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Allogeneic hematopoietic cell transplantation for patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN)


Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN) is an aggressive hematological malignancy; however, some patients achieve durable remission with allogeneic hematopoietic cell transplantation (allo-HCT). We report on all 17 patients with BPDCN who underwent allo-HCT at our center between 2000 and 2020. The median age was 39 (18–67) years. All (n = 16, 94%), except one patient, had systemic disease involving bone marrow and/or other organs. Ten patients (59%) were in first complete remission (CR1) at allo-HCT. The donor source was matched related or unrelated in ten (59%) and alternate donor in seven (41%) patients. Five (31%) patients developed acute graft-versus-host disease (GVHD), all grade I-II. The cumulative incidence (CI) of chronic GVHD at five-year was 34%. The CI of non-relapse mortality at one-year was 29%. Progression-free survival (PFS) rates at two-year and five-year were 49% (95% CI = 22–71%) and 39% (95% CI = 14–64%), respectively. The two-year and five-year overall survival (OS) rates were 65% (95% CI = 38–82%) and 40% (95% CI = 12–68%), respectively. The five-year rate for both PFS and OS was 80% in CR1 patients versus 0% in patients not in CR1. In conclusion, allo-HCT provides long-lasting remissions in BPDCN patients, particularly when performed in CR1.

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Fig. 1: Progression-free survival and overall survival.
Fig. 2: Progression-free survival and overall survival according to complete remission status.


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Author information




QB, MHQ, and NP designed the study, interpreted the data, and wrote the manuscript; DRM analyzed the data and created figures; URP, PK, CH, IFK, KR, YN, BO, SS, NS, ALO, SA, GA, GR, MYK, REC, and EJS contributed to data collection, writing, and revision of the manuscript and approved the final version.

Corresponding author

Correspondence to Naveen Pemmaraju.

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Competing interests

QB has research funding from Acrotech and Stemline. QB served on advisory board of Purdue and Spectrum. MQ has research funding from Janssen, Bioline, Angiocrine, Amgen and Neximmune. SA has served on an advisory board and received research funding from Tessa Therapeutics and SeaGen. PK has research support from Amgen and Ziopharm; has served on advisory boards for Pfizer, Kite and Novartis; consulting fees from Jazz. MK has received grants and other from AbbVie, Genentech, F. Hoffman La-Roche, Stemline Therapeutics, and Forty-Seven. MK has received grants from Eli Lilly, Cellectis, Calithera, Ablynx, Agios, Ascentage, Astra Zeneca, Rafael Pharmaceutical and Sanofi. MK has received other from Amgen, Kisoji and Reata Pharmaceutical. MK holds US patent 7,795,305 B2, “CDDO-compounds and combination therapies thereof” with royalties paid to Reata Pharmaceutical, a patent Combination Therapy with a mutant IDH1 Inhibitor and a BCL-2 licensed to Eli Lilly, and patent 62/993,166 Combination of a MCL-1 Inhibitor And Midostaurin, Uses And Pharmaceutical Composition Thereof pending to Novartis. ES has received consultant fees and honoraria from Bayer HealthCare Pharmaceuticals, Novartis, Magenta and Adaptimmune. ES has received honoraria from Partner Therapeutics, Mesoblast and Axio. ES is the co-inventor on a provisional patent application owned by MD Anderson and licensed to Takeda. All other authors report no COI. This research was supported in part by the National Institutes of Health through the University of Texas MD Anderson Cancer Center’s Support Grant (CA016672).

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Bashir, Q., Milton, D.R., Popat, U.R. et al. Allogeneic hematopoietic cell transplantation for patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN). Bone Marrow Transplant (2021).

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