Efficacy and safety of eculizumab in childhood atypical hemolytic uremic syndrome in Japan

Ito, Naoko; Hataya, Hiroshi; Saida, Ken; Amano, Yoshihiko; Hidaka, Yoshihiko; Motoyoshi, Yaeko; Ohta, Toshiyuki; Yoshida, Yasuhiro; Terano, Chikako; Iwasa, Tadashi; Wataru, Kubota; Takada, Hidetoshi; Hara, Toshiro; Fujimura, Yoshihiro; Ito, Shuichi

doi:10.1007/s10157-015-1142-y

Cited by 24 publications

(20 citation statements)

References 34 publications

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“…Few retrospective and prospective clinical studies have evaluated the safety and efficacy of eculizumab in Japanese patients with aHUS [5,6,[14][15][16][17]. The outcomes in this aHUS cohort [11].…”

Section: Discussionmentioning

confidence: 99%

Safety and effectiveness of eculizumab for pediatric patients with atypical hemolytic–uremic syndrome in Japan: interim analysis of post-marketing surveillance

et al. 2018

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Background In 2013, eculizumab was approved for treatment of the atypical hemolytic-uremic syndrome (aHUS) in Japan, which was defined as a thrombotic microangiopathy (TMA) excluding Shiga toxin-producing Escherichia coli-HUS and thrombotic thrombocytopenic purpura. Simultaneously, post-marketing surveillance was started to assess its safety and effectiveness. In 2016, Japanese clinical guide redefined terms to limit the use of "aHUS" to complement-mediated HUS only. Accordingly, TMA with other causes was defined as secondary TMA. Here we report the interim analysis of post-marketing surveillance of pediatric patients with aHUS and secondary TMA. Methods Pediatric patients treated with eculizumab from approval to 15 March 2017 were included in this observational real-world study. Clinical endpoints of effectiveness were TMA event-free status, complete TMA response, platelet count normalization, and improvement of estimated glomerular filtration rate (eGFR). Adverse reactions to eculizumab were also analyzed. Results In 27 pediatric patients with aHUS, median age at diagnosis was 4 years. Complement genes' variants were detected in 14 of 21 patients (66.7%). Median time from diagnosis to eculizumab initiation was 2.0 days. TMA event-free status, complete TMA response, platelet normalization, and improvement in eGFR were achieved in 85.2, 36.4, 78.3, and 75.0% of patients, respectively. Three patients with aHUS died. Twenty-four and 10 adverse reactions were reported in 31 aHUS patients and 17 secondary TMA patients, respectively; however, no eculizumab-related death or meningococcal infection was reported. Conclusions This interim analysis confirmed that eculizumab is well-tolerated and effective for Japanese pediatric patients with aHUS in a real-world setting.

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Section: Discussionmentioning

confidence: 99%

Safety and effectiveness of eculizumab for pediatric patients with atypical hemolytic–uremic syndrome in Japan: interim analysis of post-marketing surveillance

et al. 2018

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“…However, a recent case published by Fukui et al [19] describes a case with TMA and EGPA and TMA history has been reported in pediatric patients with aHUS [20]. Therefore, considering all the facts, it might be possible that a relationship between EGPA and aHUS exists.…”

Section: Discussionmentioning

confidence: 89%

Two cases of atypical hemolytic uremic syndrome (aHUS) and eosinophilic granulomatosis with polyangiitis (EGPA): a possible relationship

et al. 2017

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“…The authors wish to thank Mr. James Robert Valera of Redact Editing and Translation Services for his help in editing this manuscript. The information for Patients 1–3 was submitted to the Japanese National Survey of Eculizumab for aHUS . The pathological findings in Case 2 have also been reported elsewhere …”

Section: Acknowledgmentsmentioning

confidence: 99%

Practical issues in using eculizumab for children with atypical haemolytic uraemic syndrome in the acute phase: A review of four patients

et al. 2018

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Efficacy and safety of eculizumab in childhood atypical hemolytic uremic syndrome in Japan

Cited by 24 publications

References 34 publications

Safety and effectiveness of eculizumab for pediatric patients with atypical hemolytic–uremic syndrome in Japan: interim analysis of post-marketing surveillance

Safety and effectiveness of eculizumab for pediatric patients with atypical hemolytic–uremic syndrome in Japan: interim analysis of post-marketing surveillance

Two cases of atypical hemolytic uremic syndrome (aHUS) and eosinophilic granulomatosis with polyangiitis (EGPA): a possible relationship

Practical issues in using eculizumab for children with atypical haemolytic uraemic syndrome in the acute phase: A review of four patients

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