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Comprehensive mutational profiling and clinical outcome of adult acute myeloid leukemia patients with NUP98 rearrangement - PubMed

2 hours ago
  • #NUP98 rearrangement
  • #acute myeloid leukemia
  • #clinical outcomes
  • Study analyzes 95 adult AML patients with NUP98 rearrangements, characterized by young median age (50 years), high WBC count, and frequent FLT3-ITD (48%) and WT1 mutations (34%).
  • NUP98::NSD1 fusion is most common (54%), associated with younger age, more de novo AML, higher rates of normal karyotype, FLT3-BITD, and WT1 mutations compared to other NUP98 rearrangements.
  • Median overall survival for the cohort is 15.2 months; FLT3 inhibitor therapy and hematopoietic stem cell transplant improve outcomes, with FLT3 inhibitors leading to 5-year OS of 53.3%, approaching intermediate-risk patient prognosis.