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Lower Dose CPX-351 Is Safe in Patients with HR-MDS

By Melissa Badamo November 27, 2023

Lower doses of CPX-351 (cytarabine and daunorubicin) are safe in treating patients with higher-risk myelodysplastic syndromes (HR-MDS) or chronic myelomonocytic leukemia (CMML), according to a phase I/II, single-center study.

The researchers, led by Guillermo Montalban-Bravo, MD, of the University of Texas MD Anderson Cancer Center, aimed to determine the maximum tolerated dose of CPX-351, but noted that further studies are needed to evaluate its activity.

Twenty-five patients (19 with HR-MDS and six with CMML) received four doses of intravenous CPX-351 (10, 25, 50, and 75 units/m2) on days one, three, and five of induction and on days one and three of consolidation. Five patients (20%) required dose reductions after the first cycle, and three patients older than 75 years experienced cardiac toxicity at the 75 units/m2 dose.

After further enrollment at 50 units/m2, the four-week mortality was 0% and the eight-week mortality was 8%. Therefore, researchers concluded that doses of up to 50 units/m2 (22 mg/m2 units of daunorubicin and 50 mg/m2 of cytarabine) were generally safe in the high-risk population and were associated with “clinically meaningful” responses.

The overall response rate was 56%, the median relapse-free survival was 9.2 months, and the median overall survival was 8.7 months. Febrile neutropenia (48%) and lung infection (20%) were the most common grade 3-4 nonhematological, treatment-emergent adverse events. 


Montalban-Bravo G, Jabbour E, Borthakur G, et al. Phase 1/2 study of CPX-351 for patients with Int-2 or high risk International Prognostic Scoring System myelodysplastic syndromes and chronic myelomonocytic leukaemia after failure to hypomethylating agents. Br J Haematol. 2023; doi:10.1111/bjh.19193

Original Source: Lower Dose CPX-351 Is Safe in Patients with HR-MDS | Blood Cancers Today


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