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September 2, 2026Annals of HematologyOpen Access

Retrospective evaluation of venetoclax-based regimen in newly diagnosed and relapsed/refractory acute myeloid leukemia: Efficacy determinants, survival outcomes, and model validation

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Authors

AWAnan WangWeifang Medical UniversityHLHaiping LiangCRChongchong Ren

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Implication

Retrospective study reveals distinct remission and survival predictors for venetoclax therapy in acute myeloid leukemia, highlighting a novel model to estimate early response.

Key Points

  • To assess treatment response, survival determinants, and clinical outcomes associated with venetoclax-based therapy in newly diagnosed and relapsed/refractory acute myeloid leukemia, and to construct a predictive model for first-cycle remission.
  • Retrospective single-center study evaluating 216 patients with acute myeloid leukemia (165 newly diagnosed, 51 relapsed/refractory) receiving venetoclax-based regimens between March 2020 and May 2025.
  • A multivariable prediction model for composite complete remission was developed and internally validated using 1,000 bootstrap resamples.
  • Composite complete remission occurred in 74.5% of newly diagnosed patients versus 39.2% of relapsed/refractory patients, and failure to achieve remission independently predicted worse overall survival in both cohorts.
  • In newly diagnosed disease, older age and NF1 mutations were independently associated with shorter survival, while lower baseline white blood cell count and higher bone marrow blast percentage favored remission in relapsed/refractory disease.
  • The six-predictor model (AML status, age, bone marrow blast percentage, platelet count, ECOG performance status, TET2 mutation) showed an apparent AUC of 0.841 (95% CI, 0.785–0.897) for remission, with time-dependent survival AUCs of 0.788 at 6 months and 0.695 at 12 months.

Cite This Study

Wang et al. (2026) studied this question.

synapsesocial.com/papers/6a97e2d9c562ede874ec7323https://doi.org/10.1007/s00277-026-07258-9
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Also Consider

Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context:

  1. 1Real-world efficacy and prognostic modeling of venetoclax-enhanced intensive chemotherapy in fit AML2025
  2. 2Predictive Models for Long Term Survival of AML Patients Treated with Venetoclax and Azacitidine or 7+3 Based on Post Treatment Events and Responses: Retrospective Cohort Study2024 · 9 citations
  3. 3Does the Timing of Response Impact the Outcome of Relapsed/Refractory Acute Myeloid Leukemia Treated with Venetoclax in Combination with Hypomethylating Agents? A Proof of Concept from a Monocentric Observational Study2025 · 3 citations
  4. 4Prognostic Model Combining Mutational and Cytogenetic Profiles in Acute Myeloid Leukemia Treated with Venetoclax and Hypomethylating Agents2026
  5. 5Venetoclax plus hypomethylating agent as first- and second-line treatment in patients with AML: A single-center experience2025