Eculizumab in atypical hemolytic uremic syndrome: long-term clinical course and histological findings

Tschumi, Sibylle; Gugger, Mathias; Bucher, Barbara S; Riedl, Magdalena; Simonetti, Giacomo D (2011). Eculizumab in atypical hemolytic uremic syndrome: long-term clinical course and histological findings. Pediatric nephrology, 26(11), pp. 2085-8. Berlin: Springer-Verlag 10.1007/s00467-011-1989-4

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Atypical hemolytic uremic syndrome (aHUS) is a thrombotic microangiopathy associated with defective regulation of the alternative complement pathway. The prognosis for patients with aHUS is poor, and plasma exchange represents the first-line therapy. Eculizumab is a humanized monoclonal anti-C5 antibody that prevents the activation of the terminal complement pathway. Here, we report the case of a 9-year-old girl with frequent relapsing aHUS due to heterozygous factor H mutation who was initially treated with plasma exchange three times per week with 150% plasma exchange volume. This treatment frequently caused allergic reactions and school absences. Because any reduction in the frequency of plasma exchange immediately induced relapses of the aHUS, treatment with eculizumab, 600 mg every 2 weeks, was started and plasma exchange completely stopped. On this drug regimen the patient showed no evidence of disease activity during a period of more than 24 months. Renal function improved, proteinuria disappeared, the number of antihypertensive medications could be decreased, and the quality of life increased substantially. The inhibition of the terminal complement pathway by eculizumab was also confirmed by renal biopsy, which showed the absence of thrombotic microangiopathy 2 months after the initiation of eculizumab therapy. This case illustrates the long-term favorable outcome of aHUS with eculizumab treatment.

Item Type:

Journal Article (Original Article)

Division/Institute:

04 Faculty of Medicine > Department of Gynaecology, Paediatrics and Endocrinology (DFKE) > Clinic of Paediatric Medicine
04 Faculty of Medicine > Service Sector > Institute of Pathology

UniBE Contributor:

Tschumi, Sibylle; Gugger, Mathias; Bucher, Barbara and Simonetti, Giacomo

ISSN:

0931-041X

Publisher:

Springer-Verlag

Language:

English

Submitter:

Factscience Import

Date Deposited:

04 Oct 2013 14:18

Last Modified:

26 Jun 2018 14:30

Publisher DOI:

10.1007/s00467-011-1989-4

PubMed ID:

21877169

Web of Science ID:

000295131900021

BORIS DOI:

10.7892/boris.5483

URI:

https://boris.unibe.ch/id/eprint/5483 (FactScience: 210230)

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