Liposomal mitoxantrone, venetoclax and azacitidine in relapsed/refractory acute myeloid leukaemia and early induction failure: a single-centre real-world study.

INTRODUCTION

Patients with relapsed/refractory acute myeloid leukaemia have poor outcomes, particularly after failure of venetoclax-based therapy.

METHODS

We retrospectively analysed 16 adults with relapsed/refractory AML or early induction failure who received liposomal mitoxantrone, venetoclax and azacitidine (MVA) at a single centre between March 2024 and August 2025. The primary endpoint was composite complete remission (CRc), defined as complete remission or complete remission with incomplete haematological recovery.

RESULTS

Median age was 58.5 years. Eight patients had relapsed AML, five had refractory AML and three had early induction failure; nine had previously failed venetoclax-based therapy. CRc was achieved in 10/16 patients (62.5%; 95% CI, 35.4% -84.8%), and the overall response rate was 14/16 (87.5%; 95% CI, 61.7% -98.4%). Among patients with prior venetoclax failure, 7/9 achieved CRc (77.8%; 95% CI, 40.0% -97.2%). Four patients proceeded to allogeneic haematopoietic stem cell transplantation (allo-HSCT). Grade 3 -4 neutropenia and thrombocytopenia occurred in 16/16 and 14/16 patients, respectively. No treatment-related deaths occurred during MVA therapy.

DISCUSSION: These findings suggest that MVA had clinical activity as a salvage regimen for difficult-to-treat AML, including patients with prior venetoclax failure, with expected myelosuppression and potential bridging utility before allo-HSCT. Prospective studies are needed to confirm efficacy and define patient selection.