Department of Internal Medicine, The University of Texas School of Health Sciences at Houston, Houston, TX, USA.
Department of Medicine, Division of Hematologic Malignancies and Cellular Therapy, Duke University, Durham, NC, USA.
Curr Hematol Malig Rep. 2019 Dec;14(6):515-522. doi: 10.1007/s11899-019-00556-2.
Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare, clinically aggressive hematologic malignancy that has heterogeneous presentation and can involve the skin, lymph nodes, and bone marrow. Recent advancements in our patho-biologic understanding of the disease have led to the development of new targeted therapies for BPDCN. In this review, we aimed to describe some of the novel treatments that are being put forward for the management of BPDCN.
Tagraxofusp is the first CD123-targeted therapy approved as the first ever targeted treatment of BPDCN in patients aged 2 years and older. This agent was approved based on a pivotal clinical trial that showed that it was associated with high rates of clinical responses in both treatment-naïve and treatment-experienced patients. The most serious adverse event was occurrence of the capillary leak syndrome. Other targeted therapies are actively being investigated in clinical trials. These include other CD123-targeted approaches, as well as active investigation in targets beyond CD123, such as the BCL-2 inhibitor, venetoclax. BPDCN is a rare hematologic clonal disorder with historically poor outcomes. Newer targeted therapies have been recently introduced, with promising results and novel toxicities that are important to recognize and understand. Stem cell transplantation after achievement of complete remission remains the mainstay of therapy among younger/fit, eligible patients, regardless of treatment modality used.
原始血液细胞瘤性树突细胞肿瘤（BPDCN）是一种罕见的、临床上侵袭性强的血液恶性肿瘤，其表现具有异质性，可累及皮肤、淋巴结和骨髓。近年来，我们对该疾病的病理生物学认识的进步，导致了针对 BPDCN 的新靶向治疗方法的发展。在这篇综述中，我们旨在描述一些针对 BPDCN 管理提出的新治疗方法。
Tagraxofusp 是首个被批准的靶向 CD123 的治疗药物，是首个用于治疗 2 岁及以上患者的 BPDCN 的靶向治疗药物。该药物是基于一项关键性临床试验而获得批准的，该试验表明，它在治疗初治和治疗后患者中均与高临床反应率相关。最严重的不良事件是发生毛细血管渗漏综合征。其他靶向治疗方法正在临床试验中积极研究。这些方法包括其他靶向 CD123 的方法，以及对 CD123 以外的靶点的积极研究，如 BCL-2 抑制剂 venetoclax。BPDCN 是一种罕见的血液克隆性疾病，历史上预后较差。最近引入了新的靶向治疗方法，其结果令人鼓舞，但也有新的毒性作用，这一点非常重要，需要加以识别和理解。对于年轻/合适、有资格的患者，无论使用何种治疗方式，在获得完全缓解后进行干细胞移植仍然是主要的治疗方法。
