Novel agents for acute myeloid leukemia

Mario Luppi, Francesco Fabbiano, Giuseppe Visani, Giovanni Martinelli, Adriano Venditti

Research output: Contribution to journalReview articlepeer-review


Acute myeloid leukemia (AML) is a complex hematological disease characterized by genetic and clinical heterogeneity. Recent advances in the understanding of AML pathogenesis have paved the way for the development of new agents targeting specific molecules or mechanisms that contribute to finally move beyond the current standard of care, which is “3 + 7” regimen. In particular, new therapeutic options such as targeted therapies (midostaurin and enasidenib), monoclonal antibodies (gemtuzumab ozogamicin), and a novel liposomal formulation of cytarabine and daunorubicin (CPX-351) have been recently approved, and will be soon available for the treatment of adult patients with AML. In this review, we will present and describe these recently approved drugs as well as selected novel agents against AML that are currently under investigation, and show the most promising results as monotherapy or in combination with chemotherapy. The selection of these emerging treatments is based on the authors’ opinion.

Original languageEnglish
Article number429
Issue number11
Publication statusPublished - Nov 9 2018


  • Acute myeloid leukemia
  • CPX-351
  • Enasidenib
  • FLT3
  • Gemtuzumab ozogamicin
  • Midostaurin
  • Palliative care
  • “7 + 3” regimen

ASJC Scopus subject areas

  • Oncology
  • Cancer Research


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