Azacitidine (AZA) is recognized as a vital drug used in the therapy of myelodysplastic syndromes (MDS) due to its beneficial effect on survival and quality of life. Nevertheless, many patients fail to respond to AZA treatment, as prognostic factors still are not identified. The present retrospective analysis included 79 patients with MDS treated with AZA as first-line therapy in a real-life setting. The percentage of patients with good, intermediate, and poor cytogenetics was 46.8%, 11.4%, and 34.2%, respectively. The overall response rate (complete remission [CR], partial remission [PR], and hematological improvement [HI]) was 24%. The CR, PR, and HI rates were 13.9%, 2.5%, and 7.6%, respectively. Stable disease (SD) was documented in 40.5% of patients. The median overall survival (OS) and progression-free survival (PFS) were 17.6 and 14.96 months, respectively. Patients with ORR and SD had a significantly longer median OS (23.8 vs. 5.7 months,p= 0.0005) and PFS (19.8 vs. 3.5 months,p< 0.001) compared to patients who did not respond to AZA. In univariate analysis, only an unfavorable cytogenetic group was a prognostic factor of a lower response rate (p= 0.03). In a multivariate model, older age (p= 0.047), higher IPSS (International Prognostic Scoring System) risk (p= 0.014), and higher IPSS-R cytogenetic risk (p= 0.004) were independent factors of shorter OS. Independent prognostic factors for shorter PFS were age (p= 0.001), IPSS risk (p= 0.02), IPSS cytogenetic risk (p= 0.002), and serum ferritin level (p= 0.008). The safety profile of AZA was predictable and consistent with previous studies. In conclusion, our study confirms the efficacy and safety of AZA in a real-world population and identifies potential biomarkers for response and survival.
阿扎胞苷（AZA）因其对生存期和生活质量的改善作用，被公认为治疗骨髓增生异常综合征（MDS）的关键药物。然而，由于预后因素尚未明确，许多患者对AZA治疗无反应。本次回顾性分析纳入了79例在真实临床环境中接受AZA一线治疗的MDS患者。细胞遗传学良好、中等和不良的患者比例分别为46.8%、11.4%和34.2%。总体缓解率（完全缓解[CR]、部分缓解[PR]和血液学改善[HI]）为24%。CR、PR和HI率分别为13.9%、2.5%和7.6%。40.5%的患者达到疾病稳定（SD）。中位总生存期（OS）和无进展生存期（PFS）分别为17.6个月和14.96个月。与对AZA无反应的患者相比，达到客观缓解或疾病稳定的患者中位OS（23.8个月 vs. 5.7个月，p=0.0005）和PFS（19.8个月 vs. 3.5个月，p<0.001）显著延长。单因素分析显示，仅不良细胞遗传学分组是低缓解率的预后因素（p=0.03）。在多因素模型中，高龄（p=0.047）、较高IPSS（国际预后评分系统）风险（p=0.014）及较高IPSS-R细胞遗传学风险（p=0.004）是较短OS的独立影响因素。较短PFS的独立预后因素包括年龄（p=0.001）、IPSS风险（p=0.02）、IPSS细胞遗传学风险（p=0.002）和血清铁蛋白水平（p=0.008）。AZA的安全性特征可预测且与既往研究一致。总之，本研究在真实世界人群中证实了AZA的疗效和安全性，并确定了潜在的治疗反应和生存生物标志物。
肿瘤(癌症)患者之家