Venetoclax-azacitidine is the standard of treatment for unfit acute myeloid leukemia patients. In the VIALE-A study, treatment was given until progression but there are no data on its optimal duration for responding patients who do not tolerate indefinite therapy. We retrospectively analyzed the outcome of patients who discontinued venetoclax or venetoclax-azacitidine due to poor tolerance. Sixty-two newly diagnosed (ND) AML patients and 22 patients with morphological relapse or refractory AML were included. In the ND cohort (n = 62), 28 patients stopped venetoclax and azacitidine and 34 patients continued azacitidine monotherapy. With a median follow-up of 23 months (IQR, 20–32), median overall survival and treatment-free survival were 44 (IQR, 16-NR) and 16 (IQR, 8–27) months, respectively. Patients who stopped both treatments and those who continued azacitidine monotherapy had the same outcomes. Negative minimal residual disease was associated with a 2-year treatment-free survival of 80%. In the RR cohort (n = 22), median overall survival and treatment-free survival were 19 (IQR, 17–31) and 10 (IQR, 5-NR) months, respectively. Prior number of venetoclax-azacitidine cycles and IDH mutations were associated with increased overall survival. The only factor significantly impacting treatment-free survival was the number of prior cycles. This study suggests that patients who discontinued treatment in remission have favorable outcomes supporting the rationale for prospective controlled trials.

中文翻译：

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对维奈托克和阿扎胞苷有反应并停止治疗的急性髓系白血病患者的结果


维奈托克-阿扎胞苷是不适宜的急性髓性白血病患者的标准治疗方法。在 VIALE-A 研究中，治疗一直持续到疾病进展，但对于不能耐受无限期治疗的有反应患者，没有关于其最佳持续时间的数据。我们回顾性分析了因耐受性差而停用维奈托克或维奈托克-阿扎胞苷的患者的结果。纳入 62 名新诊断 (ND) AML 患者和 22 名形态学复发或难治性 AML 患者。在 ND 队列中 ( n = 62)，28 名患者停止维奈托克和阿扎胞苷，34 名患者继续阿扎胞苷单药治疗。中位随访时间为 23 个月（IQR，20-32），中位总生存期和无治疗生存期分别为 44（IQR，16-NR）和 16（IQR，8-27）个月。停止两种治疗的患者和继续阿扎胞苷单一治疗的患者具有相同的结果。微小残留病阴性与 80% 的 2 年无治疗生存率相关。在 RR 队列中 ( n = 22)，中位总生存期和无治疗生存期分别为 19 (IQR，17-31) 和 10 (IQR，5-NR) 个月。先前的维奈托克-阿扎胞苷循环次数和IDH突变与总生存期增加相关。显着影响无治疗生存的唯一因素是之前的周期数。这项研究表明，在缓解期停止治疗的患者具有良好的结果，支持前瞻性对照试验的基本原理。