Trottier Amy M, Cerquozzi Sonia, Owen Carolyn J
Division of Hematology and Hematological Malignancies, University of Calgary, Foothills Medical Centre, Calgary, AB, Canada,
Blood Lymphat Cancer. 2017 Dec 11;7:85-93. doi: 10.2147/BLCTT.S132060. eCollection 2017.
Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare CD4+ CD56+ myeloid malignancy that is challenging to diagnose and treat. BPDCN typically presents with nonspecific cutaneous lesions with or without extra-cutaneous manifestations before progressing to leukemia. Currently, there is no standard of care for the treatment of BPDCN and various approaches have been used including acute myeloid leukemia, acute lymphoblastic leukemia, and lymphoma-based regimens with or without stem cell transplantation. Despite these treatment approaches, the prognosis of BPDCN remains poor and there is a lack of prospective data upon which to base treatment decisions. Recent work examining the mutational landscape and gene expression profiles of BPDCN has identified a number of potential therapeutic targets. One such target is CD123, the α subunit of the human interleukin-3 receptor, which is the subject of intervention studies using the novel agent SL-401. Other investigational therapies include UCART123, T-cell immunotherapy, and venetoclax. Prospective trials are needed to determine the best treatment for this uncommon and aggressive neoplasm.
母细胞性浆细胞样树突状细胞肿瘤（BPDCN）是一种罕见的CD4 + CD56 +髓系恶性肿瘤，其诊断和治疗具有挑战性。BPDCN通常在进展为白血病之前表现为非特异性皮肤病变，可伴有或不伴有皮肤外表现。目前，BPDCN的治疗尚无标准治疗方案，已采用了多种方法，包括基于急性髓系白血病、急性淋巴细胞白血病和淋巴瘤的方案，有或没有干细胞移植。尽管有这些治疗方法，BPDCN的预后仍然很差，并且缺乏用于指导治疗决策的前瞻性数据。最近对BPDCN的突变图谱和基因表达谱进行研究的工作已经确定了一些潜在的治疗靶点。其中一个这样的靶点是CD123，即人白细胞介素-3受体的α亚基，它是使用新型药物SL-401进行干预研究的对象。其他研究性疗法包括UCART123、T细胞免疫疗法和维奈克拉。需要进行前瞻性试验以确定针对这种罕见且侵袭性肿瘤的最佳治疗方法。
