Feasibility of a prospective real-time monitoring system capturing real-world data from elderly patients with acute myeloid leukemia

Real-world data (RWD) are increasingly applied as external controls in phase II-III clinical trials. However, retrospectively monitored RWD may suffer from limitations, including data availability and quality. Prospective monitoring of RWD may potentially overcome such limitations. We addressed the feasibility of an automated monitoring system for prospectively capturing RWD of older patients with acute myeloid leukemia (AML) and compared them with patients in the control arm of the HOVON-155 (H155) AML trial. The system extracted predefined data from patients receiving nonintensive therapy in 10 Dutch hospitals. Patients in the RWD cohort were further selected for comparison with patients in the H155-trial by applying the trial eligibility criteria. Caliper propensity score matching (PSM) of patients in the RWD cohort with patients in the H155-control arm was performed based on age, European LeukemiaNet 2017, and Hematopoietic Cell Transplant-Comorbidity Index. Between 2020 and 2025, 193 patients in the RWD cohort were identified, of whom 120 had not participated in the H155-trial but did meet its eligibility criteria. Patients in the H155 control arm (n = 70) and patients in the RWD cohort (n = 120) differed in baseline parameters. Using PSM, 51 patients in the RWD cohort were compared with 51 patients in the H155-control arm. The 100-day mortality proportion rate was 37.3% versus 27.5% (P = 0.40) among patients in the H155 control arm and patients in the RWD cohort, respectively. Two-year overall survival was 18% ± 5% versus 18% ± 5% (hazard ratio 1.05, 95% confidence interval 0.69-1.62, P = 0.81). Bleeding and bacteremia appeared to occur more frequently among patients in the H155 control arm, whereas the frequency of other adverse events was comparable. A prospective RWD monitoring system was efficiently applied in 10 Dutch hospitals. Although patient characteristics initially differed from the H155 control arm, the outcome of the RWD cohort appeared similar following PSM. Prospective monitoring of RWD seems feasible but matching is needed for future use in clinical studies.