ArticleBlood advances2023
Clinical outcomes associated with NPM1 mutations in patients with relapsed or refractory AML.
Article in Blood advances, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 38 papers, 1 of them a synthesis that pooled it.
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The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
38 citing papers in PubMed, 1 synthesis or guideline pooled it.
- Impact of secondary-type mutations on the prognosis of AML patients with NPM1 mutation: a systematic review and meta-analysis.Annals of hematology · 2025Pooled it
- A phase 1/2 study of DS-1594 menin inhibitor in relapsed/refractory acute leukemias.Journal of hematology & oncology · 2025Trial
- Ziftomenib in Relapsed or RefractoryJournal of clinical oncology : official journal of the American Society of Clinical Oncology · 2025Trial
- Trial
- Targeting Molecular Measurable Residual Disease and Low-Blast Relapse in AML With Venetoclax and Low-Dose Cytarabine: A Prospective Phase II Study (VALDAC).Journal of clinical oncology : official journal of the American Society of Clinical Oncology · 2024Trial
- Ziftomenib with venetoclax and azacitidine for relapsed/refractory NPM1-mutated acute myeloid leukemia.Blood · 2026Article
- Population Pharmacokinetics and Exposure-Response Analysis of Ziftomenib in Relapsed or Refractory Acute Myeloid Leukemia Patients With NPM1 Mutation.CPT: pharmacometrics & systems pharmacology · 2026Article
- Article
- Outcomes of relapsed or refractory acute myeloid leukemia after menin inhibition failure.Blood advances · 2026Article
- Menin Inhibition in Acute Myeloid MLL Rearranged Leukemias: A New Target for Precision Care.Cancers · 2026Review
- Outcomes of patients with relapsed/refractory acute leukaemia treated with revumenib with a focus on post-revumenib therapies.British journal of haematology · 2026Article
- FDA-Approved Fluorine-Containing Molecules in 2024: Significance, Synthesis, and Therapeutic Applications.Current topics in medicinal chemistry · 2026Review
- Optimizing lower intensity triplet therapy in acute myeloid leukemia: a practical guide.Blood cancer journal · 2025Review
- A Mutational Landscape in Acute Myeloid Leukemia: Overview and Prognostic Impacts.Diagnostics (Basel, Switzerland) · 2025Review
- Venetoclax and azacitidine for molecular relapse after intensive chemotherapy in NPM1 or CBF AML: a FILO study.Blood cancer journal · 2025Article
- Article
- Article
- Post-Relapse Outcomes of Older Patients With NPM1-Mutated AML Are Favorable With Allo Transplant in Second Remission.European journal of haematology · 2025Article
- Genetic Profiling of Acute and Chronic Leukemia via Next-Generation Sequencing: Current Insights and Future Perspectives.Hematology reports · 2025Review
- Factors affecting response and resistance to venetoclax in acute myeloid leukemia.Frontiers in oncology · 2025Review
Corrections and comments
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Authors and funding
25 authors.
Funding
Abstract
Mutations in Nucleophosmin 1 (NPM1) are associated with a favorable prognosis in newly diagnosed acute myeloid leukemia (AML), however, their prognostic impact in relapsed/refractory (R/R) settings are unknown. In a retrospective analysis, we identified 206 patients (12%) with mutated NPM1 (NPM1c) and compared their outcomes to 1516 patients (88%) with NPM1 wild-type (NPM1wt). NPM1c was associated with higher rates of complete remission or complete remission with incomplete count recovery compared with NPM1wt following each line of salvage therapy (first salvage, 56% vs 37%; P < .0001; second salvage, 33% vs 22%; P = .02; third salvage, 24% vs 14%; P = .02). However, NPM1 mutations had no impact on relapse-free survival (RFS) and overall survival (OS) with each salvage therapy with a median OS following salvage 1, 2 or 3 therapies in NPM1c vs NPM1wt of 7.8 vs 6.0; 5.3 vs 4.1; and 3.5 vs 3.6 months, respectively. Notably, the addition of venetoclax to salvage regimens in patients with NPM1c improved RFS and OS (median RFS, 15.8 vs 4.6 months; P = .05; median OS, 14.7 vs 5.9 months; P = .02). In conclusion, NPM1 mutational status has a minimal impact on prognosis in relapsed or refractory AML; therefore, novel treatment strategies are required to improve outcomes in this entity.
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