Promising activity of Selinexor in the treatment of a patient with refractory NUP98-NSD1+/FLT3-ITD + acute myeloid leukemia.

Nucleoporin 98 (NUP98) fusion oncoproteins are associated with various hematologic malignancies. Acute myeloid leukemia (AML) with NUP98-NSD1 typically co-occurs with FLT3-ITD mutations, exhibiting poor initial responses to traditional chemotherapy. This case report describes a relapsed and refractory AML case co-expressing NUP98/NSD1 and FLT3/ITD after matched sibling haplo-identical allogeneic hematopoietic stem cell transplantation, achieving molecular remission with a salvage therapy combining selinexor, venetoclax, and azacitidine. To our knowledge, this is the first report demonstrating the effectiveness of this combination therapy for relapsed/refractory NUP98-NSD1+/FLT3-ITD + AML. This report highlights the potential synergy between selinexor and established AML therapies, suggesting a promising approach to improve outcomes for refractory AML patients.