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Effects of eculizumab treatment on quality of life in patients with paroxysmal nocturnal hemoglobinuria in Japan

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A Correction to this article was published on 06 July 2018

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Abstract

In paroxysmal nocturnal hemoglobinuria (PNH), various symptoms due to intravascular hemolysis exert a negative impact on patients’ quality of life (QOL). To determine clinical factors related with improvements in QOL in PNH patients treated, we analyzed changes in QOL scales in PNH patients treated with eculizumab based on data collected from post-marketing surveillance in Japan. Summary statistics were obtained using figures from QOL scoring systems and laboratory values, and evaluated by t test. One-year administration of eculizumab improved the most QOL items in comparison with the baseline. In particular, significant improvement of EORTC QLQ-C30 was observed in fatigue, dyspnea, physical function, and global health status. Canonical correlation analysis revealed a high correlation between QOL and laboratory values. Changes in serum lactate dehydrogenase (LDH) and hemoglobin showed strong correlations with QOL improvement. Quality of life improvement was independent of patients’ baseline characteristics of co-occurrence of bone marrow failure (BMF), or the degree of LDH. In this analysis, we found that the degree of QOL improvement was independent of the baseline LDH before eculizumab treatment and of co-occurrence of BMF. Paroxysmal nocturnal hemoglobinuria patients who have not received eculizumab treatment due to mild hemolysis may benefit from eculizumab treatment.

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Change history

  • 06 July 2018

    In the original publication of this article, Tables 2, 3 and 4 were published incorrectly. The corrected tables 2, 3 and 4 are given in the following pages.

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Acknowledgements

We thank all participating physicians and registered patients who took part in this surveillance study. We are grateful to Dr. Yasuo Ohashi of Dep. Integrated Science and Engineering for Sustainable Society, Chuo University for special advice on research design including statistical analysis. This study was sponsored by Alexion, and Alexion was involved in the process of the study design, data collection, analysis and manuscript preparation. Alexion approved the content of the manuscript and the submission of the manuscript for publication.

Funding

Logistical and financial cost for the post-marketing surveillance of eculizumab including statistical analysis was provided by Alexion Pharma GK.

Author information

Authors and Affiliations

  1. Department of Hematology and Oncology, Osaka University Graduate School of Medicine, Suita, Osaka, Japan

    Yasutaka Ueda, Jun-ichi Nishimura & Yuzuru Kanakura

  2. Japan PNH Study Group, Tokyo, Japan

    Yasutaka Ueda, Naoshi Obara, Yuji Yonemura, Hideyoshi Noji, Takayuki Ikezoe, Shigeru Chiba, Yoshinobu Kanda, Tatsuya Kawaguchi, Tsutomu Shichishima, Hideki Nakakuma, Shinichiro Okamoto, Jun-ichi Nishimura, Yuzuru Kanakura & Haruhiko Ninomiya

  3. Department of Hematology, University of Tsukuba, Tsukuba, Japan

    Naoshi Obara

  4. Department of Transfusion Medicine and Cell Therapy, Kumamoto University Hospital, Kumamoto, Japan

    Yuji Yonemura

  5. Department of Hematology, Fukushima Medical University, Fukushima, Japan

    Hideyoshi Noji, Takayuki Ikezoe & Tsutomu Shichishima

  6. Department of Stem Cell Transplantation, Niigata University Hospital, Niigata, Japan

    Masayoshi Masuko

  7. Department of Hematology, Niigata Prefectural Shibata Hospital, Shibata, Japan

    Yoshinobu Seki

  8. Department of Hematology, Uonuma Institute of Community Medicine, Niigata University Medical and Dental Hospital, Minami-Uonuma, Japan

    Yoshinobu Seki

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

    Katsuya Wada

  10. Alexion Pharma G.K, Tokyo, Japan

    Takahisa Matsuda & Hirozumi Akiyama

  11. Department of Hematology, Faculty of Medicine, University of Tsukuba, Tsukuba, Japan

    Shigeru Chiba

  12. Division of Hematology, Jichi Medical University, Shimotsuke, Japan

    Yoshinobu Kanda

  13. Departments of Hematology and Infectious Diseases, Kumamoto University, Kumamoto, Japan

    Tatsuya Kawaguchi

  14. Kagoshima Tokusyukai Hospital, Kagoshima, Japan

    Hideki Nakakuma

  15. Division of Hematology, Department of Medicine, Keio University School of Medicine, Tokyo, Japan

    Shinichiro Okamoto

  16. Department of Medical Sciences, Faculty of Medicine, University of Tsukuba, Tsukuba, Japan

    Haruhiko Ninomiya

Authors
  1. Yasutaka Ueda
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  2. Naoshi Obara
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  4. Hideyoshi Noji
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  5. Masayoshi Masuko
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  6. Yoshinobu Seki
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  7. Katsuya Wada
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  8. Takahisa Matsuda
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  9. Hirozumi Akiyama
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  10. Takayuki Ikezoe
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  11. Shigeru Chiba
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  12. Yoshinobu Kanda
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  13. Tatsuya Kawaguchi
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  14. Tsutomu Shichishima
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  15. Hideki Nakakuma
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  16. Shinichiro Okamoto
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  17. Jun-ichi Nishimura
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  18. Yuzuru Kanakura
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  19. Haruhiko Ninomiya
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Corresponding author

Correspondence to Haruhiko Ninomiya.

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Conflict of interest

Dr. Kawaguchi reported personal fees from Alexion Pharma G.K., outside the submitted work; Dr. Ueda reported personal fees from Kyowa Hakko Kirin Co.Ltd. and from Celgene, outside the submitted work; Dr. Shichishima and Dr. Yonemura, report grants from Alexion Pharma G.K., outside the submitted work; Dr. Ueda, Dr. Nishimmura, Dr. Ninomiya report grant and personal fees from Alexion Pharma G.K., during the conduct of the study; Dr. Chiba report grand from Alexion Pharma G.K., during the conduct of the study. Dr. Okamoto reports grants from Alexion Pharma G.K. and from Chugai Pharmaceutical Co. Ltd., outside the submitted work. Dr. Kanakura reports grants from Alexion Pharma G.K. and from Chugai Pharmaceutical Co. Ltd. Dr. Obara, Dr. Seki, Dr. Kanda, Dr. Nakakuma, Dr. Ikezoe, and Dr. Masuko, Dr. Noji and Dr. Wada have nothing to declare; Dr. Matsuda and Mr. Akiyama report other from Alexion Pharma G.K., outside the submitted work; Employee of Alexion Pharma G.K. and Stock/stock options in the company.

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Ueda, Y., Obara, N., Yonemura, Y. et al. Effects of eculizumab treatment on quality of life in patients with paroxysmal nocturnal hemoglobinuria in Japan. Int J Hematol 107, 656–665 (2018). https://doi.org/10.1007/s12185-018-2409-3

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  • DOI: https://doi.org/10.1007/s12185-018-2409-3

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