Venetoclax plus azacitidine as successful bridging therapy to second transplantation for post-transplant extramedullary relapse of acute myeloid leukemia

Abstract

A 43-year-old man was diagnosed with FLT3-ITD-positive acute myeloid leukemia (AML). Although initial intensive chemotherapy failed to achieve remission, he attained complete remission (CR) with gilteritinib and subsequently underwent allogeneic hematopoietic stem cell transplantation (allo-HSCT) from an unrelated donor. Maintenance therapy with gilteritinib was discontinued due to graft-versus-host disease. Eighteen months post-transplant, he relapsed with an extramedullary mass in the psoas muscle and malignant pleural effusion, but no bone marrow involvement. Salvage therapy with venetoclax plus azacitidine (Ven+AZA) resulted in the complete disappearance of the extramedullary lesion, achieving a second CR. The patient then successfully underwent a second allo-HSCT using cord blood. He has remained in CR with additional post-transplant Ven+AZA therapy. Ven+AZA can be an effective and safe bridge-to-transplant strategy for post-HSCT extramedullary relapse of AML, even without concomitant radiotherapy.