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Real-world bleeding outcomes and product utilization in people with severe-type hemophilia A before and after switching to extended half-life rFVIIIFc prophylaxis therapy

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Abstract

Background

Recombinant factor VIII-Fc (rFVIIIFc) became available in Taiwan in 2018. Before this date, no people with hemophilia A (PwHA) were enrolled in a clinical trial of rFVIIIFc. We investigated changes in bleeding outcomes and product utilization in PwHA switching from rFVIII to rFVIIIFc.

Methods

Data were collected for Taiwanese PwHA (severe-type) who switched from rFVIII to rFVIIIFc, including annualized bleeding rate (ABR) and weekly dose consumption 12 months pre-switch and > 6 months post-switch.

Results

The 51 patients were divided into 3 groups according to their pre-switch treatment: on-demand treatment, intermittent periodic prophylaxis, and regular prophylaxis. In every group, the post-switch median ABR was significantly reduced, with no significant differences between groups. Meanwhile, the post-switch median weekly dose of each group was significantly increased. In 32 patients on pre-switch prophylaxis, switching brought a further reduction in median ABR, associated with a significant increase in median weekly dose. No adverse effects or novel inhibitor development were seen.

Conclusion

This is the first report from Asia on real-world experience of rFVIIIFc, showing that switching to rFVIIIFc prophylaxis led to further reduction in ABR and increase in weekly dose for all patient groups, even those on pre-switch rFVIII prophylaxis.

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Data availability

The data that support the findings of this study are available from the corresponding author upon reasonable request. The data are not publicly available due to privacy or ethical restrictions.

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Acknowledgements

The authors would like to thank Dr. Vivek R. Sharma, Research Scientist and Medical Director at the Adult Hemophilia Program, Division of Medical Oncology/Hematology, University of Louisville School of Medicine, for his critical review and edition of this manuscript.

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Authors and Affiliations

  1. Department of Pediatrics, School of Medicine, College of Medicine, Taipei Medical University, Taipei, Taiwan

    Chia-Yau Chang & Yen-Lin Liu

  2. Department of Pediatrics, Taipei Medical University Hospital, Taipei, Taiwan

    Chia-Yau Chang, Jung-Tzu Ku & Yen-Lin Liu

  3. Hemophilia Center, Taipei Medical University Hospital, Taipei, Taiwan

    Chia-Yau Chang, Shu-Hsia Hu, Yen-Lin Liu, Jia-Ruey Tsai & Chen-Hua Tsai

  4. Division of Hematology/Oncology, Department of Internal Medicine, Tri-Service General Hospital, National Defense Medical Center, No.325, Section 2, Chenggong Road, Nei-Hu 114, Taipei, Taiwan

    Shiue-Wei Lai & Yeu-Chin Chen

  5. Hemophilia Care and Research Center, Tri-Service General Hospital, Taipei, Taiwan

    Shiue-Wei Lai, Shu-Hsia Hu & Yeu-Chin Chen

  6. Department of Pediatrics, Cheng Hsin General Hospital, Taipei, Taiwan

    Mei-Mei Cheng

  7. Division of Hematology/Oncology, Department of Internal Medicine, Taipei Medical University Hospital, Taipei, Taiwan

    Jia-Ruey Tsai

  8. Division of Hematology/Oncology, Department of Internal Medicine, Cheng Hsin General Hospital, Taipei, Taiwan

    Chen-Hua Tsai

  9. Division of Pediatric Hematology/Oncology, Department of Pediatrics, National Cheng Kung University Hospital, Tainan, Taiwan

    Chao-Neng Cheng

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  1. Chia-Yau Chang
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Contributions

C-YC, S-WL, M-MC, S-HH, Y-LL, J-RT, C-HT, C-NC, and Y-CC contributed to the conception and design of the study. C-YC, S-WL, M-MC, J-TK, S-HH, Y-LL, J-RT, C-HT, C-NC, and Y-CC contributed substantially to the data acquisition. C-YC, S-WL, J-TK, S-HH, Y-LL, J-RT, C-HT, and Y-CC contributed to statistical analysis and interpretation of data. All authors critically revised the manuscript and approved the submitted final version.

Corresponding author

Correspondence to Yeu-Chin Chen.

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Chang, CY., Lai, SW., Cheng, MM. et al. Real-world bleeding outcomes and product utilization in people with severe-type hemophilia A before and after switching to extended half-life rFVIIIFc prophylaxis therapy. Int J Hematol 117, 378–387 (2023). https://doi.org/10.1007/s12185-022-03503-9

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  • DOI: https://doi.org/10.1007/s12185-022-03503-9

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