Tagraxofusp for Blastic Plasmacytoid Dendritic Cell Neoplasm

Tagraxofusp, a CD123-targeted immunotoxin, is the first FDA-approved treatment for patients 2 years and older with blastic plasmacytoid dendritic cell neoplasm (BPDCN). It has been shown to be safe and effective in treatment-naïve and previously treated adult patients, with high rates of successful bridging to hematopoietic stem cell transplantation. The pediatric experience is more limited but demonstrates safety. Given the risk of potentially fatal capillary leak syndrome with tagraxofusp, judicious patient selection is recommended. Combination therapy with hypomethylating agents and/or BCL-2 inhibitors are rational next lines of investigation, especially in patients ineligible to receive high-dose chemotherapy.