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International Journal of Hematology

, Volume 107, Issue 2, pp 173–184 | Cite as

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

  • Keita Kirito
  • Kenshi Suzuki
  • Koichi Miyamura
  • Masahiro Takeuchi
  • Hiroshi Handa
  • Shinichiro Okamoto
  • Brian Gadbaw
  • Kyosuke Yamauchi
  • Taro Amagasaki
  • Kazuo Ito
  • Masayuki Hino
Original Article

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.

Keywords

Polycythemia vera JAK inhibitor Ruxolitinib Japan Hematocrit 

Notes

Acknowledgements

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

Compliance with ethical standards

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.

Supplementary material

12185_2017_2333_MOESM1_ESM.docx (3.2 mb)
Supplementary material 1 (DOCX 3260 kb)

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Copyright information

© The Japanese Society of Hematology 2017

Authors and Affiliations

  • Keita Kirito
    • 1
  • Kenshi Suzuki
    • 2
  • Koichi Miyamura
    • 3
  • Masahiro Takeuchi
    • 4
  • Hiroshi Handa
    • 5
  • Shinichiro Okamoto
    • 6
  • Brian Gadbaw
    • 7
  • Kyosuke Yamauchi
    • 8
  • Taro Amagasaki
    • 8
  • Kazuo Ito
    • 8
  • Masayuki Hino
    • 9
  1. 1.Department of Hematology and OncologyUniversity of YamanashiYamanashiJapan
  2. 2.Department of HematologyJapanese Red Cross Medical CenterTokyoJapan
  3. 3.Department of HematologyNagoya Daiichi HospitalNagoyaJapan
  4. 4.Department of HematologyChiba University HospitalChibaJapan
  5. 5.Department of MedicineGunma University HospitalGunmaJapan
  6. 6.Hematology Division, Department of MedicineKeio University HospitalTokyoJapan
  7. 7.Novartis Pharmaceuticals CorporationEast HanoverUSA
  8. 8.Novartis Pharma KKTokyoJapan
  9. 9.Department of HematologyOsaka City University HospitalOsakaJapan

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