Treatment Approaches for Non–Transplant Eligible Patients With BPDCN
An expert discusses how effective blastic plasmacytoid dendritic cell neoplasm (BPDCN) management requires early central nervous system (CNS) evaluation and a multidisciplinary approach, with treatment decisions guided by disease burden, patient fitness, and CNS involvement—highlighting the need for systemic tagraxofusp combined with prompt intrathecal therapy to address high-risk features.
Summary for Physicians: Clinical Considerations in BPDCN Management
Case Highlights:
- The combination of skin lesions, cytopenias, and circulating blasts raised early suspicion for BPDCN.
- Immunophenotyping confirmed the diagnosis with hallmark markers (CD4+, CD56+, CD123+, CD303+, TCL1+).
- CNS involvement at diagnosis (12% cerebral spinal fluid [CSF] blasts) was a key finding, indicating high-risk disease.
Treatment Selection Factors:
- Patient specific: Age, performance status (ECOG 1), and absence of major comorbidities beyond stable cardiac history supported the use of tagraxofusp.
- Non–patient specific: CNS involvement, disease burden, and access to CNS-penetrating agents also influenced initial therapy choice.
CNS Involvement Considerations:
- CNS disease necessitates integrated systemic and intrathecal (IT) therapy from the start.
- Tagraxofusp alone does not penetrate the CNS effectively, reinforcing the need for concurrent IT prophylaxis or treatment.
Intrathecal (IT) Chemotherapy Approach:
- Begin IT therapy promptly alongside systemic treatment.
- Standard regimen: IT methotrexate ± cytarabine and hydrocortisone, administered weekly until CSF clearance, then spaced as prophylaxis.
- Monitor CSF cytology and flow cytometry regularly to assess response and guide tapering.
This case illustrates the critical need for early CNS evaluation and a multidisciplinary strategy in BPDCN, tailoring therapy based on disease characteristics and patient fitness.
