Faysman Karolina, Rubin Joelle, Lubas Amber, Kujawa Taylor, Hinojosa Gabriel, Marx Kayleigh R, Katsetos John, Galinsky Ilene A
From UCLA Medical Center, Los Angeles, California.
Hackensack Meridian Hackensack University Medical Center, Hackensack, New Jersey.
J Adv Pract Oncol. 2025 Jul 2:1-14. doi: 10.6004/jadpro.2025.16.7.21.
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.
母细胞样浆细胞样树突状细胞肿瘤（BPDCN）是一种侵袭性的罕见血液系统恶性肿瘤，表达CD123，常出现在皮肤、骨髓、血液和内脏中。塔格拉索夫（Tagraxofusp）是首个靶向CD123的疗法，也是美国唯一获批用于治疗BPDCN的药物。其获批基于一项关键的多中心II期研究（NCT02113982），这是迄今为止最大规模的BPDCN前瞻性试验，其中塔格拉索夫单药治疗在初治和复发/难治性BPDCN患者中均显示出持久的临床反应，且常使患者在塔格拉索夫诱导缓解后接受干细胞移植。高级执业医师（APs）在提供全面且一致的监测、不良事件的支持性护理管理以及患者教育方面至关重要。一个核心的专业跨学科团队加上由APs主导的管理可优化塔格拉索夫治疗。本文结合美国药品说明书和APs的实际管理方法，综述了接受塔格拉索夫治疗的BPDCN患者临床管理的最佳实践。
