Current Therapies and Strategies for Transplant-Ineligible BPDCN Patients


Dr. Schiller will discuss the limitations of currently available BPDCN treatments, the historical use of chemotherapy regimens and their potential to address unmet needs, and effective strategies for managing the financial burden and ensuring access to tagraxofusp for BPDCN patients.

Case: Management of Elderly Transplant-Ineligible BPDCN Patients

Clinical Presentation:

  • A 74-year-old lady presents with fatigue, bruising like lesion on upper chest, and shortness of breath that had been present for around 6 months.

Initial Clinical Workup and Diagnosis:

  • Initially suspected to have acute myeloid leukemia (AML) based on preliminary workup at a community oncology clinic.
  • Referred to academic cancer center for further evaluation after not responding well to initial AML treatment.
  • PMH: Hypertension, Type II Diabetes, H/O MI in 2015
  • PE: Notable for pallor, petechiae, and no lymphadenopathy or hepatosplenomegaly
  • ECOG PS =2
  • Labs: WBC 5 x 10 K/uL, Hb 7.8 g/dL, platelets 25x 109 /L.
  • Peripheral smear shows 70% blasts.
  • Bone marrow biopsy showed numerous immature cells felt to represent acute leukemia.
  • Cytogenetics: Complex karyotype
  • LDH elevated at 1200 u/L
  • Lives in assisted living facility, requires assistance with activities of daily living.
  • No available matched sibling or unrelated donor identified.
  • Upon referral to the academic center, pathology was reviewed, and additional material assessed leading to diagnoses of BPDCN.

Initial Treatments:

  • Patient was initiated on:
    • Standard-dose cytarabine 150 mg/m2 continuous infusion x 7 days with idarubicin 12 mg/m2 or daunorubicin 60–90 mg/m2 x 3 days10
  • After failure to achieve remission, she received:
    • Tagraxofusp 12 mcg/kg IV over 15 minutes once daily on days 1–5 of a 21-day cycle

This is a synopsis of a Case-Based Peer Perspectives series featuring Gary Schiller, MD, of UCLA David Geffen School of Medicine.

Gary Schiller, MD, Chief of the Hematology Stem Cell Transplant Program at David Geffen School of Medicine, UCLA, discussed the challenges in treating patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN). Dr. Schiller identified the first challenge as making the correct diagnosis. The second challenge is the potential toxicity of tagraxofusp, the only approved drug for the management of BPDCN.

In the pivotal trial that led to the approval of tagraxofusp, capillary leak syndrome was identified as a potential adverse event, with weight gain occurring in 25% of patients and hypotension in about a quarter of the patients. Elevated alanine transaminase (ALT) and aspartate transaminase (AST) levels were less common, occurring in less than 20% of patients, and hypoalbuminemia was seen in 16% of the 89 patients originally treated. Dr. Schiller recommended administering tagraxofusp in the hospital setting, particularly during the first cycle, to monitor for signs of capillary leak syndrome and manage with albumin as appropriate.

Regarding the use of chemotherapy, Dr. Schiller noted that conventional chemotherapy with cytarabine and anthracycline has not been typically helpful for BPDCN. Some reports suggest that venetoclax-based therapy might be useful and effective in an older population. In the past, some clinicians have used acute lymphoblastic leukemia (ALL)-type regimens with vincristine and corticosteroids, but these have not been particularly helpful either.

When administering tagraxofusp, patients must have an albumin level above 3.2 g/dL. If the level is below 3.2 g/dL, patients need to receive albumin supplements until their level is above 3.5 g/dL. Serum albumin levels must be monitored prior to the initiation of each dose, and patients should be assessed for weight gain, new onset or worsening edema, pulmonary edema, hypotension, and hemodynamic instability. The drug may need to be held if patients develop severe symptoms.

Regarding the financial burden related to the use of tagraxofusp, Dr. Schiller mentioned that the manufacturer is available to help obtain approval and support patients depending on their payment plan.

*Video synopsis is AI-generated and reviewed by Targeted Oncology editorial staff.

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