# Blastic plasmacytoid dendritic cell neoplasm (BPDCN)

Source: https://onco.cc/cancers/bpdcn/  
OnCo record `bpdcn` (Cancer). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

Blastic plasmacytoid dendritic cell neoplasm is a rare aggressive leukaemia-like blood cancer of dendritic-cell precursors, a few hundred US cases a year, that often first appears as bruise-like skin lesions. Two CD123-directed drugs, tagraxofusp and pivekimab sunirine, are the first targeted therapies; allogeneic transplant in first remission is still the only route to long-term control.

## Summary

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.

## Fields

- Kind: Cancer
- Last checked: 2026-09-08
- Tags: gap-fill; haematologic; rare
- Group: haematologic
- Burden: BPDCN is very rare (a few hundred cases per year in the US); median age ~65-70, male predominance; skin lesions in most.
- Subtypes: Skin-only presentation; Leukaemic / marrow-involving disease; Cases arising with or from CMML/MDS
- Biomarkers: CD123, CD4, CD56, TCF4, TCL1 immunophenotype; Absence of MPO, lysozyme, CD3; MYC rearrangement (8q24); TET2, ASXL1, ZRSR2 mutations; CSF examination

## Sections of this record

The page is a hub with ten sections in reading order; large sections have their own page. The same plan as JSON: https://onco.cc/api/v1/cancers/bpdcn/sections.json

- Overview (on the hub): The TL;DR, the family this cancer belongs to, the organ, who gets it and what the state of the art is. https://onco.cc/cancers/bpdcn/#overview [3 state-of-the-art points]
- What it is (on the hub): Anatomy, the subtypes and how they differ, how it is staged, and where advanced disease spreads. https://onco.cc/cancers/bpdcn/#what-it-is [3 subtypes]
- Finding it (on the hub): How it shows itself, how it is confirmed, what screening exists, and the biomarkers clinicians test for. https://onco.cc/cancers/bpdcn/#finding-it [5 biomarkers]
- Treating it (on the hub): The standard of care by setting, the medicines, surgery and radiotherapy named in it, and the regimens behind them. https://onco.cc/cancers/bpdcn/#treating-it [3 settings, 3 decisions with options]
- Evidence (on the hub): Trials recruiting now, the landmark trials, the key papers and what they mean, the latest literature, and the milestones year by year. https://onco.cc/cancers/bpdcn/#evidence [1 trial, 1 key paper, 4 milestones]
- The science (on the hub): The molecular landscape: the targets and how often each appears, the pathways, the mechanics stages and the preclinical models. https://onco.cc/cancers/bpdcn/#science [2 targets, 1 pathway]
- Where you are (own page): Cases by country, the UK and NHS pathway and other country lenses, and the expert centres with trials on record. https://onco.cc/cancers/bpdcn/where-you-are/
- Living with it (on the hub): The decisions you may face, the aids that walk through them, the warnings on record, the first sixty days and the questions to ask. https://onco.cc/cancers/bpdcn/#living-with-it [14 questions, 5 red cards]
- What is coming (own page): Everything in development, the open problems and what is being done about them, the roadmaps, and what changed on this record. https://onco.cc/cancers/bpdcn/coming/ [3 medicines, 1 trial, 3 open problems]
- Data (own page): Every connected record, the notes, the JSON, Markdown and RDF twins, and where the record came from and when it was checked. https://onco.cc/cancers/bpdcn/data/ [19 connected records]

## Standard of care

- First line: Tagraxofusp (monitor albumin for capillary leak) or pivekimab sunirine (2026); alternatives hyper-CVAD or venetoclax-based regimens; CNS prophylaxis. ([Tagraxofusp](https://onco.cc/drugs/tagraxofusp/), [Pivekimab sunirine](https://onco.cc/drugs/pivekimab-sunirine/), [Venetoclax](https://onco.cc/drugs/venetoclax/))
- Consolidation: Allogeneic HSCT in first complete remission for eligible patients; autologous transplant in selected cases (Japanese data). ([Allogeneic stem cell transplantation](https://onco.cc/technologies/allogeneic-hsct/), [Autologous stem cell transplant (high-dose therapy)](https://onco.cc/technologies/autologous-stem-cell-transplant/))
- Relapsed: Alternative CD123 agent, venetoclax combinations, clinical trials; prognosis poor. ([Venetoclax](https://onco.cc/drugs/venetoclax/), [Pivekimab sunirine](https://onco.cc/drugs/pivekimab-sunirine/))

## State of the art

- 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.

## Open problems

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

## Sources

- Wikipedia: https://en.wikipedia.org/wiki/Blastic_plasmacytoid_dendritic_cell_neoplasm
- Tagraxofusp pivotal (NEJM 2019): https://doi.org/10.1056/NEJMoa1815105

## Connected records

- cancers: [Acute lymphoblastic leukaemia](https://onco.cc/cancers/all-leukemia/), [Acute myeloid leukaemia](https://onco.cc/cancers/aml/), [Langerhans cell histiocytosis (LCH)](https://onco.cc/cancers/langerhans-cell-histiocytosis/), [Leukaemia (all types)](https://onco.cc/cancers/leukaemia/)
- technologies: [Allogeneic stem cell transplantation](https://onco.cc/technologies/allogeneic-hsct/), [Antibody-drug conjugate (ADC)](https://onco.cc/technologies/adc/), [Autologous stem cell transplant (high-dose therapy)](https://onco.cc/technologies/autologous-stem-cell-transplant/), [Cytokines & engineered cytokines](https://onco.cc/technologies/cytokine-therapy/)
- targets: [BCL-2](https://onco.cc/targets/bcl2/), [CD123](https://onco.cc/targets/cd123/)
- drugs: [Pivekimab sunirine](https://onco.cc/drugs/pivekimab-sunirine/), [Tagraxofusp](https://onco.cc/drugs/tagraxofusp/), [Venetoclax](https://onco.cc/drugs/venetoclax/)
- companies: [AbbVie (incl. ImmunoGen, Capstan)](https://onco.cc/companies/abbvie/), [Menarini / Stemline](https://onco.cc/companies/menarini/)
- pathways: [Intrinsic apoptosis (BCL-2 family)](https://onco.cc/pathways/apoptosis-bcl2/)
- key papers: [Tagraxofusp in Blastic Plasmacytoid Dendritic-Cell Neoplasm](https://onco.cc/key-papers/paper-pemmaraju-n-engl-j-med/)
- terms: [Rare cancers](https://onco.cc/terms/rare-cancers/)
- trials: [Study of IMGN632 in Patients With Untreated BPDCN and Relapsed/Refractory BPDCN](https://onco.cc/trials/nct03386513/)

---
JSON: https://onco.cc/api/v1/entities/bpdcn.json