Naval G. Daver, MD, from the University of Texas MD Anderson Cancer Center, Houston, TX, discusses the immune checkpoint inhibitor landscape in acute myeloid leukemia (AML) patients at the American Society of Hematology (ASH) Congress 2016 in San Diego, CA. He explains that a number of immune checkpoint inhibitor drugs are being used in solid tumors and showing promising results, and introduces one of the first Phase IB/II studies looking at the applicability of immune checkpoint inhibitor drugs in AML, combining azacytidine with nivolumab in salvage therapy for patients who had received a median of 2 prior treatments (NCT02397720). The endpoints assessed were overall response, including complete remission (CR), complete remission with incomplete marrow recovery (CRi), and partial remission (PR), as well as durability of response and overall survival (OS). Dr Daver explains that the overall response rate was around 35% with combination treatment, compared to the expected historical response rate of 15 to 18% for similar patients treated with azacytidine alone. More importantly, a very durable response with a median of 9 months was maintained in those patients who had a response, and ongoing correlative studies show that patients with high CD4 and CD8 T-cell populations in the bone marrow were more likely to respond. Additionally, patients with a response showed an increased in bone marrow T-cell populations with treatment, as would be expected with a favorable immune infiltrate. Looking forward, Dr Daver describes that this combination immunotherapy is now being expanded into a frontline setting, as well as including additional checkpoint inhibitors such as ipilimumab.