@article{fbde7068cfdc4680b8dc6db2b8401e2a,
title = "Role of tagraxofusp in treating blastic plasmacytoid dendritic cell neoplasm (BPDCN)",
abstract = "Introduction: Advances and drug development in rare diseases, such as blastic plasmacytoid dendritic cell neoplasm (BPDCN), has historically been limited by small numbers of patients in the target population. In recent years, the development of tagraxofusp (SL-401) (ELZONRIS, Stemline Therapeutics) for the treatment of adult and pediatric BPDCN has been a successful story that led to US FDA approval in December 2018. Areas covered: In this evaluation of tagraxofusp, we briefly review chemistry; pharmacokinetics and pharmacodynamics, as we focus on the clinical experience and future directions. Expert Opinion: Tagraxofusp has been a welcome new addition and a successful initial development step in the targeted treatment of BPDCN. In phase I/II clinical trial, major responses were observed in 90% of treatment-na{\"i}ve patients, with 72% of the responses observed as complete remissions. Limitations on the usage of tagraxofusp and strategies to handle those limitations were further explored in this review.",
keywords = "BPDCN, CD123, ELZONRIS, SL-401, Tagraxofusp, blastic plasmacytoid dendritic cell neoplasm",
author = "Mansour Alfayez and Marina Konopleva and Naveen Pemmaraju",
note = "Funding Information: This study was supported in part by the MD Anderson Cancer Centre Support Grant (CCSG) CA016672, the MD Anderson Cancer Center Leukemia SPORE CA100632, the Charif Souki Cancer Research Fund and generous philanthropic contributions to the MD Anderson Moon Shots Program. Funding Information: M Konopleva: Consulting/honorarium: AbbVie, Genentech, F. Hoffman La-Roche, Stemline Therapeutics, Amgen, Forty-Seven, and Kisoji. Research funding/clinical trial support AbbVie, Genentech, F. Hoffman La-Roche, Eli Lilly, Cellectis, Calithera, Ablynx, Stemline Therapeutics, Agios, Ascentage, and Astra Zeneca. Stock options/Royalties: Reata Pharmaceutical. N Pemmaraju: Consulting/honorarium: Celgene, Stemline, Incyte Corporation, Novartis, MustangBio, Roche Diagnostics, and LFB. Research funding/clinical trials support from Stemline, Novartis, Abbvie, Samus, Cellectis, Plexxikon, Daiichi-Sankyo, Affymetrix, and SagerStrong Foundation. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed. Publisher Copyright: {\textcopyright} 2019, {\textcopyright} 2019 Informa UK Limited, trading as Taylor & Francis Group.",
year = "2020",
month = feb,
day = "1",
doi = "10.1080/14712598.2020.1701651",
language = "English (US)",
volume = "20",
pages = "115--123",
journal = "Expert Opinion on Biological Therapy",
issn = "1471-2598",
publisher = "Informa Healthcare",
number = "2",
}