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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 reports high response rates in newly diagnosed MDS patients, indicating potential benefits of Selinexor with Azacytidine.

Key Points

  • This research aims to evaluate the safety and efficacy of Selinexor sequential treatment with Azacytidine in newly diagnosed patients with MDS.
  • Conducted a single-center, single-arm, phase Ib/II trial
  • Evaluated newly diagnosed patients with MDS-EB1 or EB2
  • Primary endpoints included maximum tolerated dose and overall response rate according to IWG criteria.
  • Overall response rate was 94.4%, with complete remission at 38.9%
  • Patients with TP53 mutations had a 100% overall response rate
  • Maximum tolerated dose established with adverse events primarily from hematologic toxicities.

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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