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Combined chelation with high-dose deferiprone and deferoxamine to improve survival and restore cardiac function effectively in patients with transfusion-dependent thalassemia presenting severe cardiac complications

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Abstract

Iron overload–induced cardiomyopathy is the leading cause of death in patients with transfusion-dependent thalassemia (TDT). The mortality is extremely high in these patients with severe cardiac complications, and how to rescue them remains a challenge. It is reasonable to use combined chelation with deferiprone (L1) and deferoxamine (DFO) because of their shuttle and synergistic effects on iron chelation. Here, seven consecutive patients with TDT who had severe cardiac complications between 2002 and 2019 and received combined chelation therapy with oral high-dose L1 (100 mg/kg/day) and continuous 24-h DFO infusion (50 mg/kg/day) in our hospital were reported. Survival for eight consecutive patients receiving DFO monotherapy for their severe cardiac complications between 1984 and 2001 was compared. We found that combined chelation therapy with high-dose L1 and DFO was efficient to improve survival and cardiac function in patients with TDT presenting severe cardiac complications. Reversal of arrhythmia to sinus rhythm was noted in all patients. Their 1-month follow-up left ventricular ejection fraction increased significantly (P < 0.001). There were no deaths, and all patients were discharged from hospital with good quality of life. In contrast, all the eight patients receiving DFO monotherapy died (P < 0.001). Accordingly, combined chelation therapy with high-dose L1 and DFO should be considered in patients with TDT presenting cardiac complications.

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Funding

This study was supported by the Ministry of Science and Technology (MOST 107-2314-B-040-019).

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Authors and Affiliations

  1. Division of Pediatric Cardiology, Children’s Hospital, China Medical University, Taichung, Taiwan

    Tzu-Yao Chuang

  2. Department of Pediatrics, Chung Shan Medical University Hospital, 110, Sec. 1, Chien-Kuo N. Road, Taichung, 402, Taiwan

    Ju-Pi Li, Te-Fu Weng, Kang-Hsi Wu & Yu-Hua Chao

  3. School of Medicine, Chung Shan Medical University, Taichung, Taiwan

    Ju-Pi Li, Kang-Hsi Wu & Yu-Hua Chao

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  1. Tzu-Yao Chuang
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  4. Kang-Hsi Wu
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  5. Yu-Hua Chao
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Contributions

TYC wrote the manuscript. JPL performed the experimental laboratory procedures and data analysis. TFW was responsible for the clinical management of the patients. KHW collected data and interpreted results. YHC designed the study and revised the manuscript. All authors approved the final version of the manuscript.

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Correspondence to Yu-Hua Chao.

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The authors declare that they have no conflict of interest.

Ethical approval

This study was approved by the institutional review board of the China Medical University Hospital, and written informed consents were obtained from the patients. All procedures followed were in accordance with the 1964 Helsinki declaration and its later amendments.

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Chuang, TY., Li, JP., Weng, TF. et al. Combined chelation with high-dose deferiprone and deferoxamine to improve survival and restore cardiac function effectively in patients with transfusion-dependent thalassemia presenting severe cardiac complications. Ann Hematol 99, 2289–2294 (2020). https://doi.org/10.1007/s00277-020-04196-y

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  • DOI: https://doi.org/10.1007/s00277-020-04196-y

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