Real-World Treatment Patterns and Outcomes Among Patients With Newly Diagnosed AML in the United States

PURPOSE: The US Food and Drug Administration has approved multiple new treatments for AML since 2017 based on demonstrated efficacy and safety in clinical trials. However, the uptake of new treatments and effectiveness in the routine clinical practice are largely unknown. METHODS: A retrospective observational study was conducted to examine first-line therapy in patients with AML who were treated at institutions providing data to the COTA database. Information on demographics, disease characteristics, treatments, and outcomes was collected and analyzed descriptively. RESULTS: Among the 2,516 patients eligible for inclusion in our analysis, cytarabine-based intensive chemotherapy (IC w/cytara) was the most commonly used treatment, followed by hypomethylating agents (HMA) with venetoclax (HMA+ven), HMA alone, and investigational treatment. Use of HMA+ven increased over time since its approval in 2018, whereas use of IC w/cytara and HMA alone decreased. Complete remission rates were 45% overall and highest in patients treated with IC w/cytara (61%) or investigational therapy (62%). CONCLUSION: Overall survival was highest for patients treated with IC w/cytara, although differences in baseline patient characteristics make direct comparison infeasible. Uptake of HMA+ven as a first-line treatment for patients with AML has been more rapid compared with other newly approved therapies. Outcomes for patients treated in routine practice were similar to those seen in clinical trials. Further research may be helpful in characterizing real-world effectiveness and safety in specific subpopulations and disease subtypes.