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Ruxolitinib is effective and safe in Japanese patients with hydroxyurea-resistant or hydroxyurea-intolerant polycythemia vera with splenomegaly

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Abstract

Ruxolitinib, a potent JAK1/JAK2 inhibitor, was found to be superior to the best available therapy (BAT) in controlling hematocrit, reducing splenomegaly, and improving symptoms in the phase 3 RESPONSE study of patients with polycythemia vera with splenomegaly who experienced an inadequate response to or adverse effects from hydroxyurea. We report findings from a subgroup analysis of Japanese patients in RESPONSE (n = 18). The composite response rate (hematocrit control and spleen response) was higher in patients receiving ruxolitinib (50.0%) than in those receiving BAT (8.3%). A total of 50.0% of patients randomized to ruxolitinib achieved a spleen response vs 8.3% of those receiving BAT; 100 and 33.3% of patients in the respective groups achieved hematocrit control, with mean hematocrit in ruxolitinib-treated patients remaining stable at < 45% throughout the study. Similarly, a higher proportion of ruxolitinib-treated patients achieved complete hematologic remission (33.3 vs 16.7%). Ruxolitinib also led to rapid improvements in pruritus. All responses with ruxolitinib were durable to week 80, and its safety profile was consistent with that in the overall study. These findings suggest that ruxolitinib is an effective and well-tolerated treatment option for Japanese patients with polycythemia vera with an inadequate response to or adverse effects from hydroxyurea.

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Acknowledgements

Editorial assistance was provided by Karen Chinchilla, Ph.D., and was funded by Novartis Pharma KK.

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Authors and Affiliations

  1. Department of Hematology and Oncology, University of Yamanashi, 1110 Simokawakita, Chuou-shi, Yamanashi, 409-3898, Japan

    Keita Kirito

  2. Department of Hematology, Japanese Red Cross Medical Center, Tokyo, Japan

    Kenshi Suzuki

  3. Department of Hematology, Nagoya Daiichi Hospital, Nagoya, Japan

    Koichi Miyamura

  4. Department of Hematology, Chiba University Hospital, Chiba, Japan

    Masahiro Takeuchi

  5. Department of Medicine, Gunma University Hospital, Gunma, Japan

    Hiroshi Handa

  6. Hematology Division, Department of Medicine, Keio University Hospital, Tokyo, Japan

    Shinichiro Okamoto

  7. Novartis Pharmaceuticals Corporation, East Hanover, NJ, USA

    Brian Gadbaw

  8. Novartis Pharma KK, Tokyo, Japan

    Kyosuke Yamauchi, Taro Amagasaki & Kazuo Ito

  9. Department of Hematology, Osaka City University Hospital, Osaka, Japan

    Masayuki Hino

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  1. Keita Kirito
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Correspondence to Keita Kirito.

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Conflict of interest

K. K. has received personal fees from Novartis Pharma KK, M. H. has received grants and personal fees from Novartis Pharma KK, K. M. has received personal fees from Novartis Pharmaceuticals Corporation, Nippon Shinyaku Co Ltd, Pfizer Inc, and Alexion Pharmaceuticals, Inc; B. G. is an employee of Novartis Pharmaceuticals Corporation. T. A., K. Y., and K. I. are employees of Novartis Pharma K. K. M. T., H. H., S. O., and K. S. have nothing to disclose.

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Kirito, K., Suzuki, K., Miyamura, K. et al. Ruxolitinib is effective and safe in Japanese patients with hydroxyurea-resistant or hydroxyurea-intolerant polycythemia vera with splenomegaly. Int J Hematol 107, 173–184 (2018). https://doi.org/10.1007/s12185-017-2333-y

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  • DOI: https://doi.org/10.1007/s12185-017-2333-y

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