Krah Nathan M, Hoeg Rasmus, Shami Paul J, Elf Shannon E, Lee Lauren E, Pemmaraju Naveen, Patel Ami B
Department of Internal Medicine, University of Utah, Salt Lake City, UT, USA.
Department of Internal Medicine, University of California, Davis Health, Sacramento, CA, USA.
Leuk Res Rep. 2025 Aug 25;24:100541. doi: 10.1016/j.lrr.2025.100541. eCollection 2025.
BPDCN is an aggressive myeloid malignancy characterized by unique expression of CD123, CD4, CD56, CD303, CD304, TCL1 and TCF4. The development of tagraxofusp, a CD123-directed cytotoxin, has revolutionized BPDCN treatment, especially for patients unfit for chemotherapy. While most patients respond to frontline tagraxofusp, there are challenges associated with treatment. In this case series, we describe our institutional experience treating BPDCN both pre- and post-tagraxofusp approval. We summarize six cases, the majority of which occurred in elderly patients, and highlight unique challenges of treating BPDCN at a center that serves a large rural/frontier area with variable access to specialty care.
母细胞性浆细胞样树突状细胞肿瘤(BPDCN)是一种侵袭性髓系恶性肿瘤,其特征在于CD123、CD4、CD56、CD303、CD304、TCL1和TCF4的独特表达。靶向CD123的细胞毒素tagraxofusp的研发彻底改变了BPDCN的治疗方式,尤其是对于不适合化疗的患者。虽然大多数患者对一线tagraxofusp有反应,但治疗仍存在挑战。在这个病例系列中,我们描述了我们机构在tagraxofusp获批前后治疗BPDCN的经验。我们总结了6例病例,其中大多数发生在老年患者中,并强调了在一个服务于农村/边境地区且专科护理可及性各异的中心治疗BPDCN的独特挑战。