、国际预后评分系统（IPSS）或基于世界卫生组织分类的预后评分系统是否可预测骨髓增生异常综合征（MDS）患者接受阿扎胞苷治疗的疗效。我们回顾性分析了 113 例接受阿扎胞苷治疗的 MDS 患者的临床资料。通过连续骨髓活检、细胞遗传学研究和全血细胞计数分析评估“交替疾病自然史的反应”、“细胞遗传学反应”和“血液学改善”。在 51 例可评价患者中，完全缓解率和部分缓解率分别为 17.6%和 3.9%。在不同的细胞遗传学/IPSS/WPSS 组中，缓解率无显著差异。总的血液学反应（HR）率为 49.6%，根据 WPSS 分类为“极高”风险的患者 HR 率显著高于其他患者组。与无 HR 者相比，有 HR 者的 1 年总生存（OS）率更高（80.9%比 63.3%，p=0.046），而根据每个标准分类为高危的患者 1 年 OS 率与低危患者相似。与无 HR 者相比，有 HR 者的死亡风险比为 0.17（95%CI 0.04-0.69），这是基于 WPSS 的高危+极高危组。WPSS 高危组患者的 HR 率高于其他患者组，而达到 HR 与 OS 显著增加相关。阿扎胞苷在所有患者组中均显示出相似的疗效，即使是在细胞遗传学较差和高危组患者中也是如此。
