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

• DOI: 10.1007/s00277-025-06312-2
• Publication Date: 2025-03-18T09:09:52Z
• Authors: Kun Yang; Beibei Yang; Yali Zhou; Qiuying Huang; Xiaolin Yin
• Keywords: Hematology; Refractory (planetary science)

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