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December 8, 2025Blood

Phase I study of lower dose CPX-351 combined with venetoclax for patients with higher-risk myelodysplastic syndrome or chronic myelomonocytic leukemia after hypomethylating agents failure

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Authors

EJElias JabbourKCKelly S. ChienFRFarhad Ravandi

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Overview

Phase I trial shows combined CPX-351 and venetoclax achieves 84% overall response in patients with high-risk MDS or chronic myelomonocytic leukemia, suggesting an effective approach.

Key Points

  • Eighty-four percent of patients achieved an overall response rate after treatment with CPX-351 and venetoclax.
  • Most common grade ≥3 treatment-emergent adverse events were thrombocytopenia and febrile neutropenia affecting 50% and 33% respectively.
  • This phase I clinical trial utilized a Bayesian optimal interval design to determine maximum tolerated dose of the combination therapy.
  • Results highlight the need for continued evaluation in larger cohorts to confirm efficacy and safety of the CPX-351 and venetoclax combination.

Cite This Study

Jabbour et al. (2025) studied this question.

synapsesocial.com/papers/69362f4e4fa91c937236d898https://doi.org/10.1182/blood-2025-5645
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