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

• Genetic Diseases of the Kidneys: Non-Cystic - I
  October 25, 2018 | Location: Exhibit Hall, San Diego Convention Center
  Abstract Time: 10:00 AM - 12:00 PM

Category: Genetic Diseases of the Kidney

• 1002 Genetic Diseases of the Kidney: Non-Cystic

Authors

• Okada, Hirokazu, Saitama Medical University, Saitama, Japan

Hirokazu Okada,

• Kato, Hideki, The University of Tokyo Graduate School of Medicine, Bunkyo-ku, TOKYO, Japan

Hideki Kato,

• Miyakawa, Yoshitaka, Saitama Medical University, Saitama, Japan

Yoshitaka Miyakawa,

• Hidaka, Yoshihiko, Shinshu University School of Medicine, Matsumoto, Nagano, Japan

Yoshihiko Hidaka,

• Inoue, Norimitsu, Osaka International Cancer Institute, Osaka, Japan

Norimitsu Inoue,

• Ito, Shuichi, Department of Pediatrics, Graduate School of Medicine, Yokohama City University, Yokohama, Japan

Shuichi Ito,

• Kagami, Shoji, Tokushima Unversity, Tokushima, Japan

Shoji Kagami,

• Kaname, Shinya, Kyorin University School of Medicine, Mitaka, Tokyo, Japan

Shinya Kaname,

• Matsumoto, Masanori, Nara Medical University, Kashihara city, Japan

Masanori Matsumoto,

• Mizuno, Masashi, Nagoya University, Nagoya, Japan

Masashi Mizuno,

• Matsuda, Takahisa, Alexion Pharma GK, Tokyo, Japan

Takahisa Matsuda,

• Shimono, Akihiko, Alexion Pharma GK, Tokyo, Japan

Akihiko Shimono,

• Maruyama, Shoichi, Nagoya University Graduate School of Medicine, Nagoya, Japan

Shoichi Maruyama,

• Fujimura, Yoshihiro, Japanese Red Cross Kinki Block Blood center, Ibaraki, Japan

Yoshihiro Fujimura,

• Nangaku, Masaomi, the University of Tokyo School of Medicine, Tokyo, Japan

Masaomi Nangaku,

Background

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.

Methods

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.

Results

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.

Conclusion

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

Funding

• Commercial Support – Alexion Pharma GK