This Treatment for BPDCN is Having an Impact on the Lives of Patients


A new treatment was recently tested for blastic plasmacytoid dendritic cell neoplasm (BPDCN). A recent article in MD Anderson News points out that in the U.S., five hundred to one thousand cases, primarily involving older people, are reported each year.

BPDCN occurs in an immune cell which is typically found in bone marrow, skin, lymph nodes, blood, and spleen. Until recently, there have been few treatment options.

And Now a New Treatment Option

Dr. Naveen Pemmaraju at MD Anderson led a clinical trial to test the drug tagraxofusp that showed promising results. The drug was approved by the FDA in 2018. Tagraxofusp has become the standard treatment for BPDCN.

Dr. Pemmaraju was pleased to report that a response was achieved by more than ninety percent of patients receiving the treatment. In a disease such as BPDCN, “response” would indicate that one of its common symptoms, lesions in either skin or bone marrow, has disappeared.

Adding to the positive response, the New England Journal of Medicine (NEJM) published a paper showing results of a two-year follow-up. The Journal acknowledged that the median survival time for participants had not yet been reached. However, survival time for these subjects up to that point was impressive, as it had doubled compared to historic expectations.

Following the Markers

The markers on the surface of cancer cells bond with molecules and proteins or other markers. The marker on BPDCN cells is called CD123. The newly developed drug tagraxofusp is a targeted therapy that binds to CD123, killing the cancer cells. According to Dr. Pemmaraju, the results have been very encouraging.

About BPDCN’s Most Common Symptoms

The majority of BPDCN patients experience skin lesions. The lesions may appear on a person’s face, neck, arms, legs and other parts of the body. Other symptoms may be a swollen spleen or lymph nodes. Thus BPDCN mimics skin cancer, lymphoma, and leukemia.

These similarities may be responsible for the high rate of failure to date in treating BPDCN patients. The chemotherapies used in the past were  “borrowed” from diseases such as leukemias and lymphoma. They are powerful drugs designed for younger patients with different diseases.

The Uncertainty Surrounding Stem Cell Transplants (SCT)

Stem cell transplants are often recommended as the only cure for certain types of cancer. Tagraxofusop, however, has yet to be tested in many areas and SCT is one of those areas.

The Next Step

Dr. Pemmaraju referenced the substantial progress that has been made in the treatment of BPDCN and then alluded to many questions that are still unanswered such as:

  • Does a patient’s major response leave a small amount of cancer remaining?
  • How many patients reach a five-year survival point?
  • Do patients remain on the same drug cycle or change treatment?

Dr. Pemmaraju advises patients to ask questions and never hesitate to seek second opinions, preferably at major cancer centers.

What questions about BPDCN would you like to add to this article?

Rose Duesterwald

Rose Duesterwald

Rose became acquainted with Patient Worthy after her husband was diagnosed with Acute Myeloid Leukemia (AML) six years ago. During this period of partial remission, Rose researched investigational drugs to be prepared in the event of a relapse. Her husband died February 12, 2021 with a rare and unexplained occurrence of liver cancer possibly unrelated to AML.

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