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Eculizumab treatment and discontinuation in pediatric patients with atypical hemolytic uremic syndrome: a multicentric retrospective study

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A Correction to this article was published on 07 February 2022

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Abstract

Introduction

Eculizumab is effective treatment of pediatric atypical hemolytic uremic syndrome (aHUS). However, the optimal duration of treatment is not clearly defined. The aim of this study was to retrospectively analyze the outcome of pediatric patients with aHUS, who started eculizumab treatment but discontinued it during the follow-up period.

Methods

The clinical and laboratory findings of the pediatric patients with aHUS were recorded on a web-based, national registry system, known as the Turkish aHUS Registry. The study included 63 patients who had to have received more than four doses of eculizumab during the acute phase of the disease.

Results

The median age at diagnosis was 3.62 (IQR: 1.29–6.14) years. During the follow-up period, 39 patients continued to receive standard eculizumab treatment (standard treatment group, treatment every 2 weeks) while 24 received an extended dose of eculizumab at three-four-week intervals (non-standard treatment group). There was no significant difference between both groups in terms of clinical and laboratory parameters. Eculizumab treatment was discontinued in 18 patients (30.7%, F/M:11/7), and the median age of these patients at diagnosis and their median follow-up duration were 4.0 (IQR:2.7–10.2) and 4.2 (IQR:2.2–7) years respectively. The median eGFR at the last visit was 110 (84.7–146.1)ml/min/1.73 m2. Fourteen patients remained in remission without any sign of the disease. Recurrence occurred in four (22.2%) patients, in which eculizumab was immediately started again and complete remission was achieved.

Conclusion

Eculizumab is a successful treatment option in pediatric patients with aHUS and it can be safely discontinued with close monitoring in a selected group of patients. In case of recurrence, eculizumab should be restarted immediately to achieve complete remission.

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Funding

This manuscript did not receive any specific grant from funding agencies in the public, private, commercial, or not-for profit sectors.

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Authors and Affiliations

  1. Department of Pediatric Nephrology, Başkent University, Ankara, Turkey

    Esra Baskin & Kaan Gulleroglu

  2. Department of Pediatric Nephrology, School of Medicine, Gazi University, Ankara, Turkey

    Kibriya Fidan & Oguz Soylemezoglu

  3. Department of Pediatric Nephrology, Hacettepe University Faculty of Medicine, Ankara, Turkey

    Bora Gulhan & Fatih Ozaltin

  4. Department of Pediatric Nephrology, Istanbul University-Cerrahpasa, İstanbul, Turkey

    Nur Canpolat

  5. Department of Pediatric Nephrology, Istanbul University Istanbul School of Medicine, İstanbul, Turkey

    Alev Yilmaz

  6. Department of Pediatric Nephrology, Başkent University Adana Dr. Turgut Noyan Training and Research Center, Adana, Turkey

    Gonül Parmakiz

  7. Department of Pediatric Nephrology, Ankara University Medical School, Ankara, Turkey

    Zeynep Birsin Özçakar

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  1. Esra Baskin
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  2. Kibriya Fidan
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  10. Oguz Soylemezoglu
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Correspondence to Kibriya Fidan.

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The study was approved by the ethics committee of Hacettepe University (FON10/03-22). The authors confirmed their adherence to the Declaration of Helsinki.

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The original article has been updated: Due to co-author family name and surname update.

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Baskin, E., Fidan, K., Gulhan, B. et al. Eculizumab treatment and discontinuation in pediatric patients with atypical hemolytic uremic syndrome: a multicentric retrospective study. J Nephrol 35, 1213–1222 (2022). https://doi.org/10.1007/s40620-021-01212-w

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