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Analysis of the Japanese subgroup in LEOPOLD II: a phase 2/3 study of BAY 81-8973, a new recombinant factor VIII product

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Abstract

BAY 81-8973, a new full length recombinant FVIII product, has been developed for prophylaxis and on-demand therapy in patients with hemophilia A. LEOPOLD II was a phase 2/3 study comparing prophylaxis versus on-demand treatment with BAY 81-8973. The analysis herein evaluated the clinical profile in Japanese subjects enrolled in LEOPOLD II. The LEOPOLD II was an open-label randomized crossover study. Our analysis evaluated the efficacy using the annualized bleeding rate, safety, and pharmacokinetics in Japanese subjects with severe hemophilia A enrolled in LEOPOLD II. The median annualized bleeding rate was 59.9/year in the on-demand group and 1.9/year in the prophylaxis group for Japanese subjects. There were no study drug-related adverse events in the Japanese subjects. None of the subjects developed FVIII inhibitors. There were no apparent clinical differences in efficacy, safety, and pharmacokinetics between the Japanese and the non-Japanese subjects. Data for the Japanese subjects showed annualized bleeding rates to be remarkably lower in the prophylaxis group compared to the on-demand group and that BAY 81-8973 exhibited a good safety profile and tolerability. These results were similar for the non-Japanese subjects. The results support adoption of BAY 81-8973 for treatment of Japanese subjects with severe hemophilia A.

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Acknowledgements

This study was funded by Bayer HealthCare Pharmaceuticals. Medical writing assistance was provided by Springer Healthcare Communications and Tetsuhiko Yokoyama and was fully funded by Bayer Yakuhin, Ltd.

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Authors and Affiliations

  1. Hiroshima University Hospital, 1-2-3, Kasumi, Minami-ku, Hiroshima, 734-8511, Japan

    Teruhisa Fujii

  2. Ogikubo Hospital, Suginami-ku, Tokyo, Japan

    Hideji Hanabusa

  3. Nara Medical University Hospital, Kashihara, Nara, Japan

    Midori Shima

  4. Bayer Yakuhin, Ltd., Kita-ku, Osaka, Japan

    Takeshi Morinaga

  5. Tokyo Medical University Hospital, Shinjuku-ku, Tokyo, Japan

    Katsuyuki Fukutake

Authors
  1. Teruhisa Fujii
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  2. Hideji Hanabusa
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  3. Midori Shima
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  4. Takeshi Morinaga
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  5. Katsuyuki Fukutake
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Corresponding author

Correspondence to Teruhisa Fujii.

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Conflict of interest

T. Fujii, H. Hanabusa, M. Shima, and M. Fukutake participated as investigators in the LEOPOLD II clinical trial conducted by Bayer. T. Morinaga is an employee of Bayer Yakuhin, Ltd.

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Fujii, T., Hanabusa, H., Shima, M. et al. Analysis of the Japanese subgroup in LEOPOLD II: a phase 2/3 study of BAY 81-8973, a new recombinant factor VIII product. Int J Hematol 105, 280–286 (2017). https://doi.org/10.1007/s12185-016-2133-9

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  • DOI: https://doi.org/10.1007/s12185-016-2133-9

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