Long-term benefits of tagraxofusp for patients with blastic plasmacytoid dendritic cell neoplasm

Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is an aggressive myeloid malignancy with a poor prognosis. BPDCN is caused by transformed dendritic cells that overexpress CD123. Tagraxofusp (TAG) is a CD123-directed cytotoxin consisting of human interleukin-3 fused to truncated diphtheria toxin. Long-term analysis of a multicenter prospective trial of TAG for BPDCN showed that in patients with treatment-naïve disease TAG monotherapy resulted in high and durable responses, allowing many to bridge to stem-cell transplant. TAG was generally well-tolerated with a predictable and manageable safety profile.