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Despite recent advances in the treatment of adult acute myeloid leukemia (AML), the clinical outcome of patients continues to be unsatisfactory especially among older patients, those with a high-risk profile, and in the relapsed/refractory setting. For this reason, recent clinical trials have explored novel therapeutic agents either used alone or in combination with intensive chemotherapy or low-intensity treatments. The current paper reviews the clinical development of monoclonal antibody-based therapies in AML, their current status and phases 2 and 3 prospective trials. Monoclonal antibody-based therapies demonstrated efficacy and tolerability in several clinical trials, especially when used in combination either with ‘3 + 7’ chemotherapy or with low-intensity treatments. Additional studies are needed to determine new antigens for antibody-based therapies that target leukemia stem cells and spare normal hematopoiesis. Phase 2 and 3 additional clinical trial data are needed to assess the promise of first trials, especially regarding chimeric antigen receptor T cells redirected against myeloid antigens and immune checkpoint inhibitor therapies.

Antibody-based therapy for acute myeloid leukemia: a review of phase 2 and 3 trials