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Stanozolol for the treatment of anemic lower-risk myelodysplastic syndromes without del(5q) after failure of epoetin alfa: findings from a retrospective study

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Abstract

Options for anemic lower-risk myelodysplastic syndromes (MDS) without del(5q) after failure of erythropoiesis-stimulating agents (ESAs) are very limited. The effectiveness of second-line treatments is uncertain. We retrospectively reviewed the clinical effectiveness and overall survival (OS) of lower-risk MDS without del(5q) patients exclusively treated with stanozolol (STZ) after failure of epoetin alfa. The response was defined according to the 2006 International Working Group (IWG) criteria. Fifty-six patients were included. The median follow-up time was 55 months (range: 5–156 months). Twenty-seven patients (48.2%) achieved hematologic improvement-erythroid response (HI-E). Higher response rates were observed in patients with lower IPSS-R scores (≤3.5, P = 0.008) and hypocellular bone marrow (P = 0.002). In univariate Cox analysis, HI-E was the strongest factor associated with better OS (P = 0.0003). In multivariate Cox, HI-E, age ≤ 50, and transfusion independence (TI) at the onset of STZ were factors associated with better OS. The estimated 5-year OS was 88.6% (68.7–96.2%) and 33.8% (14.9–54.0%) in responders and non-responders (P < 0.01), respectively. The most common side effects included masculinization and liver damage, but they were manageable with supportive measures and dose adjustments. STZ may be considered an alternative treatment in lower-risk MDS after failure of epoetin alfa.

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Data availability

The datasets generated during and/or analyzed during the current study are available from the corresponding author on reasonable request.

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Authors and Affiliations

  1. Department of Hematology, Shuguang Hospital Affiliated to Shanghai University of Traditional Chinese Medicine, 528 Zhangheng Road, Shanghai, 200120, China

    Wei-ying Qu, Lin Zhao, Xv-cheng Tan & Yi-han Zhao

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  1. Wei-ying Qu
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  2. Lin Zhao
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  3. Xv-cheng Tan
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  4. Yi-han Zhao
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Contributions

·Lin Zhao and Wei-ying Qu performed the research.

·Lin Zhao designed the research study.

·Lin Zhao, Xv-cheng Tan, and Yi-han Zhao analyzed the data.

·Lin Zhao and Wei-ying Qu wrote the paper.

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Correspondence to Lin Zhao.

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The protocol of the study was approved by the IRB of Shuguang Hospital affiliated with Shanghai University of TCM.

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The authors declare no competing interests.

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Qu, Wy., Zhao, L., Tan, Xc. et al. Stanozolol for the treatment of anemic lower-risk myelodysplastic syndromes without del(5q) after failure of epoetin alfa: findings from a retrospective study. Ann Hematol 100, 1451–1457 (2021). https://doi.org/10.1007/s00277-021-04508-w

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  • DOI: https://doi.org/10.1007/s00277-021-04508-w

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