Tagraxofusp, azacitidine, and venetoclax for blastic plasmacytoid dendritic cell neoplasm.

Tagraxofusp plus azacitidine and venetoclax produced composite complete remission rates of 88% in untreated and 64% in relapsed/refractory BPDCN.

• Why it matters: BPDCN is an orphan, aggressive malignancy with high CD123 expression and limited effective options beyond tagraxofusp alone.
• What they did: In a phase 2 trial (NCT03113643), 27 patients with previously untreated or relapsed/refractory BPDCN received 28-day cycles of azacitidine 75 mg/m² (days 1–7), venetoclax 400 mg (days 1–21), and tagraxofusp 12 µg/kg (days 4–6).
• The result: Safety was manageable with capillary leak syndrome in 15% of patients, and outcomes included 2-year overall survival of 65% and 2-year progression-free survival of 53% in the untreated cohort.

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