Beziat Guillaume, Ysebaert Loïc
Hematology Department, University Hospitals of Toulouse, IUC Toulouse-Oncopole, Toulouse, France.
University Toulouse-3 Paul Sabatier, Toulouse, France.
Onco Targets Ther. 2020 Jun 9;13:5199-5205. doi: 10.2147/OTT.S228342. eCollection 2020.
Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare myeloid malignancy, for which conventional chemotherapy has poor outcomes. CD123, the α-subunit of interleukin (IL)-3 receptor, is constantly overexpressed at the surface of tumoral cells. Tagraxofusp (or SL-401) is a recombinant cytotoxin which consists of human interleukin-3 fused to a truncated diphtheria toxin. It is currently the only novel therapy with a prospective evaluation of efficacy and safety in the treatment of BPDCN and is also the only one to achieve FDA approval. In this short review, the results of tagraxofusp are summarized and perspectives of its use in BPDCN and in other malignancies are discussed. The safety profile is also summarized, since capillary leak syndrome is the main toxic effect of the drug, along with more common toxicities including an increase in transaminases and thrombocytopenia.
母细胞样浆细胞样树突状细胞肿瘤（BPDCN）是一种罕见的髓系恶性肿瘤，传统化疗对此疗效不佳。白细胞介素（IL）-3受体的α亚基CD123在肿瘤细胞表面持续过度表达。塔格昔单抗（或SL-401）是一种重组细胞毒素，由与人白细胞介素-3融合的截短白喉毒素组成。它是目前唯一一种对治疗BPDCN的疗效和安全性进行前瞻性评估的新型疗法，也是唯一获得美国食品药品监督管理局（FDA）批准的疗法。在这篇简短的综述中，总结了塔格昔单抗的治疗结果，并讨论了其在BPDCN和其他恶性肿瘤中的应用前景。还总结了其安全性概况，因为毛细血管渗漏综合征是该药物的主要毒性作用，同时还包括转氨酶升高和血小板减少等更常见的毒性反应。
