Document is current

Article History

Received: 28 January 2025

Accepted: 21 April 2025

First Online: 14 June 2025

Declarations

:

: 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 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.

: 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.

: 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.