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Challenges
in NPM1-m AML

Challenges
in NPM1-m AML

NPM1‑m is the most common menin-dependent AML—30% of all adult AML cases1

NPM1-m AML remains challenging, with considerable variability in patient outcomes across the treatment continuum.9-12

Many patients with NPM1-m AML continue to relapse with poor outcomes11,13

In patients with NPM1-m AML,

50%

will relapse—most within 1 year11,13

Duration of first remission in patients with relapsing NPM1‑m AML11,a

aBased on a retrospective analysis of patients with R/R NPM1-m AML (n=206).11

Similar to those in the relapse setting without NPM1m,

Patients with relapsing NPM1-m AML face low survival rates11

Survival associated with NPM1 mutational status at AML relapse11,b

Median OS

NPM1m

(n=206)

6.1

months

NPM1-wt

(n=1516)

5.5

months

Median RFS

NPM1m

(n=102)

5.5

months

NPM1-wt

(n=496)

5.6

months

Twelve-month survival was as low as 23% in the subset of patients aged ≥60 years with R/R NPM1-m AML.12

bBased on a retrospective analysis of patients with R/R AML with NPM1m (n=206) compared with NPM1-wt (n=1516)  treated with standard-of-care therapies.11

Understanding the underlying disease mechanism driving NPM1‑m AML may offer hope to patients3,4

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Knowing a patient’s NPM1-m status is essential for guiding treatment decisions and care1,14

NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) recommend expedited testing for NPM1m at diagnosis and for repeat testing at relapse or progression to guide treatment.14,15

Accurate NPM1m diagnosis

Detection of NPM1m drives to a critical diagnosis of AML, even at ≥10% myeloid blasts or equivalents,c regardless of prior history of MDS.1,15

cEven at ≥10% myeloid blasts or equivalents (myeloblasts, monoblasts, and megakaryoblasts detected in the bone marrow or blood).15

Guiding clinical decisions

Comprehensive mutational profiling, including NPM1m, is critical for prognosis, risk stratification, and guiding treatment decisions.14,15

cEven at ≥10% myeloid blasts or equivalents (myeloblasts, monoblasts, and megakaryoblasts detected in the bone marrow or blood).15

AML, acute myeloid leukemia; MDS, myelodysplastic syndrome; NCCN, National Comprehensive Cancer Network® (NCCN®); NPM1-m, mutated nucleophosmin 1; NPM1m, nucleophosmin 1 mutation; NPM1-wt, wild-type nucleophosmin 1; OS, overall survival; RFS, relapse-free survival; R/R, relapsed/refractory.

References

References

1. Falini B et al. Blood Cancer Discov. 2024;5(1):8-20. 2. American Cancer Society. Accessed October 22, 2024. https://www.cancer.org/cancer/types/acute-myeloid-leukemia/about/key-statistics.html 3. Issa GC et al. Leukemia. 2021;35(9):2482-2495. 4. Candoni A et al. Hematol Rep. 2024;16(2):244-254. 5. Bertrums EJM et al. Haematologica. 2023;108(8):2044-2058. 6. National Cancer Institute. Accessed October 22, 2024. https://seer.cancer.gov/seertools/hemelymph/51f6cf59e3e27c3994bd547d/ 7. National Cancer Institute. Accessed October 22, 2024. https://seer.cancer.gov/seertools/hemelymph/5a7e288d1ef557f9c8636d31/ 8. Burrows F et al. Poster presented at: AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics: Discovery, Biology, and Clinical Applications; October 26-30, 2017; Philadelphia, PA. 9. Falini B et al. Blood. 2020;136(15):1707-1721. 10. Lachowiez CA et al. Blood Adv. 2020;4(7):1311-1320. 11. Issa GC et al. Blood Adv. 2023;7(6):933-942. 12. Issa GC et al. Blood Adv. 2023;7(Suppl):933-942. 13. Wang R et al. Front Oncol. 2022;12:972606. 14. Referenced with permission from the NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) for Acute Myeloid Leukemia V.2.2026. © National Comprehensive Cancer Network, Inc. 2025. All rights reserved. Accessed October 22, 2025. To view the most recent and complete version of the guideline, go online to NCCN.org. NCCN makes no warranties of any kind whatsoever regarding their content, use, or application, and disclaims any responsibility for their application or use in any way. 15. Döhner H et al. Blood. 2022;140(12):1345-1377.

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