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

Yasutaka Ueda, Naoshi Obara, Yuji Yonemura, Hideyoshi Noji, Masayoshi Masuko, Yoshinobu Seki, Katsuya Wada, Takahisa Matsuda, Hirozumi Akiyama, Takayuki Ikezoe, Shigeru Chiba, Yoshinobu Kanda, Tatsuya Kawaguchi, Tsutomu Shichishima, Hideki Nakakuma, Shinichiro Okamoto, Jun ichi Nishimura, Yuzuru Kanakura, Haruhiko Ninomiya

Research output: Contribution to journalArticle

1 Citation (Scopus)

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.

Original languageEnglish
Pages (from-to)1-10
Number of pages10
JournalInternational Journal of Hematology
DOIs
Publication statusAccepted/In press - 2018 Jan 30

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Paroxysmal Hemoglobinuria
Japan
Quality of Life
Quality Improvement
L-Lactate Dehydrogenase
Therapeutics
Hemolysis
Bone Marrow
eculizumab
Marketing
Dyspnea
Health Status
Fatigue
Hemoglobins

Keywords

  • Complement inhibitor
  • Eculizumab
  • Patient-reported outcome
  • PNH
  • QOL

ASJC Scopus subject areas

  • Hematology

Cite this

Effects of eculizumab treatment on quality of life in patients with paroxysmal nocturnal hemoglobinuria in Japan. / Ueda, Yasutaka; Obara, Naoshi; Yonemura, Yuji; Noji, Hideyoshi; Masuko, Masayoshi; Seki, Yoshinobu; Wada, Katsuya; Matsuda, Takahisa; Akiyama, Hirozumi; Ikezoe, Takayuki; Chiba, Shigeru; Kanda, Yoshinobu; Kawaguchi, Tatsuya; Shichishima, Tsutomu; Nakakuma, Hideki; Okamoto, Shinichiro; Nishimura, Jun ichi; Kanakura, Yuzuru; Ninomiya, Haruhiko.

In: International Journal of Hematology, 30.01.2018, p. 1-10.

Research output: Contribution to journalArticle

Ueda, Y, Obara, N, Yonemura, Y, Noji, H, Masuko, M, Seki, Y, Wada, K, Matsuda, T, Akiyama, H, Ikezoe, T, Chiba, S, Kanda, Y, Kawaguchi, T, Shichishima, T, Nakakuma, H, Okamoto, S, Nishimura, JI, Kanakura, Y & Ninomiya, H 2018, 'Effects of eculizumab treatment on quality of life in patients with paroxysmal nocturnal hemoglobinuria in Japan', International Journal of Hematology, pp. 1-10. https://doi.org/10.1007/s12185-018-2409-3
Ueda, Yasutaka ; Obara, Naoshi ; Yonemura, Yuji ; Noji, Hideyoshi ; Masuko, Masayoshi ; Seki, Yoshinobu ; Wada, Katsuya ; Matsuda, Takahisa ; Akiyama, Hirozumi ; Ikezoe, Takayuki ; Chiba, Shigeru ; Kanda, Yoshinobu ; Kawaguchi, Tatsuya ; Shichishima, Tsutomu ; Nakakuma, Hideki ; Okamoto, Shinichiro ; Nishimura, Jun ichi ; Kanakura, Yuzuru ; Ninomiya, Haruhiko. / Effects of eculizumab treatment on quality of life in patients with paroxysmal nocturnal hemoglobinuria in Japan. In: International Journal of Hematology. 2018 ; pp. 1-10.
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AU - Masuko, Masayoshi

AU - Seki, Yoshinobu

AU - Wada, Katsuya

AU - Matsuda, Takahisa

AU - Akiyama, Hirozumi

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AU - Chiba, Shigeru

AU - Kanda, Yoshinobu

AU - Kawaguchi, Tatsuya

AU - Shichishima, Tsutomu

AU - Nakakuma, Hideki

AU - Okamoto, Shinichiro

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AU - Ninomiya, Haruhiko

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