ArticleBlood advances2024
Venetoclax-based low intensity therapy in molecular failure of NPM1-mutated AML.
Article in Blood advances, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 20 papers, 1 of them a synthesis that pooled it.
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Who cites it
20 citing papers in PubMed, 1 synthesis or guideline pooled it, 28 citations in OpenAlex.
- Comparative efficacy and safety in low-intensity treatment for acute myeloid leukemia in older patients: a systematic review and network meta-analysis.European journal of medical research · 2025Pooled it
- Real-world outcomes of newly diagnosed AML treated with venetoclax and azacitidine or low-dose cytarabine in the UK NHS.Blood neoplasia · 2024Trial
- Ziftomenib with venetoclax and azacitidine for relapsed/refractory NPM1-mutated acute myeloid leukemia.Blood · 2026Article
- Review
- Hypomethylating agents alone or in combination with venetoclax in very elderly acute myeloid leukemia patients: less treatment, better care?Annals of hematology · 2026Article
- Review
- Measurable residual disease-guided therapy in acute myeloid leukaemia: Practical insights.British journal of haematology · 2025Review
- Precision oncology to overcome resistance in R/R AML in children and adults requires combinations of cytotoxic, targeted, and immunological treatments.Journal of internal medicine · 2025Review
- Venetoclax and azacitidine for molecular relapse after intensive chemotherapy in NPM1 or CBF AML: a FILO study.Blood cancer journal · 2025Article
- Measurable residual disease monitoring in AML: Prospects for therapeutic decision-making and new drug development.American journal of hematology · 2025Review
- Diagnosis and treatment of AML in the context of WHO and ICC 2022 classifications: Divergent nomenclature converges on common therapies.HemaSphere · 2025Review
- Molecular measurable residual disease monitoring and transplant indications in NPM1 mutated acute myeloid leukemia.Bone marrow transplantation · 2025Review
- Pre-emptive therapeutic decisions based on measurable residual disease status in acute myeloid leukemia: ready for prime time?Leukemia · 2025Review
- Factors affecting response and resistance to venetoclax in acute myeloid leukemia.Frontiers in oncology · 2025Review
- Comparative assessment of antitumor effects between doxorubicin and mitochondria-targeted doxorubicin in combination with radiotherapy.Oncology research · 2025Article
- Article
- Mutation- and MRD-informed treatment decisions for the transplant-eligible AML patient.Hematology. American Society of Hematology. Education Program · 2024Review
- The prognostic significance of genetics in acute myeloid leukemia under venetoclax-based treatment.Annals of hematology · 2024Review
- Only FLT3-ITD co-mutation did not have a deleterious effect on acute myeloid leukemia patients with NPM1 mutation, but concomitant with DNMT3A co-mutation or a < 3log reduction of MRD2 predicted poor survival.Annals of hematology · 2024Article
- Optimal Post-Remission Consolidation Therapy in Patients with AML.Acta haematologica · 2024Review
Corrections and comments
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Authors and funding
32 authors at 20 institutions in 6 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
abstractMolecular failure in NPM1-mutated acute myeloid leukemia (AML) inevitably progresses to frank relapse if untreated. Recently published small case series show that venetoclax combined with low-dose cytarabine or azacitidine can reduce or eliminate measurable residual disease (MRD). Here, we report on an international multicenter cohort of 79 patients treated for molecular failure with venetoclax combinations and report an overall molecular response (≥1-log reduction in MRD) in 66 patients (84%) and MRD negativity in 56 (71%). Eighteen of 79 patients (23%) required hospitalization, and no deaths were reported during treatment. Forty-one patients were bridged to allogeneic transplant with no further therapy, and 25 of 41 were MRD negative assessed by reverse transcription quantitative polymerase chain reaction before transplant. Overall survival (OS) for the whole cohort at 2 years was 67%, event-free survival (EFS) was 45%, and in responding patients, there was no difference in survival in those who received a transplant using time-dependent analysis. Presence of FLT3-ITD mutation was associated with a lower response rate (64 vs 91%; P < .01), worse OS (hazard ratio [HR], 2.50; 95% confidence interval [CI], 1.06-5.86; P = .036), and EFS (HR, 1.87; 95% CI, 1.06-3.28; P = .03). Eighteen of 35 patients who did not undergo transplant became MRD negative and stopped treatment after a median of 10 months, with 2-year molecular relapse free survival of 62% from the end of treatment. Venetoclax-based low intensive chemotherapy is a potentially effective treatment for molecular relapse in NPM1-mutated AML, either as a bridge to transplant or as definitive therapy.
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