A phase 2 clinical trial led by the Dana-Farber Cancer Institute found that a three-drug combination produced high remission rates in patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN), a rare and aggressive blood cancer. The results were published in Blood.
The study enrolled 27 patients with a median age of 70. Treatment combined tagraxofusp, venetoclax, and azacitidine. Tagraxofusp was given on a shorter three-day schedule, while venetoclax and azacitidine were added to improve treatment effectiveness and help overcome resistance.
Among 16 newly diagnosed patients, 88% achieved remission. After two years, 65% were still alive and 53% remained free of disease progression. Median overall survival and progression-free survival had not yet been reached.
Among 11 patients whose disease had returned or did not respond to previous treatment, 64% achieved remission, with a median overall survival of 8.4 months.
The combination was generally well tolerated. Capillary leak syndrome, a known side effect of tagraxofusp, occurred in 15% of patients and was mostly mild to moderate.