The Alfred Hospital and Monash University, Melbourne, Australia.
The Walter and Eliza Hall Institute of Medical Research, Parkville, Melbourne, Australia.
Blood Adv. 2022 Jul 12;6(13):3879-3883. doi: 10.1182/bloodadvances.2022007083.
The clinical benefit of adding venetoclax (VEN) to hypomethylating agents or low-dose cytarabine in older and/or unfit patients with newly diagnosed acute myeloid leukemia (AML) has been confirmed in phase 3 studies. With the increased uptake of VEN-based therapies for patients with AML, a pertinent question is whether treatment can be safely ceased among patients who have achieved sustained remission. We hypothesized that a proportion of patients opting to cease therapy may benefit from a treatment-free remission (TFR) period without indefinite treatment. We report the retrospective outcomes of 29 patients in remission for a minimum of 12 months on VEN-based therapy, with 55% continuing therapy until disease progression and 45% electively ceasing treatment (STOP). With follow-up exceeding 5 years, we observed a median TFR lasting 45.8 months among the STOP cohort, with >50% of patients still in sustained remission at the data cutoff. The risk of relapse and duration of relapse-free and overall survival were similar between the 2 cohorts. Factors favoring sustained TFR within the cohort included NPM1 and/or IDH2 mutation at diagnosis, complete remission without measurable residual disease, and at least 12 months of VEN-based combination therapy prior to treatment cessation.
在新诊断的老年和/或不适合接受治疗的急性髓系白血病（AML）患者中，联合 venetoclax（VEN）与低剂量阿糖胞苷或低甲基化药物治疗可带来临床获益，这已在 3 期研究中得到证实。随着基于 VEN 的治疗方案在 AML 患者中的应用增加，一个相关问题是对于已达到持续缓解的患者，是否可以安全地停止治疗。我们假设，一部分选择停止治疗的患者可能会从无治疗缓解（TFR）期获益，而无需无限期治疗。我们报告了 29 例患者的回顾性结果，这些患者在接受基于 VEN 的治疗后缓解至少 12 个月，其中 55%的患者继续治疗直至疾病进展，45%的患者选择性停止治疗（STOP）。随访时间超过 5 年，我们观察到 STOP 队列中有 45.8 个月的中位 TFR，数据截止时仍有>50%的患者处于持续缓解状态。两组患者的复发风险、无复发生存期和总生存期相似。在该队列中，有利于持续 TFR 的因素包括诊断时存在 NPM1 和/或 IDH2 突变、无微小残留病的完全缓解，以及在停止治疗前至少接受 12 个月的基于 VEN 的联合治疗。
