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Blastic plasmacytoid dendritic-cell neoplasm (BPDCN) is a rare, highly aggressive malignancy that responds poorly to conventional therapy with intensive acute leukemia or lymphoma chemotherapy regimens.
Given that virtually all cases of BPDCN express the interleukin-3 (IL-3) receptor alpha (CD123), investigators conducted an industry-supported, multicenter, open-label study in which tagraxofusp — a CD123-directed fusion cytotoxin linking IL-3 with truncated diphtheria toxin — was administered to 47 patients with BPDCN (median age, 70 years; 32 were previously untreated). Patients received daily infusions of tagraxofusp (7 or 12 µg/kg) on days 1 to 5 of each 21-day cycle until disease progression or unacceptable toxicity.
At a median follow-up…