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CD4+/CD56+ hematodermic neoplasm (BPDCN) - Clinical Profile, Treatments & Trials | Kisho | Kisho

Rare Disease

CD4+/CD56+ hematodermic neoplasm

MONDO:0019467

Also known as:BPDCNagranular CD4+ CD56+ hematodermic neoplasm/tumoragranular CD4+ natural Killer cell leukaemia

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Overview

An aggressive immature hematologic neoplasm formerly known as blastic NK cell lymphoma, composed of cells with a lymphoblast-like morphology. Recent evidence suggests derivation from a plasmacytoid monocyte. Patients present with cutaneous tumors and bone marrow involvement.

Treatments1 FDA approved

Clinical Trials18 registered · 10 recruiting

Diagnosis

Biomarker and diagnostic research for CD4+/CD56+ hematodermic neoplasm has been reported in the published literature.

Treatment

Compiled from FDA-approved drug records, orphan drug designations, and FDA Adverse Event Reporting System (FAERS) safety data. GeneReviews management guidance is included where available.

1 FDA-approved treatment is available for CD4+/CD56+ hematodermic neoplasm, including TAGRAXOFUSP (ELZONRIS, approved 2018). An additional 2 compounds hold orphan drug designation.

Approved Therapies

Brand Name	Generic Name	Mechanism	Approved	Market Status

Prognosis

Estimated prevalence: 1-5 in 10,000 (Uncommon).

Research Landscape

Sourced from ClinicalTrials.gov and PubMed. Publication types are classified using NLM-curated article metadata; research type breakdowns reflect the nature of published work for this disease.

18 clinical trials registered, 10 recruiting. Interventions under study include drug therapy, biologic therapy, other interventions, and procedural interventions. Pipeline includes 4 PHASE2, 12 PHASE1, 1 EARLY_PHASE1. Research is sponsored by a mix of industry and academic institutions.

NCT ID	Title	Phase	Sponsor	Status
[NCT07007052](https://clinicaltrials.gov/study/NCT07007052)

Data assembled from 5 of 12 sources · Last updated Sep 19, 2026, 2:51 PM UTC

MONDOHPOClinicalTrials.govFDAPubMedHGNCClinVarClinGenGeneReviewsOrphanetOMIMRUSP

Medical References

External Resources

Orphanet

European rare disease database

Orphanet:86870

GARD

Genetic and Rare Diseases Info Center

GARD:10556

Questions for Your Care Team

→What are the benefits and potential side effects of ELZONRIS for managing CD4+/CD56+ hematodermic neoplasm?
→Are any of the 18 active clinical trials a good fit for my situation?
→Are there specialists or centers of excellence for this condition in my area?
→Is there a patient registry I should consider joining?

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Brand Name	Generic Name	Sponsor	Designated	Exclusivity End	Designation Status
autologous T cells transduced with lentiviral vector containing a chimeric antigen receptor directed against CD123	autologous T cells transduced with lentiviral vector containing a chimeric antigen receptor directed against CD123	INSERM UMR 1098	2022	—	Designated
autologous T cells genetically modified to express CD123 specific hinge-optimized CD28-costimulatory chimeric receptor and a truncated human epidermal growth factor receptor	autologous T cells genetically modified to express CD123 specific hinge-optimized CD28-costimulatory chimeric receptor and a truncated human epidermal growth factor receptor	Mustang Bio, Inc.	2018	—	Designated

Research Type	Count	% of Total
Patient case studies	35	31%
Research summaries	27	24%
Clinical study results	13	12%
Other research	10	9%
Disease patterns and progression	10	9%
Laboratory research	7	6%
Testing and diagnosis research	5	4%
New treatment approaches	5	4%

Kisho

CD4+/CD56+ hematodermic neoplasm

Overview

Diagnosis

Treatment

Approved Therapies

Prognosis

Research Landscape

Medical References

External Resources

Orphanet

GARD

Questions for Your Care Team

Questions for Your Care Team

Orphan Drug Designations

Clinical Trials

Recent Publications

CD4+/CD56+ hematodermic neoplasm

Overview

Diagnosis

Treatment

Approved Therapies

Prognosis

Research Landscape

Medical References

External Resources

Orphanet

GARD

Related Diseases

Questions for Your Care Team

Questions for Your Care Team

Orphan Drug Designations

Clinical Trials

Recent Publications