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. 2025 Jun 14.
doi: 10.1007/s10157-025-02689-6. Online ahead of print.

Effectiveness and safety of ravulizumab for Japanese patients with atypical hemolytic uremic syndrome switched from eculizumab: an analysis of a post-marketing surveillance

Shuichi Ito  1 , Hiroshi Hataya  2 , Masanori Matsumoto  3 , Akihiko Shimono  4 , Hirofumi Teranishi  4 , Masaki Okuda  4 , Yoshitaka Miyakawa  5 , Shoichi Maruyama  6
Affiliations

Effectiveness and safety of ravulizumab for Japanese patients with atypical hemolytic uremic syndrome switched from eculizumab: an analysis of a post-marketing surveillance

Shuichi Ito et al. Clin Exp Nephrol. .

Abstract

Background: Ravulizumab, a long-acting anti-C5 antibody, was approved for atypical hemolytic uremic syndrome (aHUS) in September 2020 in Japan. Post-marketing surveillance was mandated by local regulatory authorities to evaluate the effectiveness and safety of ravulizumab in patients with aHUS in real-world clinical practice.

Methods: Patients with aHUS who switched from eculizumab to ravulizumab and received at least one dose of ravulizumab between September 2020 and December 2021 were enrolled. The effectiveness was evaluated by thrombotic microangiopathy (TMA) event-free status, defined as no sign of TMA recurrence and no initiation of plasma therapy/dialysis during ravulizumab treatment. The safety of ravulizumab was evaluated by summarizing the incidence of adverse events (AEs) and serious AEs.

Results: This study included 33 patients (19 children and 14 adults). The median (range) duration of eculizumab treatment before the switch was 1233 (113-3240) days, and the duration of ravulizumab treatment was 351 (127-365) days. During ravulizumab treatment, TMA event-free status was achieved in 97.0% (32/33) of patients. The platelet count, lactate dehydrogenase levels, and serum creatinine levels remained stable during ravulizumab treatment. Twenty-nine AEs were reported in 13 patients, including nine serious AEs in seven patients. No meningococcal infections or deaths occurred during ravulizumab treatment. One patient discontinued treatment and died 478 days later from an unknown cause.

Conclusions: This study confirmed the effectiveness and safety of ravulizumab in Japanese patients with aHUS after switching from eculizumab in a real-world setting.

Keywords: Atypical hemolytic uremic syndrome; Japan; Post-marketing surveillance; Ravulizumab; Safety; Thrombotic microangiopathy.

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Conflict of interest statement

Declarations. Conflict of interest: Akihiko Shimono and Masaki Okuda are employees of Alexion Pharma GK, the study sponsor. Hirofumi Teranishi was an employee of Alexion Pharma GK at the time of the study. Shuichi Ito, Hiroshi Hataya, Masanori Matsumoto, Yoshitaka Miyakawa, and Shoichi Maruyama report payment for lectures and advisory board participation from Alexion Pharma GK. Ethical approval: Ethical approval by an institutional review board is not mandatory for PMS. Informed consent from individual patients/caregivers was not required for inclusion in this observational study mandated by the Japanese authorities. However, patients who provided consent for the use of data for publication were included in this analysis. Research involving human participants: PMS was mandated by the Japanese government as part of the regulatory approval of ravulizumab for aHUS in Japan. PMS was conducted in accordance with Good Post-Marketing Study Practice (Ministry of Health, Labour and Welfare, Ministerial Ordinance No. 171 of 2004) to evaluate the safety and effectiveness of ravulizumab in clinical practice. Informed consent: Because this PMS was mandated as a condition of ravulizumab approval and was conducted in accordance with the Japanese Good Post-Marketing Study Practice requirements (Ministry of Health, Labour and Welfare, Ministerial Ordinance No. 171 of 2004), informed consent from individual patients/caregivers was not required. However, informed consent was required for the use of data for publication.

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