Maintenance therapy for acute myeloid leukemia: sustaining the pursuit for sustained remission

Curr Opin Hematol. 2021 Mar 1;28(2):110-121. doi: 10.1097/MOH.0000000000000637.


Purpose of review: 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.

Recent findings: 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.

Summary: 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.

Trial registration: NCT02532231 NCT02275533 NCT01096602 NCT00665002 NCT01686334 NCT02632708 NCT03515512 NCT03728335 NCT03564821 NCT03466294 NCT04102020.

Publication types

  • Review

MeSH terms

  • Antineoplastic Agents / administration & dosage
  • Antineoplastic Agents / adverse effects
  • Antineoplastic Agents / therapeutic use*
  • Antineoplastic Combined Chemotherapy Protocols / adverse effects
  • Antineoplastic Combined Chemotherapy Protocols / therapeutic use*
  • Biomarkers, Tumor
  • Clinical Decision-Making
  • Combined Modality Therapy / adverse effects
  • Combined Modality Therapy / methods
  • Disease Management
  • Disease Susceptibility
  • Hematopoietic Stem Cell Transplantation
  • Humans
  • Leukemia, Myeloid, Acute / diagnosis
  • Leukemia, Myeloid, Acute / etiology
  • Leukemia, Myeloid, Acute / therapy*
  • Maintenance Chemotherapy
  • Randomized Controlled Trials as Topic
  • Remission Induction
  • Treatment Outcome


  • Antineoplastic Agents
  • Biomarkers, Tumor

Associated data