Fixed-time treatment of elderly patients with acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome (MDS) using hypomethylating agents and venetoclax – a case series

Standard therapy for elderly patients with acute myeloid leukemia (AML) consists of hypomethylating agents (HMAs) combined with venetoclax (VEN), administered long term until disease progression. However, several retrospective studies have shown that patients in complete remission (CR) who discontinued treatment electively or because of poor tolerance did not experience a negative impact on relapse-free or overall survival. Therefore, after obtaining informed consent, we offered treatment discontinuation to patients in CR after six months of HMA-VEN therapy. Here, we report the outcomes of seven consecutively treated patients who achieved CR between May 2021 and June 2025 and opted for discontinuation of therapy. Five of the seven patients showed clearance of somatic mutations to < 1% and resolution of cytogenetic abnormalities. Six of the seven patients remain in continuous remission for 7-50 months. One patient with FLT3-ITD experienced relapse after 14 months and achieved a second hematologic remission after resuming HMA-VEN therapy. These findings suggest that treatment-free remission after time-limited HMA-VEN therapy may be a feasible approach in selected patients.