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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 shows venetoclax achieves complete response in some BPDCN patients, suggesting further exploration of treatment strategies.

Key Points

  • To evaluate the clinical outcomes of venetoclax combined with hypomethylating agents in patients with BPDCN.
  • Conducted a multi-institutional retrospective case series of BPDCN patients
  • Collected clinical, genomic, and treatment data from medical records at two major cancer centers
  • Analyzed response rates, duration of response, relapse patterns, and overall survival.
  • Fourteen BPDCN patients treated with venetoclax and hypomethylating agents were included
  • Seventy-five percent of patients achieved complete response in first line therapy
  • Median overall survival was 27 months, with relapse commonly occurring.

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. 1Venetoclax combined with hypomethylating agents for favorable risk acute myeloid leukemia: A single center experience.2024 · 1 citations
  2. 2IDH1 mutation predicts response and survival in treatment-naïve Acute Myeloid Leukemia patients receiving with venetoclax with a hypomethylating agent2025
  3. 3A phase II trial of tagraxofusp, hyper-CVAD, and venetoclax for patients with newly diagnosed or relapsed/refractory BPDCN.2026
  4. 4Evaluation of hypomethylating agents with or without venetoclax in patients with accelerated phase and blast phase myeloproliferative neoplasms: a HERO consortium study2026
  5. 5Clinical 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