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

Abstract

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.

References

  1. 1.

    Guru Murthy GS, Pemmaraju N, Atallah E. Epidemiology and survival of blastic plasmacytoid dendritic cell neoplasm. Leuk Res. 2018;73:21–3.

    Article  Google Scholar 

  2. 2.

    Arber DA, Orazi A, Hasserjian R, Thiele J, Borowitz MJ, Le Beau MM, et al. The 2016 revision to the World Health Organization classification of myeloid neoplasms and acute leukemia. Blood 2016;127:2391–405.

    CAS  Article  Google Scholar 

  3. 3.

    Deconinck E, Petrella T, Garnache Ottou F. Blastic Plasmacytoid Dendritic Cell Neoplasm: Clinical Presentation and Diagnosis. Hematol Oncol Clin North Am. 2020;34:491–500.

    Article  Google Scholar 

  4. 4.

    Togami K, Chung SS, Madan V, Kenyon CM, Cabal-Hierro L, Taylor J, et al. Sex-biased <em>ZRSR2</em> mutations in myeloid malignancies impair plasmacytoid dendritic cell activation and apoptosis. bioRxiv. 2020:2020.10.29.360503.

  5. 5.

    Sapienza MR, Fuligni F, Agostinelli C, Tripodo C, Righi S, Laginestra MA, et al. Molecular profiling of blastic plasmacytoid dendritic cell neoplasm reveals a unique pattern and suggests selective sensitivity to NF-kB pathway inhibition. Leukemia 2014;28:1606–16.

    CAS  Article  Google Scholar 

  6. 6.

    Villani AC, Satija R, Reynolds G, Sarkizova S, Shekhar K, Fletcher J, et al. Single-cell RNA-seq reveals new types of human blood dendritic cells, monocytes, and progenitors. Science. 2017;356.

  7. 7.

    Beird HC, Khan M, Wang F, Alfayez M, Cai T, Zhao L, et al. Features of non-activation dendritic state and immune deficiency in blastic plasmacytoid dendritic cell neoplasm (BPDCN). Blood Cancer J 2019;9:99.

    Article  Google Scholar 

  8. 8.

    Alayed K, Patel KP, Konoplev S, Singh RR, Routbort MJ, Reddy N, et al. TET2 mutations, myelodysplastic features, and a distinct immunoprofile characterize blastic plasmacytoid dendritic cell neoplasm in the bone marrow. Am J Hematol. 2013;88:1055–61.

    CAS  Article  Google Scholar 

  9. 9.

    Pemmaraju N, Konopleva M. Approval of tagraxofusp-erzs for blastic plasmacytoid dendritic cell neoplasm. Blood Adv. 2020;4:4020–7.

    CAS  Article  Google Scholar 

  10. 10.

    Wang W, Khoury JD, Miranda RN, Jorgensen JL, Xu J, Loghavi S, et al. Immunophenotypic characterization of reactive and neoplastic plasmacytoid dendritic cells permits establishment of a 10-color flow cytometric panel for initial workup and residual disease evaluation of blastic plasmacytoid dendritic cell neoplasm. Haematologica. 2020.

  11. 11.

    Yun S, Chan O, Kerr D, Vincelette ND, Idrees A, Mo Q, et al. Survival outcomes in blastic plasmacytoid dendritic cell neoplasm by first-line treatment and stem cell transplant. Blood Adv. 2020;4:3435–42.

    Article  Google Scholar 

  12. 12.

    Sukswai N, Aung PP, Yin CC, Li S, Wang W, Wang SA, et al. Dual Expression of TCF4 and CD123 Is Highly Sensitive and Specific For Blastic Plasmacytoid Dendritic Cell Neoplasm. Am J Surg Pathol. 2019;43:1429–37.

    Article  Google Scholar 

  13. 13.

    Pemmaraju N, Konopleva M, Lane AA. More on Blastic Plasmacytoid Dendritic-Cell Neoplasms. N. Engl J Med. 2019;380:695–6.

    Article  Google Scholar 

  14. 14.

    Pagano L, Valentini CG, Pulsoni A, Fisogni S, Carluccio P, Mannelli F, et al. Blastic plasmacytoid dendritic cell neoplasm with leukemic presentation: an Italian multicenter study. Haematologica 2013;98:239–46.

    Article  Google Scholar 

  15. 15.

    Taylor J, Haddadin M, Upadhyay VA, Grussie E, Mehta-Shah N, Brunner AM, et al. Multicenter analysis of outcomes in blastic plasmacytoid dendritic cell neoplasm offers a pretargeted therapy benchmark. Blood 2019;134:678–87.

    CAS  Article  Google Scholar 

  16. 16.

    Martin-Martin L, Lopez A, Vidriales B, Caballero MD, Rodrigues AS, Ferreira SI, et al. Classification and clinical behavior of blastic plasmacytoid dendritic cell neoplasms according to their maturation-associated immunophenotypic profile. Oncotarget 2015;6:19204–16.

    Article  Google Scholar 

  17. 17.

    Pemmaraju N, Lane AA, Sweet KL, Stein AS, Vasu S, Blum W, et al. Tagraxofusp in Blastic Plasmacytoid Dendritic-Cell Neoplasm. N. Engl J Med. 2019;380:1628–37.

    CAS  Article  Google Scholar 

  18. 18.

    Roos-Weil D, Dietrich S, Boumendil A, Polge E, Bron D, Carreras E, et al. Stem cell transplantation can provide durable disease control in blastic plasmacytoid dendritic cell neoplasm: a retrospective study from the European Group for Blood and Marrow Transplantation. Blood 2013;121:440–6.

    CAS  Article  Google Scholar 

  19. 19.

    Aoki T, Suzuki R, Kuwatsuka Y, Kako S, Fujimoto K, Taguchi J, et al. Long-term survival following autologous and allogeneic stem cell transplantation for blastic plasmacytoid dendritic cell neoplasm. Blood 2015;125:3559–62.

    CAS  Article  Google Scholar 

  20. 20.

    Kharfan-Dabaja MA, Al Malki MM, Deotare U, Raj RV, El-Jurdi N, Majhail N, et al. Haematopoietic cell transplantation for blastic plasmacytoid dendritic cell neoplasm: a North American multicentre collaborative study. Br J Haematol. 2017;179:781–9.

    CAS  Article  Google Scholar 

  21. 21.

    Leclerc M, Peffault de Latour R, Michallet M, Blaise D, Chevallier P, Rohrlich PS, et al. Can a reduced-intensity conditioning regimen cure blastic plasmacytoid dendritic cell neoplasm? Blood 2017;129:1227–30.

    CAS  Article  Google Scholar 

  22. 22.

    Kharfan-Dabaja MA, Reljic T, Murthy HS, Ayala E, Kumar A. Allogeneic Hematopoietic Cell Transplantation Is an Effective Treatment for Blastic Plasmacytoid Dendritic Cell Neoplasm in First Complete Remission: Systematic Review and Meta-analysis. Clin Lymphoma Myeloma Leuk. 2018;18:703–9 e1.

    Article  Google Scholar 

  23. 23.

    Reimer P, Rudiger T, Kraemer D, Kunzmann V, Weissinger F, Zettl A, et al. What is CD4+CD56+ malignancy and how should it be treated? Bone Marrow Transpl. 2003;32:637–46.

    CAS  Article  Google Scholar 

  24. 24.

    Laribi K, Baugier de Materre A, Sobh M, Cerroni L, Valentini CG, Aoki T, et al. Blastic plasmacytoid dendritic cell neoplasms: results of an international survey on 398 adult patients. Blood Adv. 2020;4:4838–48.

    Article  Google Scholar 

Download references

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Affiliations

Authors

Contributions

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). https://doi.org/10.1038/s41409-021-01478-5

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