Multicenter analysis of outcomes in blastic plasmacytoid dendritic cell neoplasm offers a pretargeted therapy benchmark Journal Article

Authors: Taylor, J.; Haddadin, M.; Upadhyay, V. A.; Grussie, E.; Mehta-Shah, N.; Brunner, A. M.; Louissaintr, A. Jr; Lovitch, S. B.; Dogan, A.; Fathi, A. T.; Stone, R. M.; Tallman, M. S.; Rampal, R. K.; Neuberg, D. S.; Stevenson, K. E.; Horwitz, S. M.; Lane, A. A.
Article 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. © 2019 by The American Society of Hematology.
Journal Title: Blood
Volume: 134
Issue: 8
ISSN: 0006-4971
Publisher: American Society of Hematology  
Date Published: 2019-08-22
Start Page: 678
End Page: 687
Language: English
DOI: 10.1182/blood.2019001144
PUBMED: 31243042
PROVIDER: scopus
PMCID: PMC6706810
Notes: Article -- Export Date: 1 October 2019 -- Source: Scopus
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MSK Authors
  1. Martin Stuart Tallman
    461 Tallman
  2. Steven M Horwitz
    390 Horwitz
  3. Raajit Kumar Rampal
    146 Rampal
  4. Ahmet Dogan
    194 Dogan
  5. Neha Mehta Shah
    19 Shah
  6. Justin   Taylor
    27 Taylor