@article{f08e574ddf35404eb502147ce2c6c0d4,
title = "Multicenter analysis of outcomes in blastic plasmacytoid dendritic cell neoplasm offers a pretargeted therapy benchmark",
abstract = "Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is an uncommon hematologic malignancy with poor outcomes. Existing data on the clinical behavior of BPDCN are limited because reported outcomes are from small retrospective series, and standardized treatment guidelines are lacking. The interleukin-3 cytotoxin conjugate tagraxofusp was recently tested in phase 1/2 trials that led to US Food and Drug Administration approval, the first ever for BPDCN. However, because there was no matched internal comparator in this or any clinical study to date, results of BPDCN trials testing new drugs are difficult to compare with alternative therapies. We therefore sought to define the clinical characteristics and outcomes of a group of patients with BPDCN treated at 3 US cancer centers in the modern era but before tagraxofusp was available. In 59 studied patients with BPDCN, the median overall survival from diagnosis was 24 months, and outcomes were similar in patients with “skin only” or with systemic disease at presentation. Intensive first-line therapy and “lymphoid-type” chemotherapy regimens were associated with better outcomes. Only 55% of patients received intensive chemotherapy, and 42% of patients underwent stem cell transplantation. Clinical characteristics at diagnosis associated with poorer outcomes included age >60 years, abnormal karyotype, and terminal deoxynucleotidyltransferase (TdT) negativity in the BPDCN cells. We also identified disease responses to pralatrexate and enasidenib in some patients. This study highlights poor outcomes for patients with BPDCN in the modern era and the need for new treatments. Outcomes from ongoing clinical trials for BPDCN can be evaluated relative to this contemporary cohort.",
author = "Justin Taylor and Michael Haddadin and Upadhyay, {Vivek A.} and Erwin Grussie and Neha Mehta-Shah and Brunner, {Andrew M.} and Abner Louissaintr and Lovitch, {Scott B.} and Ahmet Dogan and Fathi, {Amir T.} and Stone, {Richard M.} and Tallman, {Martin S.} and Rampal, {Raajit K.} and Neuberg, {Donna S.} and Stevenson, {Kristen E.} and Horwitz, {Steven M.} and Lane, {Andrew A.}",
note = "Funding Information: Conflict-of-interest disclosure: N.M.-S. has received research funding from Roche/Genentech, Celgene, Verastem, AstraZeneca, and Bristol-Myers Squibb and is a consultant for Kyowa-Hakka-Kirin. R.K.R. has received research funding from Incyte, Stemline Therapeutics, and Constellation and is a consultant for Incyte, Blueprint, Celgene, Agios, Jazz, BeyondSpring, Apexx, and Partner Therapeutics. S.M.H. has received research funding from Forty-Seven, Aileron, ADCT Therapeutics, Celgene, Infinity/Verastem, Millennium/Takeda, Seattle Genetics, and Trillium, and is a consultant for Kyowa-Hakka-Kirin, Millennium/Takeda, Infinity/Verastem, Seattle Genetics, Celgene, Miragen, Innate Pharma, Portola, Affimed, Kura, Mundipharma, and Astex. A.A.L. has received research funding from AbbVie and Stemline Therapeutics and is a consultant for N-of-One. The remaining authors declare no competing financial interests. Funding Information: J.T. is supported by the Conquer Cancer Foundation of the American Society of Clinical Oncology, the American Association for Cancer Research, the American Society of Hematology, the Robert Wood Johnson Foundation, and the National Cancer Institute of the National Institutes of Health (1K08CA230319-01). A.A.L. is supported by the National Cancer Institute of the National Institutes of Health (5R37CA225191-01) and was an American Society of Hematology Scholar. Publisher Copyright: {\textcopyright} 2019 by The American Society of Hematology.",
year = "2019",
month = aug,
day = "22",
doi = "10.1182/blood.2019001144",
language = "English",
volume = "134",
pages = "678--687",
journal = "Blood",
issn = "0006-4971",
number = "8",
}