A randomized comparison of CPX-351 and FLAG-Ida in high-risk myelodysplastic syndrome: A subgroup analysis of UK NCRI AML19

CPX-351 improves outcomes for patients with secondary acute myeloid leukemia, but evidence of its use in high-risk myelodysplastic syndromes (MDS) is limited. This exploratory subgroup analysis of the randomized, controlled, open-label, phase 3 study-UK NCRI AML19-compared outcomes with CPX-351 versus FLAG-Ida in patients with high-risk MDS, to provide insights into the potential role of CPX-351 in this population. A total of 57 patients were included (CPX-351, n = 34; FLAG-Ida, n = 23). Compared with FLAG-Ida, CPX-351 prolonged median overall survival (OS; 17.8 vs 10.8 months) and median relapse-free survival (19.1 vs 7 months). Hematopoietic cell transplantation (HCT) rate was 59% and 52% for CPX-351 and FLAG-Ida, respectively. Median OS post-HCT was longer with CPX-351 than FLAG-Ida (22.3 vs 11.6 months). CPX-351 had a lower rate of serious adverse events than FLAG-Ida (9%vs 61%). These findings suggest that CPX-351 may be a promising frontline treatment option for high-risk MDS, and warrant confirmation in prospective studies.