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Acute Myeloid Leukemia Research / Retinoids in Leukemia and Cellular Processes · Journal article
Cancer · August 13, 2026
Encouraging direction, but not yet definitive.
This retrospective single-center cohort of 396 NPM1-mutated AML patients identifies older age, poor performance status, FLT3-ITD mutation, and extramedullary disease as adverse prognostic factors in those receiving high-intensity chemotherapy. A combination regimen of cladribine, low-dose cytarabine, and venetoclax alternating with azacitidine and venetoclax showed superior 5-year survival (74% vs. 24%, p=0.048) compared to hypomethylating agent plus venetoclax in an age-matched subset, though absolute event counts and therapy group sizes are not provided.
Retrospective cohort study. 396 patients (18% of the institution's newly diagnosed AML cases) with NPM1-mutated AML treated at The University of Texas MD Anderson Cancer Center. Intervention: High-intensity chemotherapy, hypomethylating agent (HMA) plus venetoclax, or cladribine plus low-dose cytarabine plus venetoclax alternating with azacitidine plus venetoclax. Compared with: Multiple treatment arms compared; high-intensity chemotherapy versus HMA plus venetoclax; cladribine-based alternating regimen versus HMA plus venetoclax. n = 396. The University of Texas MD Anderson Cancer Center (single center).
High-intensity chemotherapy group: median OS 84.7 months, 5-year OS rate 53% (95% CI, 44%–61%) HMA and venetoclax group: median OS 23.3 months, 5-year OS rate 19% (95% CI, 5%–39%) Cladribine, low-dose cytarabine, and venetoclax alternating with azacitidine and venetoclax vs. HMA and venetoclax: 5-year OS 74% vs. 24% in age-matched analysis (p=0.048)
Adverse prognostic factors in high-intensity chemotherapy group: older age, poor performance status, FLT3-ITD mutation, extramedullary disease
These findings provide outcome benchmarks and prognostic markers for risk-stratification of newly diagnosed NPM1-mutated AML patients. The superior outcome with cladribine-based alternating regimen warrants prospective validation before adoption, but may inform treatment selection in this subtype.
Retrospective cohort analysis identifying prognostic factors and comparing outcomes across therapies in NPM1-mutated AML, with clear survival endpoints but limited by single-center design and observational methodology.
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These findings provide outcome benchmarks and prognostic markers for risk-stratification of newly diagnosed NPM1-mutated AML patients. The superior outcome with cladribine-based alternating regimen warrants prospective validation before adoption, but may inform treatment selection in this subtype.
Graded across the dimensions that decide whether you should act, each from what the source actually supports. There is no single score, and where a dimension was not assessed it says so.
Abstract Background Nucleophosmin 1‐mutated ( NPM1mt ) acute myeloid leukemia (AML) is associated with a relatively favorable prognosis though long‐term outcomes remain suboptimal without clear predictors identified by therapy. Methods In a retrospective analysis, the authors identified 396 patients (18%) with newly diagnosed (ND) NPM1mt AML treated at The University of Texas MD Anderson Cancer Center and analyzed their outcomes. Using a Cox regression model, they analyzed predictors of survival in newly diagnosed NPM1mt AML across various therapies. Results Those treated with high intensity chemotherapy had a median overall survival (OS) of 84.7 months with a 5‐year rate of 53% (95% CI, 44%–61%) whereas those treated with a combination of a hypomethylating agent (HMA) and venetoclax had a median OS of 23.3 months with a 5‐year rate of 19% (95% CI, 5%–39%). The combination of cladribine, low‐dose cytarabine, and venetoclax alternating with azacitidine and venetoclax yielded better survival compared with HMA and venetoclax in an age‐matched analysis (5‐year OS rate of 74% vs. 24%) ( p =.048). Conclusion Among patients with NPM1mt treated with high‐intensity chemotherapy, older age, a poor performance status, and presence of a FLT3‐ITD mutation or extramedullary disease predicted a worse overall survival. For patients treated with a hypomethylating agent and venetoclax, older age was the only predictor of worse long‐term outcomes. These findings can be used for risk‐stratification of newly diagnosed NPM1mt AML and provide benchmarks of response and survival for this subtype.
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