A capsule form of combination cytarabine and daunorubicin — known as CPX-351 — appears to be safe, and potentially effective, among patients with higher-risk myelodysplastic syndrome (MDS) or chronic myelomonocytic leukemia (CML), according to research published in The Lancet Haematology.

Not all patients with MDS or CML are eligible for allogeneic hematopoietic stem-cell transplantation (HSCT) and it is linked with a high risk of serious adverse events.

Previous research has shown that CPX-351 may be effective among patients with secondary acute myeloid leukemia, including among patients who receive HSCT. For this single-arm phase 2 study, researchers evaluated the safety and efficacy of CPX-351 among patients with higher-risk MDS or CML.

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Overall, 31 patients were enrolled. The median age was 62 years, 68% of patients were male, the average bone marrow blasts were 13%, and 71% of patients had an Eastern Cooperative Oncology Group performance status of 0.

The median follow-up was 16.1 months. Analysis showed that 87% (95% CI, 70-96) of patients responded to therapy and 52% of patients received at least 1 cycle of consolidation therapy; the complete remission rate was 52%. Furthermore, 94% of patients underwent HSCT.

Of the 28 patients who, at baseline, had less than 10% bone marrow blasts, 89% reached less than 5% blasts after induction. The most common grade 3/4 adverse events were pulmonary (26%) and cardiovascular (19%).

A total of 5 patients were hospitalized for infection; however, no treatment-related deaths were noted.

“In conclusion, results of this study provide a renewed interest for intensive chemotherapy with CPX-351 in patients with higher-risk myelodysplastic syndromes and chronic myelomonocytic leukaemia, especially to achieve blast clearance as a bridge to allogeneic HSCT,” the authors wrote in their report.

Disclosures: This research was supported by Jazz Pharmaceuticals. Please see the original reference for a full list of disclosures.


Peterlin P, Le Bris Y, Turlure P, et al. CPX-351 in higher risk myelodysplastic syndrome and chronic myelomonocytic leukaemia: a multicentre, single-arm, phase 2 study. Lancet Haematol. Published online May 25, 2023. doi:10.1016/S2352-3026(23)00090-X