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Targeting CD123 in BPDCN: an emerging field
Adam J DiPippo1, Nathaniel R Wilson2, Naveen Pemmaraju3
1Clinical Pharmacy Specialist, Pharmacy Clinical Programs, The University of Texas Md Anderson Cancer Center, Houston,Texas US.
Introduction:
Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare and aggressive hematologic malignancy with historically poor outcomes for patients, often refractory to traditional chemotherapy. Recent research has focused on targeted therapy to improve responses and limit potential toxicity.
Areas Covered:
CD123 (also known as IL-3 Rα) is a cell surface marker and attractive therapeutic target for many myeloid malignancies, particularly BPDCN, whose cells ubiquitously overexpress CD123. We review the history of CD123 research regarding BPDCN, recent advances including FDA approval of tagraxofusp (formerly SL-401) for BPDCN, and ongoing clinical studies utilizing novel therapeutic strategies to target CD123.
Expert Opinion:
The approval of tagraxofusp for the treatment of BPDCN in December 2018 drastically changed the treatment landscape for patients with this rare neoplasm. While tagraxofusp is better tolerated than traditional multi-agent chemotherapy regimens, it requires close monitoring and sound clinical judgment by providers to prevent and mitigate severe treatment-related complications with special attention to the recognition and management of capillary leak syndrome (CLS). Several other promising strategies for targeting CD123 in BPDCN are currently under investigation, including antibody-drug conjugates, T-cell engagers, and CAR-T cellular therapeutics. These CD123 targeted approaches may soon become standard of care for patients with this difficult to treat malignancy.
Insights
Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare cancer. Targeting the CD123 marker with therapies like tagraxofusp has improved outcomes and offers new hope for patients.
Area of Science:
- Hematologic Malignancies
- Oncology
- Immunotherapy
Background:
- Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare, aggressive hematologic malignancy.
- BPDCN is often refractory to traditional chemotherapy, leading to poor patient outcomes.
- CD123, a cell surface marker, is overexpressed on BPDCN cells, making it an attractive therapeutic target.
Purpose of the Study:
- To review the history of CD123 research in BPDCN.
- To discuss recent advances in CD123-targeted therapies for BPDCN.
- To highlight ongoing clinical studies and novel therapeutic strategies.
Main Methods:
- Review of historical CD123 research in BPDCN.
- Analysis of recent clinical advances and FDA-approved therapies.
- Examination of ongoing clinical trials for novel CD123-targeted agents.
Main Results:
- The FDA approval of tagraxofusp (SL-401) in 2018 significantly altered BPDCN treatment.
- Tagraxofusp, while better tolerated than chemotherapy, requires careful monitoring for complications like capillary leak syndrome (CLS).
- Several novel CD123-targeted strategies, including antibody-drug conjugates, T-cell engagers, and CAR-T therapies, are under investigation.
Conclusions:
- Targeting CD123 has transformed BPDCN treatment, offering improved responses and reduced toxicity.
- Close monitoring and management of treatment-related complications, such as CLS, are crucial for tagraxofusp therapy.
- Emerging CD123-targeted therapies hold promise to become the future standard of care for BPDCN.
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