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Teaching pack: Blastic plasmacytoid dendritic cell neoplasm (BPDCN)

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  1. Teaching pack · Cancer · haematologic

    Blastic plasmacytoid dendritic cell neoplasm (BPDCN)

    A very rare aggressive blood cancer of dendritic-cell precursors that often shows up as bruise-like skin lesions. Two CD123-directed drugs are the first targeted therapies; transplant is still needed for cure.

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  2. What it is

    In two paragraphs

    BPDCN derives from plasmacytoid dendritic cell precursors, expresses CD4, CD56, CD123 (IL-3 receptor alpha), TCF4 and TCL1, and presents with skin lesions, marrow involvement and cytopenias, often evolving to a leukaemic phase. Historically it was treated with ALL- or AML-type chemotherapy with brief responses and a median survival around a year.

    Tagraxofusp (CD123-directed IL-3–diphtheria toxin fusion) was the first BPDCN-specific drug (2018), with ~70% response in untreated patients and capillary leak syndrome as its signature toxicity. Pivekimab sunirine (CD123 ADC) was approved in 2026 (CADENZA). Venetoclax-based regimens and hyper-CVAD are alternatives, and allogeneic transplant in first remission is the only route to long-term survival. CNS prophylaxis is recommended because of frequent occult CNS involvement.

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  3. Standard of care

    What is given today, by setting

    SettingApproachGuideline
    First lineTagraxofusp (monitor albumin for capillary leak) or pivekimab sunirine (2026); alternatives hyper-CVAD or venetoclax-based regimens; CNS prophylaxis.NCCN Category 2A (tagraxofusp)
    ConsolidationAllogeneic HSCT in first complete remission for eligible patients; autologous transplant in selected cases (Japanese data).not mapped
    RelapsedAlternative CD123 agent, venetoclax combinations, clinical trials; prognosis poor.not mapped
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  4. State of the art

    Where the field stands

    • Two CD123-directed approvals (tagraxofusp 2018, pivekimab 2026) give a rare disease dedicated therapies.
    • Transplant in first remission remains essential for cure; response to CD123 agents makes more patients eligible.
    • BCL2 dependence makes venetoclax a rational partner.
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  5. History

    How we got here

    1. 2008WHO names BPDCN
    2. 2018Tagraxofusp approved
    3. 2019Tagraxofusp NEJM pivotal data (Pemmaraju)
    4. 2026Pivekimab sunirine approved
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  6. Pipeline

    What is coming

    • Pivekimab sunirine (product)
    • Venetoclax (product)
    • Allogeneic stem cell transplantation (technology)
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  7. Open problems

    What nobody has solved

    • No randomised trials; sequencing of CD123 agents unknown.
    • Capillary leak syndrome with tagraxofusp.
    • Relapse after transplant.
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  8. Quiz

    Check understanding

    1. Why does venetoclax work in leukaemia?
      Answer
      It blocks BCL-2, the protein that stops leukaemia cells from self-destructing, so the built-in death programme (apoptosis) can run; used in CLL (fixed duration with obinutuzumab) and AML (with azacitidine).
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  9. Sources

    Read the primary sources

    • Tagraxofusp pivotal (NEJM 2019): https://doi.org/10.1056/NEJMoa1815105
    • NCI: BPDCN: https://www.cancer.gov/pediatric-adult-rare-tumor/rare-tumors/rare-blood-cancers/bpdcn
    • Wikipedia: https://en.wikipedia.org/wiki/Blastic_plasmacytoid_dendritic_cell_neoplasm
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