Improvement in cutaneous disease with menin inhibitor monotherapy in KMT2A rearranged AML

Abstract

We report this case of a 48-year-old man with multiple relapsed KMT2A–MLLT10 rearranged acute myeloid leukaemia (AML). He was initially treated with intensive chemotherapy followed by a reduced intensity (RIC) haploidentical sibling transplant. At relapse, he presented with extensive cutaneous extramedullary disease without bone marrow involvement. Salvage chemotherapy with FLAG-IDA (fludarabine, cytarabine, idarubicin and G-CSF) followed by donor lymphocyte infusion (DLI) achieved a brief remission, but there was widespread recurrence of his skin lesions (left panel).

His extramedullary disease continued to progress (left panel), and he developed minimal residual disease (MRD) positivity without any morphologic disease in his bone marrow. At this point, he was commenced on monotherapy with a menin inhibitor.¹ Within the first cycle of therapy, there was a marked reduction in the burden of extramedullary disease. After 8 weeks of therapy, there was a near resolution of his cutaneous disease (right panel). Alongside this, menin inhibition resulted in achievement of MRD negativity in his bone marrow.