Abstract
Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is an aggressive, orphan hematologic malignancy that expresses CD123 and frequently presents in skin, bone marrow, blood, and viscera. Tagraxofusp is a first-in-class CD123-targeted therapy and the only US-approved drug to treat BPDCN. Approval was based on a pivotal, multicenter, phase II study (NCT02113982), the largest prospective BPDCN trial to date, in which tagraxofusp monotherapy demonstrated durable clinical responses across treatment-naive and relapsed/refractory BPDCN, and often resulted in patients proceeding to stem cell transplant following tagraxofusp-induced remissions. Advanced practitioners (APs) are critical in providing comprehensive and consistent monitoring, supportive care management for adverse events, and patient education. A core specialized interdisciplinary team coupled with AP-led management optimizes tagraxofusp treatment. This paper reviews best practices for the clinical management of patients with BPDCN receiving tagraxofusp in the context of the US package insert and APs’ real-world management approaches.
