Meet-the expert: Treatment of AML

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Primario presso la divisione di Ematologia dell’Ospedale Cardarelli di Napoli

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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.


Felicetto Ferrara
Felicetto Ferrara
Primario presso la divisione di Ematologia dell’Ospedale Cardarelli di Napoli

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