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Venetoclax combined with azacitidine for treatment of a blastic plasmacytoid dendritic cell neoplasm: A case report and literature review

  • Authors:
    • Yunching Lai
    • Xiaolei Chen
    • Hebing Zhou
  • View Affiliations / Copyright

    Affiliations: School of Basic Medicine Sciences, Capital Medical University, Beijing 101100, P.R. China, Department of Hematology, Beijing Luhe Hospital Affiliated to Capital Medical University, Beijing 101100, P.R. China
    Copyright: © Lai et al. This is an open access article distributed under the terms of Creative Commons Attribution License.
  • Article Number: 20
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    Published online on: November 10, 2025
       https://doi.org/10.3892/etm.2025.13015
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Abstract

Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare and highly aggressive hematological malignancy. The neoplasm is associated with a poor prognosis and frequent diagnostic challenges, mainly due to its cutaneous manifestations that can delay an accurate diagnosis or lead to its misclassification as other hematological disorders. In the absence of standardized protocols, the management of BPDCN relies on crucial judgment based on individual cases. The present study reports the case of a 51‑year‑old male with BPDCN who presented with a fever and bilateral limb pain, and achieved complete remission after only one cycle of azacitidine‑venetoclax (VA) therapy. As the patient required concurrent administration of posaconazole (a potent CYP3A4 inhibitor), the dose of venetoclax was adjusted to a low dose of 200 mg per day, and the patient still achieved successful disease remission. The findings from this case study highlight the potential of VA therapy in the management of BPDCN. The study also emphasizes the diversity of the clinical manifestations of BPDCN and the crucial role of combination therapy in its management.
View Figures

Figure 1

Wright-Giemsa-stained BM aspirate
smear images (A and B) before treatment and (C and D) after one
cycle of treatment, with a magnification of x1,000). In the
pre-treatment BM aspirate smear, the cytoplasm is basophilic, with
occasional (A) vacuoles (arrow) and (B) tail-like protrusions
(arrow). (C) After one cycle of treatment, mature erythrocytes,
promyelocytes, myelocytes and other hematopoietic cells were
observed, with no BPDCN cells identified. (D) The remaining fields
showed hematopoietic cells consistent with those in (C), and no
BPDCN cells were identified. BM, bone marrow.

Figure 2

Flow cytometry prior to treatment
indicating that the R3 population consists of abnormal cells,
accounting for ~75.07% of nucleated cells. (A) This abnormal cell
population showed 100% positive expression of CD4 (dim), CD56,
CD123 (dim), CD304, TdT, HLA-DR, CD99, CD2, CD71 (dim) and CD38,
while being negative for CD303, CD3, CD14, CD19, CD34 and MPO. (B)
After one treatment cycle, the proportion of abnormal cells in the
bone marrow decreased to 0.00%, and the positive expression rates
of CD4, CD56, CD123 and CD304 were all 0%. (This figure only
displays the major relevant immunophenotypes and does not present
the complete detection results of all immunophenotypes). FSC,
forward scatter; SSC, side scatter; TdT, terminal deoxynucleotidyl
transferase; HLA-DR, human leukocyte antigen-DR; APC,
allophycocyanin; FITC, fluorescein isothiocyanate; PE,
phycoerythrin.
View References

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Copy and paste a formatted citation
Spandidos Publications style
Lai Y, Chen X and Zhou H: Venetoclax combined with azacitidine for treatment of a blastic plasmacytoid dendritic cell neoplasm: A case report and literature review. Exp Ther Med 31: 20, 2026.
APA
Lai, Y., Chen, X., & Zhou, H. (2026). Venetoclax combined with azacitidine for treatment of a blastic plasmacytoid dendritic cell neoplasm: A case report and literature review. Experimental and Therapeutic Medicine, 31, 20. https://doi.org/10.3892/etm.2025.13015
MLA
Lai, Y., Chen, X., Zhou, H."Venetoclax combined with azacitidine for treatment of a blastic plasmacytoid dendritic cell neoplasm: A case report and literature review". Experimental and Therapeutic Medicine 31.1 (2026): 20.
Chicago
Lai, Y., Chen, X., Zhou, H."Venetoclax combined with azacitidine for treatment of a blastic plasmacytoid dendritic cell neoplasm: A case report and literature review". Experimental and Therapeutic Medicine 31, no. 1 (2026): 20. https://doi.org/10.3892/etm.2025.13015
Copy and paste a formatted citation
x
Spandidos Publications style
Lai Y, Chen X and Zhou H: Venetoclax combined with azacitidine for treatment of a blastic plasmacytoid dendritic cell neoplasm: A case report and literature review. Exp Ther Med 31: 20, 2026.
APA
Lai, Y., Chen, X., & Zhou, H. (2026). Venetoclax combined with azacitidine for treatment of a blastic plasmacytoid dendritic cell neoplasm: A case report and literature review. Experimental and Therapeutic Medicine, 31, 20. https://doi.org/10.3892/etm.2025.13015
MLA
Lai, Y., Chen, X., Zhou, H."Venetoclax combined with azacitidine for treatment of a blastic plasmacytoid dendritic cell neoplasm: A case report and literature review". Experimental and Therapeutic Medicine 31.1 (2026): 20.
Chicago
Lai, Y., Chen, X., Zhou, H."Venetoclax combined with azacitidine for treatment of a blastic plasmacytoid dendritic cell neoplasm: A case report and literature review". Experimental and Therapeutic Medicine 31, no. 1 (2026): 20. https://doi.org/10.3892/etm.2025.13015
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