BPDCN Treatment Options That Help Manage Symptoms and Support Better Outcomes

Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare type of blood cancer that can involve the skin, bone marrow, lymph nodes, and central nervous system. It often appears with skin lesions and progresses quickly, making early identification and tailored treatment important for care planning.

There are various treatment options for BPDCN, but no standardized recommended regimen. The appropriate therapy depends heavily on individual factors such as age, overall health, and disease progression.

Because BPDCN is aggressive and presents uniquely in each patient, understanding the available treatment strategies is important for improving outcomes.

Treatment Goals: Controlling Symptoms and Prolonging Life 

There is currently no cure for BPDCN. However, available therapies help to manage symptoms, reduce cancer cell levels, and extend survival.  Some of the therapies commonly used in treating BPDCN include chemotherapy, targeted therapy, and stem cell transplant.  Clinical trials continue to explore new treatment approaches that may improve long-term outcomes.

Chemotherapy remains a commonly used initial treatment for BPDCN, especially in individuals diagnosed early

Chemotherapy: A Commonly Used Treatment for BPDCN

Chemotherapy is a treatment that uses medication to slow or stop the growth of cancer cells.

In the absence of BPDCN-specific therapies, providers have historically relied on chemotherapy regimens developed to treat acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), and certain lymphomas.  

As our understanding of BPDCN biology has advanced, newer therapies have emerged that focus on specific molecular targets to improve effectiveness and reduce side effects. 

Targeted Therapy: Focusing on Specific Markers 

Targeted therapy uses medicines to focus on specific proteins or markers found on cancer cells, while aiming to reduce impact on healthy cells.  

 The first FDA approved targeted therapy for BPDCN is tagraxofusp (Elzonris), a CD123-directed cytotoxin. Elzonris works by binding to CD123, a protein that is overexpressed on BPDCN cells, and delivering a toxin that eliminates the cells. 

 As treatment responses improve, other options like stem cell transplantation may play a role in broader care planning, depending on the individual's overall health and response to therapy.

Allogeneic Stem Cell Transplantation: A Potential Option for Long-Term Remission

Stem cell transplant (SCT) is a treatment used to replace cells that have been damaged by cancer or by treatments such as chemotherapy or radiation. 

An allogeneic stem-cell transplant (allo-SCT) uses stem cells from a donor rather than the individual receiving the transplant. This option is often considered for people who respond well to initial treatment and are healthy enough to undergo the procedure, as it can offer longer remission, especially when performed early.

Staying Informed and Participating in Research

In addition to chemotherapy, targeted therapy, and stem cell transplantation, other treatment approaches for BPDCN are being explored, including immunotherapies, combination regimens, and supportive care strategies tailored to individual needs, disease progression, and central nervous system involvement.

Ongoing clinical trials are evaluating new therapies that may improve outcomes and extend remission. Staying informed and partnering with a specialized care team ensures access to the most up-to-date, personalized care.

Treatment decisions are guided by individual factors and current clinical evidence. Your care team will work with you to develop a plan aligned with your specific needs and goals

You can contribute to real-world data studies by completing simple surveys in HealthTree Cure Hub. Your participation helps improve treatment decisions and outcomes for others living with blood cancer.

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Sources:

• How I treat Blastic Plasmacytoid Dendritic Cell Neoplasm
• Leukemia and Lymphoma Society: Stem Cell Transplant
• Facts About Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)