Acute myeloid leukemia (AML) is a heterogenous disease associated with multiple chromosomal abnormalities and gene mutations. This diversity contributes to differential responses to chemotherapy regimens, and consequently survival rates, meaning treating AML presents a challenge for clinicians. 1Current treatment protocols for AML involve induction chemotherapy, usually cytarabine and an anthracycline, followed by consolidation for favorable-risk patients or allogeneic hematopoietic stem cell transplant (allo-HSCT) for adverse-risk patients. 2Allo-HSCT is potentially curative in eligible patients, but many patients are unable to receive transplants due to existing comorbidities, a lack of suitable donor, or the risk of complications. 3
Over the last two decades, few drugs have been approved for the treatment of AML, however increased understanding of the leukemic genome has allowed the development of targeted therapeutic agents. 2,3Between April 2017 and November 2018, eight new drugs in various different classes were approved, including: enasidenib, ivosidenib, midostaurin, glasdegib, venetoclax, and gilteritinib. 4An overview of mutations that can be targeted in AML is available on the AML Global Portal (AGP).
Some of these targeted agents are being investigated in novel combination therapies, as different lines of AML treatment, and some as monotherapy compared to standard chemotherapy. This article provides a summary of AGP content relating to novel agent-containing combinations to date and introduces the new editorial theme of ‘Novel combination therapies using targeted agents.’
Expert opinions: What are targeted therapies and how might they impact the treatment of AML?
The AGP were pleased to speak to Andrew Weiduring the European School of Haematology(ESH) Translational Research Conference on AML, 2019, about how targeted therapies will be used in the future treatment of AML. In this interview, available below, Andrew Wei discusses whether these targeted agents should be used in combination or sequentially.