Opinion: ADC Offers Hope for Patients with Rare Leukemia

Amanda Brink, DNP, APRN, FNP-BC, AOCNP

Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare and aggressive hematologic malignancy that originates from plasmacytoid dendritic cells, which are part of the body’s immune system. BPDCN accounts for less than 1% of all acute leukemias¹ and primarily affects older adults, with a median age at diagnosis of 65 years.² However, it can even occur in pediatric patients.³ The disease shows a slight male predominance.⁴

BPDCN often presents initially with characteristic skin lesions, such as bruise-like patches or nodules.² The bone marrow, lymph nodes, and sometimes the central nervous system are frequently involved as well. Because BPDCN is rare and shares features with other hematologic cancers, diagnosis can be challenging.

The disease’s aggressive nature leads to rapid progression without prompt and specialized treatment. For oncology nurses, understanding the clinical presentation, diagnostic challenges, and emerging treatment options for BPDCN is vital in facilitating early recognition of this disease.

As new targeted therapies become available for BPDCN, the role of oncology nurses expands to include patient education and support throughout treatment.

Recent Advances in BPDCN Treatment

Recent data presented at the American Society of Clinical Oncology 2025 annual meeting by Naveen Pemmaraju, MD, discussed findings from the phase 1/2 CADENZA trial (NCT03386513) targeting CD123, a protein highly expressed on BPDCN cells.⁵ Pivekimab sunirine (PVEK) is a first-in-class antibody-drug conjugate designed to deliver targeted chemotherapy directly to these cells.

The clinical trial evaluated PVEK in adults with both newly diagnosed (frontline) and relapsed/refractory BPDCN. Patients received PVEK intravenously every 21 days as an outpatient infusion. Among frontline patients, 70% (95% CI, 51.3%-84.4%) achieved complete or clinical complete response (CR; cCR), with a median duration of response (DOR) of 9.8 months (95% CI, 4.6-not reached [NR]). The overall response rate (ORR) was 85%, and median overall survival (OS) exceeded 16 months (OS, 16.6; 95% CI, 11.4-NR). Notably, 39% of frontline patients were able to proceed to stem cell transplant, with even higher response rates observed in this group. Patients with relapsed/refractory disease had lower response rates but still showed some benefit.

PVEK was generally well tolerated. The most common treatment-emergent adverse event (TEAE) was peripheral edema, and no cases of capillary leak syndrome (CLS), a condition where fluid leaks from small blood vessels causing swelling and low blood pressure, which is a serious concern with some BPDCN treatments, were reported. A few patients experienced veno-occlusive disease (VOD), a blockage of small veins in the liver that can cause liver damage.⁶ Management of VOD typically includes supportive care such as fluid and electrolyte balance, pain control, and close monitoring of liver function. In more severe cases, treatment with defibrotide, a medication that helps protect blood vessels and improve blood flow in the liver, may be used. Early recognition and prompt intervention of VOD are critical to improving outcomes.

Nursing Considerations

Oncology nurses caring for patients receiving PVEK for BPDCN should closely monitor for common side effects such as peripheral edema and promptly manage symptoms to prevent complications. Although no cases of CLS were reported in the trial, nurses should remain vigilant for signs like sudden swelling, low blood pressure, and breathing difficulties, as early recognition is key. Additionally, nurses should watch for symptoms of VOD, including abdominal pain, jaundice, and unexplained weight gain, and ensure timely communication with the healthcare team for prompt intervention.

Since many frontline patients may proceed to stem cell transplant after PVEK therapy, nurses should prepare patients and caregivers for transplant-related care and provide symptom management throughout the treatment journey.

References

1. Bueno C, Almeida J, Lucio P, et al. Incidence and characteristics of CD4(+)/HLA DRhi dendritic cell malignancies. Haematologica. 2004;89(1):58-69.
2. Cazzato G, Capuzzolo M, Bellitti E, et al. Blastic plasmocytoid dendritic cell neoplasm (BPDCN): clinical features and histopathology with a therapeutic overview. Hematol Rep. 2023;15(4):696-706. Published 2023 Dec 8. doi:10.3390/hematolrep15040070
3. Bai J, Wang S, Fang H, Qiao J. Blastic plasmacytoid dendritic cell neoplasm presenting with bruise-like macules: case report and literature review. Pediatr Dermatol. Published online June 2, 2025. doi:10.1111/pde.15931
4. Togami K, Chung SS, Madan V, et al. Sex-Biased ZRSR2 Mutations in Myeloid Malignancies Impair Plasmacytoid Dendritic Cell Activation and Apoptosis. Cancer Discov. 2022;12(2):522-541. doi:10.1158/2159-8290.CD-20-1513
5. Pemmaraju N, et al. Efficacy and safety of pivekimab sunirine (PVEK) in patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN) in the CADENZA study. J Clin Oncol. 2025;43(16_suppl):6502. doi:10.1200/JCO.2025.43.16_suppl.6502
6. Canadian Cancer Society. Veno-occlusive disease (VOD). Published 2025. Accessed June 8, 2025. https://cancer.ca/en/treatments/side-effects/veno-occlusive-disease