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<p>Nucleoporin 98 (NUP98) fusion oncoproteins are associated with various hematologic malignancies. Acute myeloid leukemia (AML) with <i>NUP98-NSD1</i> typically co-occurs with <i>FLT3-ITD</i> mutations, exhibiting poor initial responses to traditional chemotherapy. This case report describes a relapsed and refractory AML case co-expressing <i>NUP98/NSD1</i> and <i>FLT3/ITD</i> 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 <i>NUP98-NSD1+/FLT3-ITD +</i> AML. This report highlights the potential synergy between selinexor and established AML therapies, suggesting a promising approach to improve outcomes for refractory AML patients.</p>

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

  • 期刊论文

  • 出版模式：

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  • 发表日期：

    2025年3月18日

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  • Kun Yang,
  • Beibei Yang,
  • Yali Zhou,
  • Qiuying Huang,
  • Xiaolin Yin

摘要

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.