Acute myeloid leukemia after myeloproliferative neoplasms: Real-world outcomes in the new treatment era in the United States.

Abstract

Background: 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. The treatment of AML has been revolutionized in recent years with the introduction of novel targeted therapies including the BCL2 inhibitor venetoclax (VEN). However, evidence regarding outcomes in patients with post-MPN AML remains limited.

Methods: To evaluate the impact of novel therapies on the outcomes of patients with post-MPN AML, the authors conducted a retrospective analysis of 392 patients who were diagnosed with post-MPN AML during 2014-2024 in the United States and were included in the Flatiron Health Research Database.

Results: Although the proportion of patients treated with lower-intensity therapies (LIT) including VEN in combination with hypomethylating agents increased over time, OS was very similar among patients diagnosed before and after VEN approval (median OS, 7.1 [95% confidence interval (CI), 5.7-9.5] months vs. 7.6 [95% CI, 5.8-10.1] months; p = .39). Only 15% of post-MPN AML patients underwent an allogeneic hematopoietic cell transplant (allo-HCT). Those who received allo-HCT experienced better OS than patients who did not receive allo-HCT (median OS, 20.5 [95% CI, 15.4-36.2] months vs. 5.8 [95% CI, 5.0-6.8] months; p < .01). Although patients treated with intensive chemotherapy (IC) had longer OS than those treated with LIT (hazard ratio, 1.95; 95% CI, 1.12-3.41; p = .02), patients receiving IC and LIT as a bridge to allo-HCT had comparable OS (p = .37).

Conclusions: This study highlights allo-HCT as the only potentially curative option with both IC and LIT serving as effective bridging therapies.