How should we diagnose and treat blastic plasmacytoid dendritic cell neoplasm patients?

Blood Adv. 2019 Dec 23;3(24):4238-4251. doi: 10.1182/bloodadvances.2019000647.


Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare and aggressive leukemia for which we developed a nationwide network to collect data from new cases diagnosed in France. In a retrospective, observational study of 86 patients (2000-2013), we described clinical and biological data focusing on morphologies and immunophenotype. We found expression of markers associated with plasmacytoid dendritic cell origin (HLA-DRhigh, CD303+, CD304+, and cTCL1+) plus CD4 and CD56 and frequent expression of isolated markers from the myeloid, B-, and T-lymphoid lineages, whereas specific markers (myeloperoxidase, CD14, cCD3, CD19, and cCD22) were not expressed. Fifty-one percent of cytogenetic abnormalities impact chromosomes 13, 12, 9, and 15. Myelemia was associated with an adverse prognosis. We categorized chemotherapeutic regimens into 5 groups: acute myeloid leukemia (AML)-like, acute lymphoid leukemia (ALL)-like, lymphoma (cyclophosphamide, doxorubicin, vincristine, and prednisone [CHOP])-like, high-dose methotrexate with asparaginase (Aspa-MTX) chemotherapies, and not otherwise specified (NOS) treatments. Thirty patients received allogeneic hematopoietic cell transplantation (allo-HCT), and 4 patients received autologous hematopoietic cell transplantation. There was no difference in survival between patients receiving AML-like, ALL-like, or Aspa-MTX regimens; survival was longer in patients who received AML-like, ALL-like, or Aspa-MTX regimens than in those who received CHOP-like regimens or NOS. Eleven patients are in persistent complete remission after allo-HCT with a median survival of 49 months vs 8 for other patients. Our series confirms a high response rate with a lower toxicity profile with the Aspa-MTX regimen, offering the best chance of access to hematopoietic cell transplantation and a possible cure.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Acute Disease
  • Biomarkers
  • Blood Cell Count
  • Bone Marrow / pathology
  • Chromosome Aberrations
  • Clonal Evolution / genetics
  • Dendritic Cells / metabolism
  • Dendritic Cells / pathology*
  • Disease Management
  • Hematopoietic Stem Cell Transplantation
  • Humans
  • Immunophenotyping
  • Leukemia / diagnosis*
  • Leukemia / etiology
  • Leukemia / metabolism
  • Leukemia / therapy*
  • Neoplasm Metastasis
  • Neoplasm Staging
  • Prognosis
  • Treatment Outcome


  • Biomarkers