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Blastic plasmacytoid dendritic cell neoplasm: challenges and future prospects

机译：弹性浆细胞样树突状细胞瘤：挑战和未来前景

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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.
机译：母细胞浆样树突状细胞瘤（BPDCN）是一种罕见的CD4 + CD56 +髓样恶性肿瘤，难以诊断和治疗。 BPDCN在进展为白血病之前通常表现为非特异性皮肤病变，有或没有皮肤外表现。目前，尚无用于治疗BPDCN的标准护理，已使用了各种方法，包括急性髓细胞性白血病，急性淋巴细胞性白血病以及有或没有干细胞移植的基于淋巴瘤的治疗方案。尽管有这些治疗方法，但BPDCN的预后仍然很差，并且缺乏前瞻性数据作为治疗决策的依据。最近研究BPDCN的突变态势和基因表达谱的工作已经确定了许多潜在的治疗靶标。这样的靶标之一是人白细胞介素3受体的α亚基CD123，它是使用新型药物SL-401进行干预研究的对象。其他研究疗法包括UCART123，T细胞免疫疗法和venetoclax。需要进行前瞻性试验以确定这种罕见的侵袭性肿瘤的最佳治疗方法。

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