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September 3, 2026European journal of medical researchOpen Access

Efficacy and prognosis of hypomethylating agent therapy in patients with myelodysplastic syndromes with bone marrow fibrosis

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Authors

YZYoushan ZhaoJGJuan Guo洪洪明华

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Overview

Retrospective analysis reveals poorer survival and higher toxicity with hypomethylating agents in myelodysplastic syndromes with fibrosis, indicating that severe fibrosis impairs long-term outcomes.

Key Points

  • To evaluate the impact of bone marrow fibrosis on treatment response, overall survival, and adverse events in patients with myelodysplastic syndromes with excess blasts undergoing hypomethylating agent monotherapy.
  • Analyzed 200 patients with myelodysplastic syndromes with excess blasts (118 with MDS-EB-1 and 82 with MDS-EB-2; median age 65 years) receiving hypomethylating agent monotherapy between March 2010 and March 2023 at Shanghai Sixth People's Hospital.
  • Assessed response rates, response duration, and safety profiles across fibrosis grades (MF-2/3 vs. MF-0/1) using Kaplan–Meier survival analysis and Cox proportional hazards regression models.
  • Overall response rates (50.9% vs. 53.7%, P = 0.873) and complete remission rates (26.4% vs. 25.2%, P = 1.000) did not differ significantly between MF-2/3 and MF-0/1 cohorts.
  • Patients with MF-2/3 exhibited significantly shorter median overall survival (14 vs. 18 months, P = 0.020; multivariate HR = 1.449, 95% CI 1.022–2.055, P = 0.037) and median duration of response (5 vs. 9 months, P = 0.036).
  • Severe adverse events were more frequent in the MF-2/3 cohort, including grade 4 neutropenia (88.7% vs. 74.8%, P = 0.035), grade 4 infections (66.0% vs. 44.9%, P = 0.008), and treatment-related mortality (26.4% vs. 7.5%, P = 0.001).

Cite This Study

Zhao et al. (2026) studied this question.

synapsesocial.com/papers/6a9934ea636c6408cfa7cb9ehttps://doi.org/10.1186/s40001-026-05127-9
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Also Consider

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

  1. 1Outcomes of hypomethylating agent (HMA)-based therapy in patients with myelodysplastic syndrome with fibrosis2025
  2. 2Outcomes of hypomethylating agent failure in patients with myelodysplastic syndromes2025 · 2 citations
  3. 3Impact of myelofibrosis on patients with myelodysplastic syndromes following allogeneic hematopoietic stem cell transplantation2024 · 3 citations
  4. 4Outcomes of patients with bone marrow fibrosis in de novo and secondary acute myeloid leukemia.2024
  5. 5Management of myelodysplastic syndromes after failure of response to hypomethylating agents2019 · 61 citations