Oral azacitidine as time-limited, PCR-dependent maintenance in acute myeloid leukemia with inv(16): two case reports

Core-binding factor acute myeloid leukemia (CBF-AML) in fit patients is treated with curative-intent induction chemotherapy followed by 3-4 cycles of high-dose cytarabine (HiDAC) consolidation. No standard therapy exists for patients with contraindications to HiDAC consolidation. Oral azacitidine is approved as indefinite maintenance therapy for intermediate/high risk AML but has not been studied in CBF-AML, nor in a time-limited fashion. We present two patients with CBF-AML who achieved morphologic remission after induction but had medical (case 1) or psychiatric (case 2) contraindications to HiDAC, therefore receiving oral azacitidine in a minimal residual disease (MRD)-guided, time-limited fashion. In case 1, drug-induced liver injury precluded HiDAC therapy; after four cycles, CBFB::MYH11 became PCR-undetectable, and azacitidine continued for a year. In case 2, a suicide attempt related to anticipated hospitalization precluded HiDAC; azacitidine was administered intermittently. Both patients remain MRD-negative 1.5 years post-treatment suggesting time-limited, MRD-guided azacitidine as a potential alternative to HiDAC consolidation.