从历史上看，母细胞性浆细胞样树突状细胞肿瘤（BPDCN）的治疗选择仅限于传统化疗，采用用于治疗急性髓系白血病或急性淋巴细胞白血病或淋巴瘤的方案。如今，一种针对 CD123 的新疗法可用于治疗 BPDCN。然而，无论选择何种治疗方法，实现首次完全缓解 (CR1) 都是治疗的主要目标，因为如果提供同种异体造血细胞移植 (allo-HCT) 作为巩固治疗，它代表了延长 BPDCN 生存期的最佳机会。尽管没有特定的预处理方案被认为是符合异基因 HCT 条件的患者的标准治疗方案，但两个大型登记中心的最新数据报告称，在提供基于全身照射的清髓性预处理 (MAC) 方案时具有生存优势。不幸的是，MAC 方案不适用于老年/身体不适的患者，而这些患者在全世界就诊的患者中占相当大的比例。在这种情况下，降低强度的调理方案是下一个最佳选择。对于在初次就诊和手术时没有 BM 受累的老年/不健康患者，可以考虑自动 HCT，尽管支持这一选择的数据较少。未来的研究需要破译该疾病的临床、遗传和分子特征之间的相互作用，从而通过提高疗效和避免不必要的毒性来相应地进行个性化治疗。版权所有 © 2024 美国血液学会。

Historically, treatment options for blastic plasmacytoid dendritic cell neoplasm (BPDCN) were limited to conventional chemotherapy, adopted from regimens used to treat acute myeloid or acute lymphoblastic leukemias, or lymphomas. Nowadays, a novel therapy targeting CD123 is available to treat BPDCN. Yet, regardless of treatment choice, achieving a first complete remission (CR1) represents the main goal of therapy, because it represents the best opportunity to prolong survival in BPDCN, if offered an allogeneic hematopoietic cell transplant (allo-HCT) as consolidative therapy. Although no specific conditioning regimen is considered standard-of-care in allo-HCT eligible patients, recent data from two large registries reported a survival advantage when offering total body irradiation-based myeloablative conditioning (MAC) regimens. Unfortunately, applicability of MAC regimens is not feasible in older/unfit patients, which represents a considerable proportion of patients presenting worldwide. In such cases, reduced intensity conditioning regimens represent the next best option. Auto-HCT could be considered in older/unfit patients who did not have BM involvement at initial presentation and at time of the procedure, albeit data supporting this option is less abundant. Future research is needed to decipher the interplay between clinical, genetic, and molecular features of the disease to personalize treatment accordingly, by enhancing efficacy and avoiding unnecessary toxicities.Copyright © 2024 American Society of Hematology.