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      Novel Therapeutic Targets in Acute Myeloid Leukemia (AML)

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          Abstract

          Purpose of Review

          This review seeks to identify and describe novel genetic and protein targets and their associated therapeutics currently being used or studied in the treatment of acute myeloid leukemia (AML).

          Recent Findings

          Over the course of the last 5–6 years, several targeted therapies have been approved by the FDA, for the treatment of both newly diagnosed as well as relapsed/refractory AML. These novel therapeutics, as well as several others currently under investigation, have demonstrated activity in AML and have improved outcomes for many patients.

          Summary

          Patient outcomes in AML have slowly improved over time, though for many patients, particularly elderly patients or those with relapsed/refractory disease, mortality remains very high. With the identification of several molecular/genetic drivers and protein targets and development of therapeutics which leverage those mechanisms to target leukemic cells, outcomes for patients with AML have improved and continue to improve significantly.

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          Most cited references78

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          Genomic Classification and Prognosis in Acute Myeloid Leukemia

          New England Journal of Medicine, 374(23), 2209-2221
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            Azacitidine and Venetoclax in Previously Untreated Acute Myeloid Leukemia

            Older patients with acute myeloid leukemia (AML) have a dismal prognosis, even after treatment with a hypomethylating agent. Azacitidine added to venetoclax had promising efficacy in a previous phase 1b study.
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              Midostaurin plus Chemotherapy for Acute Myeloid Leukemia with a FLT3 Mutation

              Patients with acute myeloid leukemia (AML) and a FLT3 mutation have poor outcomes. We conducted a phase 3 trial to determine whether the addition of midostaurin - an oral multitargeted kinase inhibitor that is active in patients with a FLT3 mutation - to standard chemotherapy would prolong overall survival in this population.
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                Author and article information

                Contributors
                mwysota@montefiore.org
                marina.konopleva@einsteinmed.edu
                Journal
                Curr Oncol Rep
                Curr Oncol Rep
                Current Oncology Reports
                Springer US (New York )
                1523-3790
                1534-6269
                19 March 2024
                19 March 2024
                2024
                : 26
                : 4
                : 409-420
                Affiliations
                [1 ]Department of Oncology, Montefiore Medical Center, ( https://ror.org/044ntvm43) 111 East 210 Street, Bronx, NY 10467 USA
                [2 ]Montefiore Medical Center/Albert Einstein College of Medicine, Albert Einstein College of Medicine, Jack and Pearl Resnick Campus, Ullmann Building, ( https://ror.org/05cf8a891) 1300 Morris Park AvenueRoom 915, Bronx, NY 10461 USA
                [3 ]Albert Einstein College of Medicine, ( https://ror.org/05cf8a891) Bronx, NY USA
                Article
                1503
                10.1007/s11912-024-01503-y
                11021231
                38502417
                fc608f41-a395-490d-abc8-27973b3f442a
                © The Author(s) 2024

                Open Access This article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made. The images or other third party material in this article are included in the article's Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article's Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this licence, visit http://creativecommons.org/licenses/by/4.0/.

                History
                : 1 February 2024
                Categories
                Review
                Custom metadata
                © Springer Science+Business Media, LLC, part of Springer Nature 2024

                Oncology & Radiotherapy
                acute myeloid leukemia,novel therapeutics,molecular targets,aml,immune therapy

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