Multicenter phase 2 trial of CPX-351 for higher-risk myelodysplastic syndrome and chronic myelomonocytic leukemia

CPX-351 is an encapsulated form of cytarabine and daunorubicin. A phase 2 trial at 12 centers in France evaluated CPX-351 for higher-risk myelodysplastic syndrome and chronic myelomonocytic leukemia. Response was seen in 87% of patients. Allogeneic HSCT could be performed in 94%. The most common grade 3 or 4 adverse events were pulmonary (26% of patients) and cardiovascular (19%). Among 31 patients, there was only one treatment-related serious adverse event. The authors conclude that CPX-351 appears to be active and safe in patients with higher-risk MDS and CMML, allowing bridging to allo-HSCT in most patients.