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Kidney Week

Abstract: TH-PO714

Safety and Effectiveness of Eculizumab for Adult Patients with Atypical Hemolytic-Uremic Syndrome in Japan: Interim Analysis of Post-Marketing Surveillance

Session Information

Category: Genetic Diseases of the Kidney

  • 1002 Genetic Diseases of the Kidney: Non-Cystic


  • Okada, Hirokazu, Saitama Medical University, Saitama, Japan
  • Kato, Hideki, The University of Tokyo Graduate School of Medicine, Bunkyo-ku, TOKYO, Japan
  • Miyakawa, Yoshitaka, Saitama Medical University, Saitama, Japan
  • Hidaka, Yoshihiko, Shinshu University School of Medicine, Matsumoto, Nagano, Japan
  • Inoue, Norimitsu, Osaka International Cancer Institute, Osaka, Japan
  • Ito, Shuichi, Department of Pediatrics, Graduate School of Medicine, Yokohama City University, Yokohama, Japan
  • Kagami, Shoji, Tokushima Unversity, Tokushima, Japan
  • Kaname, Shinya, Kyorin University School of Medicine, Mitaka, Tokyo, Japan
  • Matsumoto, Masanori, Nara Medical University, Kashihara city, Japan
  • Mizuno, Masashi, Nagoya University, Nagoya, Japan
  • Matsuda, Takahisa, Alexion Pharma GK, Tokyo, Japan
  • Shimono, Akihiko, Alexion Pharma GK, Tokyo, Japan
  • Maruyama, Shoichi, Nagoya University Graduate School of Medicine, Nagoya, Japan
  • Fujimura, Yoshihiro, Japanese Red Cross Kinki Block Blood center, Ibaraki, Japan
  • Nangaku, Masaomi, the University of Tokyo School of Medicine, Tokyo, Japan

Eculizumab has been available for the treatment of atypical hemolytic–uremic syndrome (aHUS) in Japan since 2013. To assess safety and effectiveness of eculizumab in adult aHUS patients in the real-life setting, we performed interim analysis of a post-marketing surveillance mandated by Japanese regulations.


This study enrolled any patient diagnosed with TMA excluding Shiga toxin-producing Escherichia coli-HUS or thrombotic thrombocytopenic purpura based on Japanese clinical guideline published in 2013 as inclusion criteria and treated with eculizumab. According to the guideline revised in 2016, the enrolled patients were with aHUS (complement-mediated HUS) and secondary TMA.


Thirty-three patients with aHUS and 27 patients with secondary TMA were enrolled. Median treatment duration of aHUS was 24weeks. Complement genes variants were detected in 11 of 18 aHUS patients who underwent genetic analysis (61.1%). Among the 29 aHUS patients with available baseline data, platelet count (PLT), lactic dehydrogenase and serum creatinine (SCr) improved within 1-month after eculizumab initiation. TMA event–free status, complete TMA response, PLT normalization, and SCr decrease were achieved in 67.9% (19/28), 27.8% (5/18), 56.5% (13/23), and 57.1% (16/28) of patients, respectively. Thirty-three adverse reactions, i.e., hypertension, hyperuricemia, nasopharyngitis and edema, were observed in 13 of 33 aHUS patients.


This interim analysis confirmed the acceptable safety profile and effectiveness of eculizumab for Japanese adult aHUS patients in real-world settings.


  • Commercial Support – Alexion Pharma GK