Section of Hematology, Department of Internal Medicine, Yale University School of Medicine and Yale Cancer Center, New Haven, Connecticut, USA.
Curr Opin Hematol. 2021 Mar 1;28(2):110-121. doi: 10.1097/MOH.0000000000000637.
Maintenance therapy for acute myeloid leukemia (AML) has been studied for decades with mixed results. However, the application of modern agents has renewed interest and the recent data from randomized trials has provided evidence for the use of maintenance therapy in certain populations of AML patients.
Unselected patients are unlikely to benefit from maintenance therapy as has been previously and consistently demonstrated. The increasing availability of newer and targeted agents like oral hypomethylating agents, protein modifiers, as well as FLT3, IDH1/2 BCL-2 and immune checkpoint inhibitors have restoked interest in maintenance therapy for which randomized, placebo-controlled trials have recently demonstrated benefits, including in the post-transplant setting. Patients with high-risk disease, suboptimal consolidation or remission associated with measurable residual disease (MRD) appear to be beneficiaries of this strategy. The influence of MRD status and the platform by which it is measured are important factors in the current understanding of when maintenance therapy works and how future studies should be designed.
The recent positive findings in support of maintenance therapy for certain AML patient populations are practice changing and bolster the need for properly designed, randomized studies using unified and standardized MRD techniques.
急性髓系白血病(AML)的维持治疗已研究了数十年,但结果喜忧参半。然而,现代药物的应用重新引起了人们的兴趣,最近的随机试验数据为 AML 患者的某些人群使用维持治疗提供了证据。
与之前一致的研究结果表明,未选择的患者不太可能从维持治疗中获益。新的和靶向药物(如口服低甲基化剂、蛋白调节剂,以及 FLT3、IDH1/2、BCL-2 和免疫检查点抑制剂)的可用性不断增加,重新激起了人们对维持治疗的兴趣,最近的随机、安慰剂对照试验表明该疗法具有益处,包括移植后环境中。高危疾病、巩固或缓解不理想且与可测量残留疾病(MRD)相关的患者似乎是该策略的受益者。MRD 状态及其测量平台的影响是当前理解维持治疗何时有效的重要因素,也是未来研究设计的重要因素。
总之,最近支持某些 AML 患者人群进行维持治疗的阳性发现改变了临床实践,并且需要使用统一和标准化的 MRD 技术进行精心设计、随机研究。