Characteristics and Outcomes of Patients With Blastic Plasmacytoid Dendritic Cell Neoplasm Treated With Frontline HCVAD

Authors

Naveen Pemmaraju
Nathaniel R Wilson
Guillermo Garcia-Manero
Koji Sasaki
Joseph D Khoury
Nitin Jain
Gautam Borthakur
Farhad Ravandi
Naval Daver
Tapan Kadia
Courtney DiNardo
Elias Jabbour
Sherry Pierce
Muzaffar Qazilbash
Marina Konopleva
Hagop Kantarjian

Publication Date

5-24-2022

Journal

Blood Advances

Abstract

Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a clinically aggressive blood cancer, often involving the skin, bone marrow, lymph nodes, and central nervous system (CNS) in 20% to 30% of patients. Despite significant progress in CD123- and BCL-2-targeted therapy, most patients are not cured without hematopoietic stem cell transplant (HSCT), and CNS relapses occur quite frequently. Combination approaches with targeted and chemotherapy agents plus incorporation of prophylactic CNS-directed therapy are urgently needed. In this setting, we sought to analyze outcomes using the cytotoxic chemotherapy backbone regimen hyperfractionated cyclophosphamide, vincristine, adriamycin, and dexamethasone (HCVAD). We conducted a retrospective analysis of patients with BPDCN (n = 100), evaluating complete remission (CR) and median overall survival (OS) among 3 groups: those who received frontline HCVAD-based therapy (n = 35), SL-401 (n = 37), or other regimens (n = 28). HCVAD-based regimens yielded higher CR (80% vs 59% vs 43%; P = .01). There was no significant difference in OS (28.3 vs 13.7 vs 22.8 months; P = .41) or remission duration probability among treatment groups (38.6 vs not reached vs 10.2 months; P = .24). HSCT was performed in 51% vs 49% vs 38%, respectively (P = .455). These results suggest a continued important role for HCVAD-based chemotherapy in BPDCN, even in the modern targeted-therapy era, with high CR rates in the frontline setting. Further studies must establish the clinical activity, feasibility, and safety of doublet/triplet combinations of targeted therapies plus cytotoxic agents and the addition of CNS prophylaxis, with the ultimate goal of durable long-term remission for patients with BPDCN.

Keywords

Antineoplastic Agents, Cyclophosphamide, Dendritic Cells, Doxorubicin, Humans, Myeloproliferative Disorders, Neoplasm Recurrence, Local, Retrospective Studies, Skin Neoplasms, Vincristine

DOI

10.1182/bloodadvances.2021006645

PMID

35061885

PMCID

PMC9131912

PubMedCentral® Posted Date

5-17-2022

PubMedCentral® Full Text Version

Post-print

Published Open-Access

yes