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New Treatment Options for Acute Myeloid Leukemia in 2019
Marco Cerrano1,2, Raphael Itzykson3,4,5
1Department of Hematology, Hopital Saint-Louis, Assistance Publique Hopitaux de Paris, Paris Diderot University, Paris, France.
Purpose Of Review:
The extensive genomic characterization of acute myeloid leukemia (AML) led to the identification of a vast number of potential therapeutic targets. We review relevant data that have led to recent approval of new targeted therapies in AML and discuss the most promising drugs currently in development in this disease.
Recent Findings:
New formulations of cytotoxic agents, namely CPX-351 and gemtuzumab ozogamicin, improve the outcome of defined subgroup of patients. Midostaurin added to intensive chemotherapy is approved in FLT3-mutated AML. More selective FLT3 inhibitors and the IDH inhibitors enasidenib and ivosidenib have shown significant single agent activity in the relapsed setting, and preliminary results of combination strategies are encouraging. The addition of the BCL2 inhibitor venetoclax appears to markedly improve the results of hypomethylating agents. The therapeutic armamentarium of AML now includes novel cytotoxic drugs, drugs targeting recurrent oncogenes, or functional vulnerabilities of leukemic cells. Further work is required to optimize their integration to the current framework of AML management, including allogeneic stem cell transplantation.
Insights
New targeted therapies are transforming acute myeloid leukemia (AML) treatment. Approved drugs and promising agents, including FLT3 and IDH inhibitors, offer new hope for AML patients.
Area of Science:
- Hematology
- Oncology
- Genomics
Background:
- Acute myeloid leukemia (AML) genomic characterization has identified numerous therapeutic targets.
- Recent advancements have led to the approval of novel targeted therapies for AML.
Purpose of the Study:
- To review data supporting new targeted therapies in AML.
- To discuss promising drugs currently in development for AML.
Main Methods:
- Review of clinical trial data and published literature.
- Analysis of recent drug approvals and ongoing clinical investigations.
Main Results:
- New formulations of cytotoxic agents (CPX-351, gemtuzumab ozogamicin) improve outcomes in specific AML subgroups.
- Midostaurin is approved for FLT3-mutated AML when added to chemotherapy.
- Selective FLT3 inhibitors and IDH inhibitors (enasidenib, ivosidenib) show efficacy in relapsed AML.
- BCL2 inhibitor venetoclax combined with hypomethylating agents shows promising results.
Conclusions:
- The AML therapeutic landscape now includes novel cytotoxic drugs, oncogene-targeted agents, and drugs targeting leukemic vulnerabilities.
- Further research is needed to optimize the integration of these new therapies into AML management, including allogeneic stem cell transplantation.
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