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December 8, 2025Blood

Safety and efficacy of selinexor sequential azacytidine in newly diagnosed patients with myelodysplastic syndromes EB1 or EB2: A single-center, single-arm, phase ib/II trial

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Authors

LMLiya MaWYWenli YangXZXinping Zhou

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Overview

Single-arm trial found high response rates with Selinexor plus azacitidine in patients with MDS, suggesting effective therapy options.

Key Points

  • The overall response rate was high at 94.4%, with significant complete response noted among patients with TP53 mutations.
  • Patients experienced adverse events, most commonly neutropenia and anemia, emphasizing the need for monitoring during treatment.
  • Selinexor acts as an oral inhibitor, providing a novel approach for treating high-risk myelodysplastic syndromes in this cohort.
  • Results imply the combination therapy may improve outcomes for MDS patients, highlighting a potential new standard of care.

Cite This Study

Ma et al. (2025) studied this question.

synapsesocial.com/papers/69362f694fa91c937236df7fhttps://doi.org/10.1182/blood-2025-5637
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