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

被引：21

作者：

Ueda, Yasutaka ^{[1
,2
]}

Obara, Naoshi ^{[2
,3
]}

Yonemura, Yuji ^{[2
,4
]}

Noji, Hideyoshi ^{[2
,5
]}

Masuko, Masayoshi ^{[6
]}

Seki, Yoshinobu ^{[7
,8
]}

Wada, Katsuya ^{[9
]}

Matsuda, Takahisa ^{[10
]}

Akiyama, Hirozumi ^{[10
]}

Ikezoe, Takayuki ^{[2
,5
]}

Chiba, Shigeru ^{[2
,11
]}

Kanda, Yoshinobu ^{[2
,12
]}

Kawaguchi, Tatsuya ^{[2
,13
,14
]}

Shichishima, Tsutomu ^{[2
,5
]}

Nakakuma, Hideki ^{[2
,15
]}

Okamoto, Shinichiro ^{[2
,16
]}

Nishimura, Jun-ichi ^{[1
,2
]}

Kanakura, Yuzuru ^{[1
,2
]}

Ninomiya, Haruhiko ^{[2
,17
]}

机构：

[1] Osaka Univ, Grad Sch Med, Dept Hematol & Oncol, Suita, Osaka, Japan

[2] Japan PNH Study Grp, Tokyo, Japan

[3] Univ Tsukuba, Dept Hematol, Tsukuba, Ibaraki, Japan

[4] Kumamoto Univ Hosp, Dept Transfus Med & Cell Therapy, Kumamoto, Japan

[5] Fukushima Med Univ, Dept Hematol, Fukushima, Japan

[6] Niigata Univ Hosp, Dept Stem Cell Transplantat, Niigata, Japan

[7] Niigata Prefectural Shibata Hosp, Dept Hematol, Shibata, Japan

[8] Niigata Univ Med & Dent Hosp, Uonuma Inst Community Med, Dept Hematol, Minami Uonuma, Japan

[9] Matsushita Mem Hosp, Dept Hematol, Moriguchi, Osaka, Japan

[10] Alex Pharma GK, Tokyo, Japan

[11] Univ Tsukuba, Dept Hematol, Fac Med, Tsukuba, Ibaraki, Japan

[12] Jichi Med Univ, Div Hematol, Shimotsuke, Japan

[13] Kumamoto Univ, Dept Hematol, Kumamoto, Japan

[14] Kumamoto Univ, Dept Infect Dis, Kumamoto, Japan

[15] Kagoshima Tokusyukai Hosp, Kagoshima, Japan

[16] Keio Univ, Sch Med, Dept Med, Div Hematol, Tokyo, Japan

[17] Univ Tsukuba, Dept Med Sci, Fac Med, Tsukuba, Ibaraki, Japan

来源：

INTERNATIONAL JOURNAL OF HEMATOLOGY | 2018年 / 107卷 / 06期

关键词：

PNH; QOL; Patient-reported outcome; Complement inhibitor; Eculizumab; COMPLEMENT INHIBITOR ECULIZUMAB; DISEASE; VALIDATION; EFFICACY; REGISTRY; CANCER; SAFETY; ANEMIA; TRIAL;

D O I：

10.1007/s12185-018-2409-3

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

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.

引用

页码：656 / 665

页数：10

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