Exploring the Use of Triplet Therapy to Treat BPDCN

Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare type of blood cancer with various treatment options. Because it is rare, there is not one standardized recommended regimen. Results of a recent research showed that a triplet therapy that combines tagraxofusp (Elzonris), azacitidine, and venetoclax (Venclexta) improved outcomes for people with BPDCN. 

New research is changing the treatment landscape

The right treatment for BPDCN depends on individual factors such as age, overall health, and disease progression. BPDCN is an aggressive blood cancer that presents uniquely in each patient, and understanding the available treatment strategies is important for improving outcomes.

Triplet therapy is a combination of three different medications. A triplet therapy can be a helpful strategy as each medication works in different ways to target cancer cells to lead to more long-lasting remission.  

The first FDA-approved targeted therapy for BPDCN is tagraxofusp (Elzonris). Elzonris works by binding to CD123. CD123 is a protein that is overexpressed on BPDCN cells. Overexpressed means there is more CD123 on BPDCN cells than healthy cells. By targeting this protein, the medication delivers a toxin that eliminates BPDCN cells. 

Doctors often also separately prescribe medications like venetoclax and azacitidine to people with BPDCN. These medications are approved to treat certain types of leukemia, like acute myeloid leukemia (AML) and acute lymphoblastic leukemia (ALL). 

Clinical trial results: Triplet therapy for BPDCN

Recently, researchers at Dana-Farber Cancer Institute completed a phase 2 clinical trial studying the triplet therapy tagraxofusp, venetoclax and azaciditine. The trial included 27 people with BPDCN. There were 16 people with newly diagnosed BPDCN and 11 people with relapsed or refractory BPDCN. 

Relapsed means BPDCN that has come back after treatment previously worked to control the disease. Refractory means other treatments did not work to control the BPDCN. Because relapsed or refractory BPDCN can be more difficult to treat, researchers measured the results for two groups: those with newly diagnosed BPDCN and those with relapsed or refractory BPDCN.

Of those with newly diagnosed BPDCN, 88% achieved a complete response. This is a measurement to understand how effective a treatment is. A complete response means there are no remaining cancer cells. Eleven of the participants had relapsed or refractory BPDCN. Of those, 64% achieved a complete response. 

For people with BPDCN, the next treatment step after remission is a stem cell transplant which offers long-lasting disease control. For patients who had been previously untreated, 63% went on to receive a stem cell transplant. Of those with relapsed or refractory BPDCN, 55% went on to receive a stem cell transplant.  

The study also measured overall survival. Overall survival is -how long a patient lives after treatment.  For those who had not previously been treated, overall survival at 1 to 2 years was 60%. For those who had previously been treated, overall survival was 36% at 1 year and 18% at 2 years.  

The research also found that there were improved outcomes related to capillary leak syndrome, a serious complication where fluid leaks from blood vessels into surrounding tissue.  

Find ongoing BPDCN clinical trials

Clinical trials evaluate the safety and effectiveness of new therapies and play a role in advancing cancer treatment. Ongoing clinical trials are evaluating new therapies that may improve outcomes and extend remission. 

You can access HealthTree’s clinical trial finder to explore active clinical trials. 

HealthTree Clinical Trial Finder

Sources: 

• Triplet Therapy Shows Promise for BPDCN, a rare blood cancer, in Dana-Farber Study  
• The Future of CD123 Targeting in BPCDN Treatment 
• BPDCN Treatment Options That Help Manage Symptoms and Support Better Outcomes