Advances in targeted therapy for acute myeloid leukaemia

Sabine Kayser, Mark J. Levis

Research output: Contribution to journalReview articlepeer-review

41 Scopus citations


In the past few years, research in the underlying pathogenic mechanisms of acute myeloid leukaemia (AML) has led to remarkable advances in our understanding of the disease. Cytogenetic and molecular aberrations are the most important factors in determining response to chemotherapy as well as long-term outcome, but beyond prognostication are potential therapeutic targets. Our increased understanding of the pathogenesis of AML, facilitated by next-generation sequencing, has spurred the development of new compounds in the treatment of AML, particularly the creation of small molecules that target the disease on a molecular level. Various new agents, such as tyrosine kinase inhibitors, immune checkpoint inhibitors, monoclonal or bispecific T-cell engager antibodies, metabolic and pro-apoptotic agents are currently investigated within clinical trials. The highest response rates are often achieved when new molecularly targeted therapies are combined with standard chemotherapy. Presented here is an overview of novel therapies currently being evaluated in AML.

Original languageEnglish (US)
Pages (from-to)484-500
Number of pages17
JournalBritish journal of haematology
Issue number4
StatePublished - Feb 2018


  • BCL-2 inhibitor
  • acute myeloid leukaemia
  • immune-checkpoint inhibitors
  • targeted therapy
  • tyrosine kinase inhibitors

ASJC Scopus subject areas

  • Hematology


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