International Journal of Hematology

DOI: 10.1007/s12185-018-2409-3 Pages: 656-665

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

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

2. Japan PNH Study Group

3. University of Tsukuba, Department of Hematology

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

5. Fukushima Medical University, Department of Hematology

6. Niigata University Hospital, Department of Stem Cell Transplantation

7. Niigata Prefectural Shibata Hospital, Department of Hematology

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

9. Matsushita Memorial Hospital, Department of Hematology

10. Alexion Pharma G.K

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

12. Jichi Medical University, Division of Hematology

13. Kumamoto University, Departments of Hematology and Infectious Diseases

14. Kagoshima Tokusyukai Hospital

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

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

Correspondence to:
Haruhiko Ninomiya



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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