Topline Presentation Points 

Overview of Acute myeloid leukemia (AML)  

  • Aggressive hematological malignancy with globally poor clinical outcomes  
  • Median age at diagnosis is 68 years old 
  • Older patients with AML ineligible for intensive chemotherapy have a particularly poor outcome and novel therapies are needed 
  • AML is a heterogeneous disease characterized by recurrent genetic alterations potentially targetable for novel therapeutic approaches  


New drug approval for AML in the recent years 

Richard-Carpentier G. & DiNardo C. Hematology. 2019 (Updated figure) 

Recurrent genetic alterations in AML 

  • Potential targets 
    • FLT3 mutations (25-30%)
    • IDH1/2 mutations (15-20%)
    • NPM1 mutations (25-30%)
    • TP53 mutations (5-10%)
    • KMT2A rearrangements (5-10%)

Evaluation of eligibility for intensive chemotherapy  

Patient-related factors

Is patient fit enough to undergo intensive chemotherapy?

Disease-related factors

What is the likelihood of achieving complete remission?

Global treatment plan
Goals of care

Goal for cure or disease control?
Is there a plan for HSCT?

Treatment Options 

  • Non-intensive treatments for patients with AML 
  • BCL-2 inhibition in AML 
    • Venetoclax-based regimens in AML 
      • Remission rates with venetoclax-based regimens 
      • Preventive measures for tumor lysis syndrome 
      • Cytopenia and dose adjustments 
  • Glasdegib in combination with LDAC 
  • Pathophysiology of IDH1/2-mutated AML 
    • Targeting IDH1/2 mutations in AML 
  • FLT3 mutations in AML 
    • Gilteritinib for relapsed/refractory FLT3-mut AML 


Future Perspectives 

  • Targeted agents available for patients with relapsed / refractory AML 
  • Utilization of lesser intensity regimens in younger patients with AML or older patients who are eligible for intensive chemotherapy 
  • Evaluation of triplet-drug regimens in older patients with AML 
  • Integration of FLT3 inhibitors 
  • Integration of IDH1/2 inhibitors 
  • Identification of subsets of patients for treatment-free remission?  
  • Very-high risk AML remain an urgent unmet need (t-AML, TP53 mut, CK) 
  • Anti-CD47 antibodies (e.g. magrolimab) 
  • Checkpoint inhibitors (e.g. anti-TIM3 antibodies)