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Meet-the expert: Treatment of AML

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Questions and Answers with Prof. G.J. Ossenkoppele



Which are the today most important unmet clinical needs in AML?

  • Improving CR rate in elderly AML.
  • Preventing relapse in elderly and younger AML.
  • Approval of new promising drugs.
  • Determine who is fit for intensive treatment.

Which are the most promising new drugs for the treatment of AML?

  • FLT3-ITD inhibitors (midostaurin, quizartib).
  • Hypomethylating agents (HMAs) and new “second generation” HMAs.
  • Vosaroxin (topoisomerase II inhibitor).
  • Bcl-2 inhibitors (venetoclax).
  • CRM 1 inhibition (selinexor).

Which were the most exciting news from the recent ASH Meeting ?

  • Positive outcome  in the Phase III  RATIFY study in FLT3-ITD/TKD mutated patients of midostaurin added to standard therapy.
  • Promising combinations in addition to HMAs (i.e. azacytidine plus pracinostat).

How far is a really targeted therapy for AML (apart APL) ?

  • Not so close because of the heterogeneity of AML; FLT3ITD is the first example although midostaurin is not very specific.

Has immunotherapy a future in AML ?

  • Absolutely, allogeneic stem cell transplantation has already been an example for many years. But new modalities are emerging:
    • monoclonal antibodies (anti-CD33, anti-CD123, and anti-CD47)
    • immunoconjugates
    • BiTEs (anti-CD33, anti-CLEC12A)
    • CARTs will soon be explored in AML
    • Immune checkpoint inhibitors.

Gert J.Ossenkoppele, M.D., PhD., is Professor of Hematology at the VU University Medical Center, Amsterdam, The Netherlands.


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