Comprehensive mutational profiling and clinical outcome of adult acute myeloid leukemia patients with NUP98 rearrangement
In a large study of aggressive blood cancer, FLT3 inhibitor drugs and stem cell transplant each independently improved five-year survival to over 50%.
This largest reported adult cohort of NUP98-rearranged AML (n=95) from multiple French centers provides detailed mutational profiling showing NUP98::NSD1 as the predominant fusion (54%), characteristically co-occurring with FLT3-ITD in younger patients. FLT3 inhibitor use and HSCT each independently improved survival, with FLT3i-treated patients achieving 53.3% 5-year OS, providing actionable treatment guidance for this adverse-risk AML subset.
What the study was
- Study design
- Retrospective multicenter cohort study
- Population
- Adult AML patients with NUP98 rearrangements across multiple French hematology centers
- Sample size
- 95
- Category
- Diagnostics
- Maturity
- Validated
- Journal
- Leukemia
Why it surfaced
Largest adult NUP98r AML cohort to date; confirms FLT3i benefit with 5-year OS improvement. Leukemia publication. Useful for clinical stratification and FLT3i usage guidance in this molecularly defined subset.
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