Acute myeloid leukaemia

Overview 
Theory 
Diagnosis 
Management 
Follow up 
Resources 

Decitabine/cedazuridine

Decitabine/cedazuridine (oral fixed-dose combination) is approved in Europe for patients with newly diagnosed acute myeloid leukaemia (AML) who are ineligible for standard induction chemotherapy. In a phase 3 study, decitabine/cedazuridine demonstrated pharmacokinetic equivalence to standard intravenous decitabine in AML patients not suitable for standard induction chemotherapy.[120]

Guadecitabine

Guadecitabine is a second-generation hypomethylating agent. It is formulated as a dinucleotide of the drug decitabine with deoxyguanosine, which increases the half-life of decitabine.[121] In a phase 3 study of patients with untreated AML who were unsuitable for intensive chemotherapy, guadecitabine did not demonstrate improved survival compared with a preselected treatment (e.g., azacitidine, decitabine, or low-dose cytarabine).[122]

Crenolanib

Crenolanib is a second-generation tyrosine kinase inhibitor with activity against FLT3-ITD- and FLT3-TKD-mutated AML. Results from one small study suggest that crenolanib plus intensive chemotherapy results in high response rates, with acceptable toxicity, in AML patients.[123] Longer-term data (30 FLT3-mutated AML patients, median follow-up 5 years) indicate that crenolanib may be well tolerated in the post-transplant setting.[124] The US Food and Drug Administration (FDA) and European Medicines Agency (EMA) have granted orphan drug designation to crenolanib for the treatment of AML.

Flotetuzumab

Flotetuzumab is a dual-affinity re-targeting (DART) antibody-based molecule that recognises CD123 with CD3. Leukaemic stem cells are characterised by high levels of CD123 expression. Flotetuzumab is currently being evaluated in phase 1/2 studies of patients with relapsed or refractory AML.[125] The FDA has granted orphan drug status to flotetuzumab.

Gilteritinib plus venetoclax and azacitidine

In a phase 1/2 study, the addition of gilteritinib to venetoclax plus azacitidine resulted in a high rate of complete remission/complete remission with incomplete haematological recovery (96%) and a deep FLT3 molecular response in patients with newly diagnosed FLT3-mutated AML.[126]

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