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Post-Transplant Maintenance Therapy for Patients with Acute Myeloid Leukemia: Current Approaches and the Need for More Trials

Authors Assi R, Masri N, Abou Dalle I, El-Cheikh J, Bazarbachi A

Received 23 October 2020

Accepted for publication 5 January 2021

Published 26 January 2021 Volume 2021:12 Pages 21—32

DOI https://doi.org/10.2147/JBM.S270015

Checked for plagiarism Yes

Review by Single anonymous peer review

Peer reviewer comments 2

Editor who approved publication: Dr Martin Bluth


Rita Assi,1 Nohad Masri,1 Iman Abou Dalle,2 Jean El-Cheikh,2 Ali Bazarbachi2

1Division of Hematology-Oncology, Lebanese American University and Lebanese American University Medical Center-Rizk Hospital, Beirut, Lebanon; 2Division of Hematology-Oncology, American University of Beirut Medical Center, Beirut, Lebanon

Correspondence: Ali Bazarbachi
Department of Internal Medicine, American University of Beirut, Medical Center, P.O. Box 113-6044, Beirut, Lebanon
Email bazarbac@aub.edu.lb

Abstract: Relapse rates following allogeneic stem cell transplantation for acute myeloid leukemia remain unacceptably high and a major cause of death. Maintenance therapies post-transplant administered either to patients with impending relapse or at high risk of relapse could present a strategy to improve survival and overall outcomes. With the increasing use of molecular and genomic characterization of the disease, more novel therapies became available as maintenance strategies. These options were, however, hindered by excessive toxicities, mostly hematologic, especially with the use of myeloablative conditioning regimens. Several key questions have also emerged including the efficacy of these therapies, the duration of maintenance, as well as the potential modulation of the graft and the immune microenvironment. These issues are further complicated by the paucity of well-designed prospective randomized clinical trials evaluating these agents. Future directions in this field should include better risk stratification and patient selection based on assays of minimal residual disease, as well as the incorporation of novel targets and pathways of leukemogenesis. In this article, we highlight the current evidence behind the use of post-transplant maintenance therapy, the optimal patient and disease selection, as well as the challenges faced by these strategies in an area that remains quite controversial. We will focus on therapies targeting leukemia stem cells that directly or indirectly modulate the allografted immune microenvironment and augment the graft-versus-leukemia impact.

Keywords: AML, maintenance, relapse, target, MRD

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