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December 8, 2025BloodOpen Access

Azacitidine and luspatercept metronomic therapy for intermediate- to high-risk myelodysplastic syndrome: A prospective single-center phase II clinical trial

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Authors

CYChenlu YangYWYu Wu

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Overview

Phase II trial demonstrates metronomic therapy improves quality of life in high-risk MDS patients, suggesting a new treatment approach.

Key Points

  • To evaluate the safety, tolerability, and efficacy of metronomic azacitidine combined with luspatercept in high-risk myelodysplastic syndrome.
  • Conducted a single-center, single-arm phase II clinical trial
  • Enrolled adult patients with intermediate- to high-risk MDS
  • Administered azacitidine and luspatercept according to specified dosing regimens
  • Responses assessed using modified International Working Group criteria
  • Adverse events graded per CTCAE v5.0
  • Overall response rate was 67% with a complete remission rate of 21%
  • 32% of transfusion-dependent patients achieved transfusion independence
  • No grade 4 myelosuppression documented; only transient neutropenia observed
  • 1 death occurred due to pulmonary infection associated respiratory failure

Cite This Study

Yang et al. (2025) studied this question.

synapsesocial.com/papers/693624d44fa91c937236cfaahttps://doi.org/10.1182/blood-2025-5625
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Also Consider

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

  1. 1Luspatercept for the treatment of myelodysplastic syndrome: A retrospective cohort study utilizing trinetx2025
  2. 2SURVIVAL OUTCOMES AND PROGNOSTIC MARKERS IN AZACITIDINE-TREATED MYELODYSPLASTIC SYNDROME: A SINGLE-CENTER EXPERIENCE2026
  3. 3Randomized Phase II Study of Azacitidine Alone or in Combination With Lenalidomide or With Vorinostat in Higher-Risk Myelodysplastic Syndromes and Chronic Myelomonocytic Leukemia: North American Intergroup Study SWOG S11172017 · 239 citations
  4. 4Real-World Clinical Outcomes of Azacitidine Versus Best Supportive Care in Higher-Risk Myelodysplastic Neoplasms: A Single-Center Cohort Study2026
  5. 5Preliminary safety and efficacy of oral azacitidine (Oral-AZA) in patients (pts) with low-/Intermediate (Int)-risk myelodysplastic syndromes (MDS): Phase 2 results from the ASTREON trial.2024 · 2 citations