TY - JOUR
T1 - Effects of eculizumab treatment on quality of life in patients with paroxysmal nocturnal hemoglobinuria in Japan
AU - Ueda, Yasutaka
AU - Obara, Naoshi
AU - Yonemura, Yuji
AU - Noji, Hideyoshi
AU - Masuko, Masayoshi
AU - Seki, Yoshinobu
AU - Wada, Katsuya
AU - Matsuda, Takahisa
AU - Akiyama, Hirozumi
AU - Ikezoe, Takayuki
AU - Chiba, Shigeru
AU - Kanda, Yoshinobu
AU - Kawaguchi, Tatsuya
AU - Shichishima, Tsutomu
AU - Nakakuma, Hideki
AU - Okamoto, Shinichiro
AU - Nishimura, Jun ichi
AU - Kanakura, Yuzuru
AU - Ninomiya, Haruhiko
N1 - Funding Information:
Conflict of interest Dr. Kawaguchi reported personal fees from Alex-ion 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. Nakaku-ma, 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.
Funding Information:
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. 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.
Publisher Copyright:
© 2018, The Japanese Society of Hematology.
PY - 2018/6/1
Y1 - 2018/6/1
N2 - 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.
AB - 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.
KW - Complement inhibitor
KW - Eculizumab
KW - PNH
KW - Patient-reported outcome
KW - QOL
UR - http://www.scopus.com/inward/record.url?scp=85041239583&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=85041239583&partnerID=8YFLogxK
U2 - 10.1007/s12185-018-2409-3
DO - 10.1007/s12185-018-2409-3
M3 - Article
C2 - 29383624
AN - SCOPUS:85041239583
SN - 0925-5710
VL - 107
SP - 656
EP - 665
JO - International journal of hematology
JF - International journal of hematology
IS - 6
ER -