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Comparison of outcomes after plasma therapy or eculizumab in pediatric patients with atypical hemolytic uremic syndrome

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Abstract

Background

Atypical hemolytic uremic syndrome (aHUS) is an ultra-rare and life-threatening disease. For decades, plasma therapy was used to manage patients with aHUS. Since eculizumab, a recombinant humanized anti-C5 monoclonal antibody, was approved for treatment of aHUS, it has been used to treat patients with aHUS. Here, we examined the effectiveness of eculizumab and plasma therapy, respectively in the treatment of pediatric patients with aHUS.

Methods

Data were collected from questionnaires sent to 75 institutions known to be treating thrombotic microangiopathy (TMA).

Results

A total of 24 patients were evaluable, in which no recurrence of TMA was reported at last observation. There were four therapy groups: two patients receiving supportive therapy, one receiving plasma therapy alone, 17 switching from plasma therapy to eculizumab (therapy switched), and four receiving eculizumab alone. Among 17 patients of therapy-switched group, only one patient achieved complete remission at the end of plasma therapy, 15 patients achieved complete remission after eculizumab initiation, and two patients reached end-stage renal disease. Adverse events were reported in nine cases; among these, meningococcal infection, anaphylaxis, and eculizumab-related infusion reaction were reported among those treated with eculizumab.

Conclusion

This study provided substantial evidence from a Japanese population that the conversion from plasma therapy to eculizumab therapy should be considered in patients with aHUS who show an incomplete response to plasma therapy. In addition, although no new safety events were detected, careful attention should be paid to meningococcal infection, eculizumab-related infusion reactions and allergic reactions with administration of eculizumab.

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Data availability

The data that support the findings of this study are available on reasonable request from the corresponding author. The data are not publicly available due to privacy or ethical restrictions.

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Acknowledgements

Alexion, AstraZeneca Rare Disease was involved in the study design and provided a formal review of the publication. All of authors retain control and final authority of publication content and decisions, including the choice of journal. The authors thank all patients and physicians who participated in this study.

Funding

This study was funded by Alexion GK.

Author information

Authors and Affiliations

  1. Department of Pediatrics, Osaka Medical and Pharmaceutical University, 2-7 Daigakumachi, Takatsuki, Osaka, 569-8686, Japan

    Akira Ashida, Hideki Matsumura, Yuko Fujii & Satoshi Yamazaki

  2. Alexion Pharma GK, 3-1-1 Shibaura, Minato-Ku, Tokyo, 108-0023, Japan

    Akihiko Shimono

Authors
  1. Akira Ashida
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  2. Hideki Matsumura
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  3. Akihiko Shimono
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  4. Yuko Fujii
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  5. Satoshi Yamazaki
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Contributions

AA and AS: designed the study. AA, HM, YF, and SY: collected and analyzed the clinical information and drafted the manuscript. All authors critically read and approved the final manuscript.

Corresponding author

Correspondence to Akira Ashida.

Ethics declarations

Conflict of interest

Employment: Akihiko Shimono (Alexion Pharma GK), Stock options: Akihiko Shimono (Alexion, AstraZeneca Rare Disease), Advisory role: Akira Ashida (Alexion Pharma GK), Honoraria: Akira Ashida (Alexion Pharma GK), Grants Received: Akira Ashida (Alexion Pharma GK,).

Ethical approval

The survey was conducted in accordance with the ethical principles contained within the 1964 Declaration of Helsinki, and the ethical guidelines for epidemiological studies issued by the Ministry of Health, Labour and Welfare, Japan. The survey was approved by the ethics board of Osaka Medical College (approved number: 2675–2) before the study commenced.

Informed consent

For this type of study, formal consent was not required.

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Ashida, A., Matsumura, H., Shimono, A. et al. Comparison of outcomes after plasma therapy or eculizumab in pediatric patients with atypical hemolytic uremic syndrome. Clin Exp Nephrol 27, 161–170 (2023). https://doi.org/10.1007/s10157-022-02293-y

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  • DOI: https://doi.org/10.1007/s10157-022-02293-y

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