Acute myeloid leukemia after myeloproliferative neoplasms: recent real-world outcomes in the United States

Progression to acute myeloid leukemia (AML) is a rare complication of myeloproliferative neoplasms (MPNs) with limited treatment options and median overall survival (OS) of 3-6 months. In recent years, novel targeted therapies have been introduced, including the BCL2 inhibitor venetoclax (VEN). Retrospective analysis of the USA-wide Flatiron Health Research Database investigated real-world outcomes of post-MPN AML during 2014-2024. Although the proportion of patients treated with lower-intensity therapies (LIT) including VEN in combination with hypomethylating agents increased over time, OS was similar among patients diagnosed before and after VEN approval in 2018 (median OS 7.1 versus 7.6 months; p=0.39). Only 15% of post-MPN AML patients underwent allogeneic hematopoietic cell transplant (allo-HCT), which was associated with better OS (median 20.5 versus 5.8 months; p<0.01). Although patients receiving intensive chemotherapy (IC) had longer OS than those receiving LIT (HR 1.95; p=0.02), patients receiving IC and LIT as a bridge to allo-HCT had comparable OS (p=0.37). The authors conclude that allo-HCT is the only potentially curative option for post-MPN AML.