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December 8, 2025BloodOpen Access

Real-world use of venetoclax and hypomethylating agents (HMA) in blastic plasmacytoid dendritic cell neoplasm (BPDCN): A multi-institutional case series

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Authors

SSShai ShimonySMShefali Mehra

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Overview

Retrospective case series reveals response rates with venetoclax in BPDCN patients, suggesting viable off-label treatment options.

Key Points

  • Venetoclax shows promise, achieving a 70% overall response rate in off-label use for BPDCN patients.
  • Among 14 patients, the median overall survival was 27 months, with many experiencing relapses involving CNS.
  • Evaluation of treatment outcomes indicates venetoclax retains efficacy across treatment lines with comparable response rates.
  • Heightened need for centralized monitoring of BPDCN due to frequent CNS and extramedullary relapses calls for future studies.

Cite This Study

Shimony et al. (2025) studied this question.

synapsesocial.com/papers/69362f5d4fa91c937236dbe7https://doi.org/10.1182/blood-2025-6401
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Also Consider

Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context:

  1. 1IDH1 mutation predicts response and survival in treatment-naïve Acute Myeloid Leukemia patients receiving with venetoclax with a hypomethylating agent2025
  2. 2Clinical efficacy and safety of venetoclax combined with hypomethylating agents in relapsed high-risk acute myeloid leukemia patients after allogeneic hematopoietic stem cell transplantation2025 · 4 citations
  3. 3Evaluation of venetoclax and hypomethylating agent combination therapy in acute myeloid leukemia: a real-life, single-center experience2026
  4. 4Hypomethylating agent with venetoclax versus hypomethylating agent alone in frontline management of myelodysplastic syndrome - impact of subgroups on clinical outcomes2025
  5. 5Efficacy of a venetoclax-based, anthracycline-free regimen in newly diagnosed CBFβ::MYH11(+) acute myeloid leukemia2025