Effects of eculizumab treatment on quality of life in patients with paroxysmal nocturnal hemoglobinuria in Japan
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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.
KeywordsPNH QOL Patient-reported outcome Complement inhibitor Eculizumab
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.
Logistical and financial cost for the post-marketing surveillance of eculizumab including statistical analysis was provided by Alexion Pharma GK.
Compliance with ethical standards
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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