Exploration of Targeted Anti-tumor Therapy

Table 2. Overview of Targeted Therapies for blastic plasmacytoid dendritic cell neoplasm (BPDCN).

Therapeutic agent	Mechanism of action	Molecular target	Key clinical results	Main toxicities	Development status	Reference
Tagraxofusp	Recombinant fusion protein [interleukin-3 (IL-3) + diphtheria toxin]	Interleukin-3 receptor alpha chain (CD123)	Overall response rate (ORR) 75–90% in treatment-naive; bridge to allogeneic stem cell transplantation (allo-SCT).	Capillary leak syndrome (CLS), hepatotoxicity.	Approved (US, EU)	[4]
Pivekimab sunirine (IMGN632)	Antibody-drug conjugate (ADC)	CD123	ORR 21% [acute myeloid leukemia (AML)], promising preliminary activity in BPDCN.	Fever, neutropenia, peripheral edema, veno-occlusive disease.	In clinical trials (Breakthrough Therapy Designation)	[45]
Venetoclax	B-cell lymphoma 2 (BCL2) inhibitor	BCL2	Significant responses in relapsed/refractory (R/R) patients; in clinical trials in combination.	Cytopenias, tumor lysis syndrome.	In clinical trials (for BPDCN)	[46]
Azacitidine	Hypomethylating agent	Epigenetic regulators	Remodels tumor microenvironment (TME), reactivates pro-immunogenic genes.	Cytopenias, gastrointestinal toxicity.	In clinical trials in combination	[24]

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Acknowledgments

During the preparation of this work, the author(s) used an AI-based tool (Gemini) for figure generation. After using this tool, the author(s) reviewed and edited the content as needed in Canva and PowerPoint and take full responsibility for the content of the publication.

Author contributions

PPP: Conceptualization, Investigation, Writing—original draft, Supervision, Funding acquisition, Writing—review & editing. ML, LC: Investigation, Validation, Writing—original draft, Writing—review & editing. VT, PC, IL: Investigation, Validation. All authors read and approved the submitted version.

Conflicts of interest

Pier Paolo Piccaluga, who is the Editorial Board Member and Guest Editor of Exploration of Targeted Anti-tumor Therapy, had no involvement in the decision-making or the review process of this manuscript. The other authors declare no conflicts of interest.

Ethical approval

Not applicable.

Consent to participate

Not applicable.

Consent to publication

Not applicable.

Availability of data and materials

The datasets supporting the findings of this study are available from the corresponding author upon reasonable request.

Funding

This work was supported by the Italian Ministry of Health (RC-2024-2790173), and PRIN 2022NXK38S. The funders had no role in study design, data collection and analysis, decision to publish, or preparation of the manuscript.

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