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Clinical characteristics of Japanese patients with myelodysplastic/myeloproliferative neoplasm with ring sideroblasts and thrombocytosis

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Abstract

Myelodysplastic/myeloproliferative neoplasm with ring sideroblasts and thrombocytosis (MDS/MPN-RS-T) is a rare disease, which presents with features of myelodysplastic syndromes with ring sideroblasts and essential thrombocythemia, as well as anemia and marked thrombocytosis. SF3B1 and JAK2 mutations are often found in patients, and are associated with their specific clinical features. This study was a retrospective analysis of 34 Japanese patients with MDS/MPN-RS-T. Median age at diagnosis was 77 (range, 51–88) years, and patients had anemia (median hemoglobin: 9.0 g/dL) and thrombocytosis (median platelet count: 642 × 109/L). Median overall survival was 70 (95% confidence interval: 68-not applicable) months during the median follow-up period of 26 (range: 0–91) months. A JAK2V617F mutation was detected in 46.2% (n = 12) of analyzed patients (n = 26), while an SF3B1 mutation was detected in 87.5% (n = 7) of analyzed patients (n = 8). Like those with myelodysplastic syndromes or myeloproliferative neoplasms, patients often received erythropoiesis-stimulating agents and aspirin to improve anemia and prevent thrombosis. This study, which was the largest to describe the real-world characteristics of Japanese patients with MDS/MPN-RS-T, showed that the patients had similar characteristics to those in western countries.

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Authors and Affiliations

  1. Department of Advanced Hematology, Juntendo University Graduate School of Medicine, 2-1-1 Hongou, Bunkyo-Ku, Tokyo, Japan

    Yoko Edahiro, Tomonori Ochiai, Yoshinori Hashimoto, Soji Morishita, Shuichi Shirane & Norio Komatsu

  2. Department of Hematology, Juntendo University School of Medicine, Tokyo, Japan

    Yoko Edahiro, Tomonori Ochiai, Shuichi Shirane, Tadaaki Inano, Chiho Furuya, Yasuo Aota, Miki Ando & Norio Komatsu

  3. Laboratory for the Development of Therapies Against MPN, Juntendo University Graduate School of Medicine, Tokyo, Japan

    Yoko Edahiro, Tomonori Ochiai, Soji Morishita, Shuichi Shirane & Norio Komatsu

  4. Department of Hematology, Tottori Prefectural Central Hospital, Tottori, Japan

    Yoshinori Hashimoto & Hiromi Omura

  5. Department of Hematology, Juntendo University Shizuoka Hospital, Izunokuni, Japan

    Michiaki Koike

  6. Department of Hematology, Juntendo University Urayasu Hospital, Urayasu, Japan

    Masaaki Noguchi

  7. Department of Hematology, NTT Medical Center Tokyo, Tokyo, Japan

    Kensuke Usuki

  8. Department of Hematology, Iizuka Hospital, Iizuka, Japan

    Motoaki Shiratsuchi

  9. Department of Hematology/Oncology, University of Yamanashi, Kofu, Japan

    Kei Nakajima

  10. Department of Hematology, Oita Prefectural Hospital, Oita, Japan

    Eiichi Ohtsuka

  11. Department of Hematology, Asahi General Hospital, Asahi, Japan

    Hiroaki Tanaka

  12. Department of Hematology, Matsushita Memorial Hospital, Moriguchi, Japan

    Eri Kawata

  13. Department of Hematology, Graduate School of Medicine, Osaka Metropolitan University, Osaka, Japan

    Mika Nakamae

  14. Department of Hematology/Oncology, Kurashiki Central Hospital, Kurashiki, Japan

    Yasunori Ueda

  15. Department of Internal Medicine, Kohsei Chuo General Hospital, Tokyo, Japan

    Yasuo Aota

  16. Department of Hematology, Oami Municipal Hospital, Oamishirasato, Japan

    Yasumasa Sugita

  17. Department of Hematology, Eiju General Hospital, Tokyo, Japan

    Shin Ohara

  18. Department of Internal Medicine, Kyushu University Beppu Hospital, Beppu, Japan

    Satoshi Yamasaki

  19. Department of Hematology & Oncology, Shiga General Hospital, Moriyama, Japan

    Kohsuke Asagoe

  20. Department of Hematology, National Hospital Organization, Kyushu Medical Center, Fukuoka, Japan

    Shuro Yoshida

  21. Division of Blood Transfusion and Cell Therapy, Ehime University Hospital, Toon, Japan

    Jun Yamanouchi

  22. Department of Hematology, Tottori University Hospital, Yonago, Japan

    Sayaka Suzuki

  23. Department of Hematology, Kawasaki Medical School, Kurashiki, Japan

    Toshinori Kondo

  24. Department of Hematology and Oncology, Oji General Hospital, Tomakomai, Japan

    Yuji Kanisawa

  25. Department of Hematology, Fujioka General Hospital, Fujioka, Japan

    Kohtaro Toyama

  26. Department of Hematology, Tokyo Teishin Hospital, Tokyo, Japan

    Daisuke Mizuchi

  27. Department of Hematology, Hokuou Hospital, Sapporo, Japan

    Sumio Sakamaki

  28. PharmaEssentia Japan KK, Tokyo, Japan

    Norio Komatsu

Authors
  1. Yoko Edahiro
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  2. Tomonori Ochiai
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  3. Yoshinori Hashimoto
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  4. Soji Morishita
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  5. Shuichi Shirane
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  6. Tadaaki Inano
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  7. Chiho Furuya
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  8. Michiaki Koike
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  9. Masaaki Noguchi
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  10. Kensuke Usuki
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  11. Motoaki Shiratsuchi
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  12. Kei Nakajima
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  13. Eiichi Ohtsuka
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  14. Hiroaki Tanaka
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  15. Eri Kawata
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  16. Mika Nakamae
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  17. Yasunori Ueda
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  18. Yasuo Aota
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  19. Yasumasa Sugita
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  20. Shin Ohara
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  21. Satoshi Yamasaki
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  22. Kohsuke Asagoe
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  23. Shuro Yoshida
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  24. Jun Yamanouchi
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  25. Sayaka Suzuki
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  26. Toshinori Kondo
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  27. Yuji Kanisawa
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  28. Kohtaro Toyama
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  29. Hiromi Omura
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  30. Daisuke Mizuchi
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  31. Sumio Sakamaki
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  32. Miki Ando
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  33. Norio Komatsu
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Corresponding author

Correspondence to Yoko Edahiro.

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Conflict of interest

YE reports grants or contracts from PharmaEssentia Japan K.K., Meiji Seika Pharma and AbbVie G.K.; honoraria for lectures, presentations, speakers bureaus, manuscript writing or educational events from PharmaEssentia Japan K.K., Takeda Pharmaceutical Co., Ltd. and Novartis Pharma K.K.; and participated on a data safety monitoring board or advisory board of Novartis Pharma K.K. and PharmaEssentia Japan K.K. TO reports grants or contracts from PharmaEssentia Japan K.K. and Meiji Seika Pharma. YH reports honoraria for lectures, presentations, speakers bureaus, manuscript writing or educational events from Takeda Pharmaceutical Co., Ltd. and Novartis Pharma K.K. SJ reports supports for the present manuscript from Otsuka Pharmaceutical Co., Ltd., Chugai Pharmaceutical Co., Ltd., Kyowa Kirin Co., Ltd., Takeda Pharmaceutical Co., Ltd., Novartis Pharma K.K., Sumitomo Pharma Co., Ltd., and Bristol-Myers Squibb K.K.; grants or contracts from FUJIFILM Wako Pure Chemical Corporation, Fuso Pharmaceutical, Pfizer Japan Inc., PharmaEssentia Japan K.K, Shire International GmbH, Perseus Proteomics Inc., Meiji Seika Pharma. SS reports grants or contracts from PharmaEssentia Japan K.K. and Meiji Seika Pharma. KU reports grants or contracts from Amgen-Astellas Biopharma K.K., Otsuka Pharmaceutical Co., LTD., Apellis Pharmaceuticals, Inc., SynBio Pharmaceuticals Ltd., Takeda Pharmaceutical Co., Ltd., Nippon-Shinyaku Co. Ltd., Novartis Pharma K.K., AbbVie G.K., Janssen Pharmaceutical K.K., Bristol-Myers Squibb K.K., Ono Pharmaceutical Co., LTD., Chugai Pharmaceutical Co., Ltd., Daiichi Sankyo Co., Ltd, MSD K.K., Astellas Pharma Inc., Alexion Pharmaceuticals, Inc., Kyowa Kirin Co., Ltd., Gilead Sciences, Inc., Pfizer Japan Inc., Incyte Biosciences Japan G.K., SynBio Pharmaceuticals Ltd., Celgene K.K., Sumitomo Dainippon Pharma Co., Ltd., Mundipharma K.K., Yakult Honsha Co., and Eisai Co., Ltd.; consulting fees from Alexion Pharmaceuticals, Inc., SynBio Pharmaceuticals Ltd., Nippon-Shinyaku Co. Ltd., Otsuka Pharmaceutical Co., LTD., Chugai Pharmaceutical Co., Ltd., Sanofi K.K., Takeda Pharmaceutical Co., Ltd., Kyowa Kirin Co., Ltd., Astellas Pharma Inc., SOBI, and Alnylam Japan; and honoraria for lectures, presentations, speakers bureaus, manuscript writing or educational events from Novartis Pharma K.K., Astellas Pharma Inc., Alexion Pharmaceuticals, Inc., Eisai Co., Ltd., MSD, Otsuka Pharmaceutical Co., LTD., Ono Pharmaceutical Co., LTD., Kyowa Kirin Co., Ltd., Celgene K.K., Daiichi Sankyo Co., Ltd., Takeda Pharmaceutical Co., Ltd., Nippon-Shinyaku Co. Ltd., PharmaEssentia Japan K.K., Bristol-Myers Squibb K.K., Yakult Honsha Co., Sanofi K.K., Pfizer Japan Inc., AbbVie G.K., and Chugai Pharmaceutical Co., Ltd. MN reports honoraria for lectures from Takeda Pharmaceutical Co., Ltd., Nippon-Shinyaku Co. Ltd., Novartis Pharma K.K. and Bristol-Myers Squibb K.K. YU participated on a data safety monitoring board or advisory board of Sanofi K.K. and Otsuka Pharmaceutical Co., LTD. JY reports honoraria for lectures, presentations, speakers bureaus, manuscript writing or educational events from Takeda Pharmaceutical Co., Ltd. and Chugai Pharmaceutical Co., Ltd. TK reports consulting fees from Sanwa Kagaku Kenkyusyo Co., LTD.; and honoraria for lectures, presentations, speakers bureaus, manuscript writing or educational events from Novartis Pharma K.K., Takeda Pharmaceutical CO., LTD., Ono Pharmaceutical Co., LTD., Otsuka Pharmaceutical C0., LTD., Nippon-Shinyaku Co., LTD. and Bristol-Myers Squibb K.K. MA reports grants or contracts from Century Therapeutics and Daiichi Sankyo Co., Ltd; and honoraria for lectures, presentations, speakers bureaus, manuscript writing or educational events from AbbVie G.K., Sumitomo Pharma Co., Ltd., Novartis Pharma K.K. and Sanofi K.K. NK reports grants or contracts from Otsuka Pharmaceutical Co., Ltd., Kyowa Kirin Co., Ltd., Sumitomo Pharma Co., Ltd., PharmaEssentia Japan K.K., Chugai Pharmaceutical Co., Ltd., Takeda Pharmaceutical Co., Ltd., Perseus Proteomics Inc. and Meiji Seika Pharma Co., Ltd.; consulting fees from Japan Tobacco Inc. and PharmaEssentia Japan K.K.; honoraria for lectures, presentations, speakers bureaus, manuscript writing or educational events from Novartis Pharma K.K. and Takeda Pharmaceutical Co., Ltd.; and is a board member of PharmaEssentia Japan.

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Edahiro, Y., Ochiai, T., Hashimoto, Y. et al. Clinical characteristics of Japanese patients with myelodysplastic/myeloproliferative neoplasm with ring sideroblasts and thrombocytosis. Int J Hematol 118, 47–53 (2023). https://doi.org/10.1007/s12185-023-03592-0

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  • DOI: https://doi.org/10.1007/s12185-023-03592-0

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