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Long-term safety and efficacy of hydroxyurea in patients with non-transfusion-dependent β-thalassemia: a comprehensive single-center experience

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Abstract

Over the past 20 years, hydroxyurea (HU) has emerged as an effective therapeutic agent in thalassemic patients to improve anemia and decrease the transfusion dependency. We evaluated long-term safety and clinical response to HU in patients with non-transfusion-dependent β-thalassemia (NTDT). In this retrospective study, medical records of 181 patients with NTDT were evaluated during October to December 2020 in Southern Iran. No requirement to blood transfusion was considered as sustained transfusion independence response. All patients were regularly examined and monitored for the occurrence of any adverse event (AE) of HU. The mean duration of HU consumption ± SD was 18.2 ± 4.0 (8–22) years. Overall, 149 patients (82.3%) had sustained transfusion independence response. β-globin gene mutations and XmnI polymorphisms were not significantly associated with clinical response (P > 0.05). Mild and transient AEs were reported in 60 patients (33%) with no requirement to drug interruption. Hydroxyurea with the dose of 8–15 mg/kg can be used as a safe and effective treatment in NTDT patients. It was well tolerated in long term without any serious complication or secondary malignancy. No relationship between XmnI or β-globin gene mutations with HU response was observed in this geographic area of the world.

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Data availability statement

The data presented in this study are available on request from the corresponding author.

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Acknowledgements

We would like to thank Shiraz University of Medical Sciences for their approval support.

Funding

This research was funded by the Research Vice-Chancellor of Shiraz University of Medical Sciences, grant number (21488).

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Authors and Affiliations

  1. Hematology Research Center, Shiraz University of Medical Sciences, Shiraz, Iran

    Mehran Karimi, Tahereh Zarei & Sezaneh Haghpanah

  2. Health Policy Research Center, Institute of Health, Shiraz University of Medical Sciences, Shiraz, Iran

    Ardeshir Bahmanimehr

  3. Thalassemia and Hemophilia Research Center, Shahid Dastgheib Hospital, Shiraz University of Medical Sciences, Shiraz, Iran

    Ardeshir Bahmanimehr, Azam Aramesh & Saeed Daryanoush

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  1. Mehran Karimi
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  2. Tahereh Zarei
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Contributions

Conceptualization, M.K.; methodology, S.H., T.Z.; data curation: T. Z., A.A; software, S.H. A.B; validation, S.H.; formal analysis, S.H., A.B., A.A., and S.D.; writing original draft preparation, S.H. and T.Z.; writing review and editing, M.K. and S.H; supervision, M.K. and S.H.; project administration, M.K. and S.H.; funding acquisition, S.H. All authors have read and agreed to the published version of the manuscript. All authors have contributed substantially to the work reported.

Corresponding author

Correspondence to Sezaneh Haghpanah.

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Institutional review board statement

The study was conducted according to the guidelines of the Declaration of Helsinki, and approved by the Ethics Committee of Shiraz University of Medical Sciences (protocol code IR.SUMS.REC.1399.805 on September 23th, 2020).

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Written informed consent has been obtained from the patients to publish this paper.

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The authors declare no competing interests.

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The funders had no role in the design of the study; in the collection, analyses, or interpretation of data; in the writing of the manuscript; or in the decision to publish the results.

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Karimi, M., Zarei, T., Bahmanimehr, A. et al. Long-term safety and efficacy of hydroxyurea in patients with non-transfusion-dependent β-thalassemia: a comprehensive single-center experience. Ann Hematol 100, 2901–2907 (2021). https://doi.org/10.1007/s00277-021-04627-4

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