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First-Line, Early and Long-Term Eculizumab Therapy in Atypical Hemolytic Uremic Syndrome: A Case Series in Pediatric Patients

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Abstract

Introduction

Studies relating to first-line, early, and long-term eculizumab treatment and outcomes in children with atypical hemolytic uremic syndrome (aHUS) are scarce and unclear. The aim of this case-series study was to evaluate the outcomes of first-line, early, and long-term eculizumab treatment in our aHUS patients.

Materials and Methods

We reviewed the data from four pediatric patients with aHUS who were treated with eculizumab. In three of them, eculizumab was used as a first-line therapy, and the follow-up period was ≥2 years in three patients.

Results

Plasma exchange could not be performed in any patient. Plasma infusions were used only in Patient 1 (a 14-month-old boy) for 8 days without any response. Therefore, eculizumab was started on day 11 after admission. Patient 2 (a 16-month-old boy), Patient 3 (an 11-year-old girl), and Patient 4 (a 32-month-old girl) were treated with eculizumab as a first-line therapy, which was started 2–4 days after admission. The dosage of eculizumab was adjusted according to body weight. The hematologic parameters (the time frames were 3–17 days) and C 3 (the time frames were 10–17 days) returned to normal in all patients after receipt of eculizumab. Although Patient 1 developed stage III chronic kidney disease, complete renal recovery occurred in Patients 2 and 4. Patient 3 also had reflux nephropathy with bilateral grade III vesicoureteral reflux and renal scars. Her creatinine clearance returned to the baseline value after receiving eculizumab. No complications related to eculizumab were observed in any patient during the follow-up period.

Conclusion

Eculizumab can be successfully used as a first-line therapy in pediatric aHUS patients. We observed that the early initiation of eculizumab was associated with the complete recovery of renal function.

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Acknowledgments

The authors would like to acknowledge Professor Dr Aynur Karadenizli (Kocaeli University Faculty of Medicine, Department of Medical Microbiology) for verotoxin testing.

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Authors and Affiliations

  1. Department of Pediatric Nephrology, Pamukkale University School of Medicine, Kınıklı, Denizli, Turkey

    Selçuk Yüksel, Havva Evrengül & Tülay Becerir

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

    Z. Birsin Özçakar

  3. Department of Pathology, Pamukkale University School of Medicine, Denizli, Turkey

    Nagihan Yalçın

  4. Department of Pediatric Nephrology and Nephrogenetics Laboratory, Hacettepe University School of Medicine, Ankara, Turkey

    Emine Korkmaz & Fatih Ozaltin

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  1. Selçuk Yüksel
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Correspondence to Selçuk Yüksel.

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Conflict of interest

Dr. Selçuk Yüksel, Dr. Havva Evrengül, Dr. Z. Birsin Özçakar, Dr. Tülay Becerir, Dr. Nagihan Yalçın, Dr. Emine Korkmaz, and Dr. Fatih Özaltın have no financial disclosures or conflicts of interest that are directly relevant to the content of this article.

The data for this article was obtained from patient records.

Informed consent for genetic testing and for publication of their child’s clinical information was obtained from the parents.

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Yüksel, S., Evrengül, H., Özçakar, Z.B. et al. First-Line, Early and Long-Term Eculizumab Therapy in Atypical Hemolytic Uremic Syndrome: A Case Series in Pediatric Patients. Pediatr Drugs 18, 413–420 (2016). https://doi.org/10.1007/s40272-016-0194-0

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