Successful treatment of relapsed KMT2A rearranged AML with central nervous system involvement using revumenib in combination with venetoclax and azacitidine: A case report

Abstract

This case report describes the successful treatment of a patient with acute monocytic leukemia (AML-M5), positive for the KMT2A::AFDN fusion gene, who relapsed and developed central nervous system involvement after allogeneic hematopoietic stem cell transplantation. The patient was a 32-year-old male initially treated with conventional chemotherapy followed by transplantation; however, he experienced relapse with AML infiltration of the CNS. Based on the MLL abnormality, salvage therapy was administered using the menin inhibitor Revumenib combined with venetoclax and azacitidine, followed by donor lymphocyte infusion (DLI). After one course of treatment, he achieved minimal residual disease (MRD)-negative status in both bone marrow and cerebrospinal fluid, with sustained complete remission. The main adverse effects observed were grade IV myelosuppression and grade II gastrointestinal symptoms. This case indicates that the salvage strategy incorporating Revumenib, venetoclax, azacitidine, and DLI not only exhibits potential for achieving deep remission in KMT2A-rearranged AML but also aids in controlling central nervous system infiltration, thereby providing a novel therapeutic direction for patients with relapsed AML. Further studies involving larger sample sizes are required to assess the long-term survival benefits of this strategy.