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Potential therapeutic options for MDS, available but not approved by EMA

This section is under developement and will be expanded over time.

Please click on a heading to view information on that therapy. 

Azacytidine in lower risk MDS

Aza is not approved for treatment of lower-risk MDS, but has been evaluated in a few minor studies, mainly on transfusion dependent patients. Transfusion-independency can be achieved in a minor subset of the patients and the clinical value seems to be limited  to this cohort of patients. Survival benefit has not been demonstrated.

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Decitabine (DAC) in higher-risk MDS

  • DAC can induce responses in MDS but has not demonstrated a clear survival advantage over conventional treatment in MDS, and is not EMA approved for this indication. It is however approved for AML.
  • A 10 day regimen of DAC may give high response rates in patients with AML or MDS  with TP53 mutation (Welch et al). These results  however need  to be evaluated in controlled studies.

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