International Journal of Hematology

, Volume 98, Issue 4, pp 406–416

Long-term efficacy and safety of eculizumab in Japanese patients with PNH: AEGIS trial


    • Department of Hematology and OncologyOsaka University Hospital
  • Kazuma Ohyashiki
    • Tokyo Medical University
  • Tsutomu Shichishima
    • Department of Cardiology and Hematology, School of MedicineFukushima Medical University
  • Shinichiro Okamoto
    • Division of Hematology, Department of Internal MedicineKeio University Hospital
  • Kiyoshi Ando
    • Department of Hematology and OncologyTokai University School of Medicine
  • Haruhiko Ninomiya
    • Institute of Clinical MedicineUniversity of Tsukuba
  • Tatsuya Kawaguchi
    • Second Department of Internal MedicineKumamoto University School of Medicine
  • Shinji Nakao
    • Department of Hematology and OncologyKanazawa University Hospital
  • Hideki Nakakuma
    • Department of Hematology/OncologyWakayama Medical University
  • Jun-ichi Nishimura
    • Department of Hematology and Oncology, Graduate School of MedicineOsaka University
  • Taroh Kinoshita
    • Department of Immunoregulation, Research Institute for Microbial DiseasesOsaka University
  • Camille L. Bedrosian
    • Alexion Pharmaceuticals
  • Keiya Ozawa
    • Division of HematologyJichi Medical University
  • Mitsuhiro Omine
    • Division of HematologyShowa University Fujigaoka Hospital
Original Article

DOI: 10.1007/s12185-013-1404-y

Cite this article as:
Kanakura, Y., Ohyashiki, K., Shichishima, T. et al. Int J Hematol (2013) 98: 406. doi:10.1007/s12185-013-1404-y


Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, progressive hematopoietic stem cell disorder characterized by chronic complement-mediated hemolysis leading to life-threatening complications and early mortality. Eculizumab, a humanized anti-C5 monoclonal antibody, inhibits terminal complement activation, reduces hemolysis, decreases the risk of thrombosis, and improves renal function and quality of life in PNH patients. The long-term efficacy and safety of eculizumab in Japanese patients were assessed in a 2-year extension to a 12-week, open-label study (AEGIS). Eculizumab treatment led to an immediate and sustained reduction in intravascular hemolysis (P < 0.001) and red blood cell transfusions (P = 0.0016) compared with baseline levels. There were no reports of thromboembolism during eculizumab treatment. The majority of patients had stable (56 %) or improved (41 %) renal function and an improved quality of life (P = 0.015), with sustained reductions in fatigue and dyspnea. Eculizumab was well tolerated; no deaths or serious hemolytic events were reported, and the rate of infections declined over time. There were no significant differences in the response to eculizumab in patients with or without bone marrow dysfunction. These results demonstrate that eculizumab is an effective, well-tolerated long-term treatment for Japanese PNH patients and leads to continued amelioration of some hemolytic complications.


Paroxysmal nocturnal hemoglobinuriaComplement-inactivating agentsHemolysisEculizumabHematopoietic stem cell

Copyright information

© The Japanese Society of Hematology 2013