Therapeutic Approaches for Blastic Plasmacytoid Dendritic Cell Neoplasm: Allogeneic Hematopoietic Cell Transplantation and Novel Therapies

Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a clinically aggressive hematologic malignancy derived from precursors of plasmacytoid dendritic cells. There is no established standard therapy for BPDCN and the efficacy of conventional chemotherapy is limited, with an anticipated median overall survival ranging from 8 to 14 months. No randomized controlled trials have ever been performed to evaluate the benefit of frontline consolidation with an allogeneic hematopoietic cell transplant (allo-HCT) in BPDCN. Yet, offering an allograft has become the de facto option in BPDCN, and remains the only known long-term curative option for these patients, even in the modern era of targeted therapies. In our opinion, allo-HCT is recommended as part of frontline consolidation, especially in patients achieving first complete remission and who are deemed capable of tolerating the procedure, as published data show 3- to 4-year progression-free survival ranging from 69% to 74% in this population. Prompt referral to a transplant center, at the time of a diagnosis of BPDCN, is important to confirm allo-HCT candidacy and to initiate the process of identifying a suitable human leukocyte antigen (HLA)-compatible donor. Because disease relapse remains a major concern, additional strategies, such as post-allograft consolidation/maintenance therapy, are certainly needed to help further improve outcomes. Finally, patients deemed ineligible to receive an allo-HCT, due to lack of response and/or poor performance status, should be considered for enrollment in clinical trials.