Background/Objectives: After first-line treatment, elderly patients with acute myeloid leukemia (AML) often become unfit to continue intensive chemotherapy despite having achieved a response. This trial aimed to determine the efficacy of maintenance treatment with azacitidine in AML patients who are ineligible to continue intensive treatment after remission.Methods: A single-arm, multicenter, phase II clinical trial (EudraCT: 2010-020432-18) including patients with AML with complete (CR) or partial remission (PR) after one or two cycles of intensive induction chemotherapy and ineligible to continue intensive treatment was conducted. Efficacy was measured as the response rate, progression-free survival (PFS), and overall survival (OS), and was assessed in the intention-to-treat (ITT) population (all patients). Quality of life was also assessed.Results: Thirty-two patients were included, with a mean age of 73.3 years (SD 3.8) (53.1% male); sixteen patients (50.0%) reached the sixth treatment cycle. The best response was CR in 11 patients (68.8%) and PR in 2 patients (12.5%) at cycle six, and CR in 15 patients (46.9%) and PR in 5 patients (15.6%) overall. The median PFS was 6.7 months (95% CI 3.1–8.7), and OS was 11.5 months (95% CI 6.6–15.9). The daily function (p= 0.0296), cognitive function (p= 0.0412), and social function (p= 0.0275) scales of the EORTC QLQ-C30 questionnaire significantly improved. Thirty-one patients (96.9%) experienced adverse events; six (1.9%) were serious.Conclusions: Azacitidine is a safe and well-tolerated maintenance treatment option for AML patients unfit for intensive therapy following a response to induction, although the single-arm phase II design precludes direct causal inference. Patients achieved promising results regarding PFS and remission rates, with improved quality of life.
背景/目的：急性髓系白血病老年患者经一线治疗后，即使获得缓解，也常因身体状况无法继续接受强化化疗。本研究旨在评估阿扎胞苷作为维持治疗，对于诱导缓解后不适合继续强化治疗的AML患者的疗效。 方法：本研究为单臂、多中心II期临床试验（EudraCT：2010-020432-18），纳入经一至两个周期强化诱导化疗后获得完全缓解或部分缓解，且不适合继续强化治疗的AML患者。疗效评估指标包括缓解率、无进展生存期和总生存期，在意向治疗人群中进行评估，同时评估患者生活质量。 结果：共纳入32例患者，平均年龄73.3岁（标准差3.8），男性占53.1%。16例患者完成第六周期治疗。第六周期时最佳缓解情况为：完全缓解11例，部分缓解2例；总体缓解情况为：完全缓解15例，部分缓解5例。中位无进展生存期为6.7个月，总生存期为11.5个月。EORTC QLQ-C30量表的日常功能、认知功能和社会功能评分均显著改善。31例患者出现不良事件，其中6例为严重不良事件。 结论：对于诱导治疗后获得缓解但不适合强化治疗的AML患者，阿扎胞苷是一种安全且耐受性良好的维持治疗选择。尽管单臂II期研究设计无法直接推断因果关系，但患者在无进展生存期和缓解率方面获得良好结果，生活质量得到改善。
肿瘤(癌症)患者之家