Traitement par eculizumab dans le syndrome hémolytique et urémique typique de l’enfant : étude rétrospective contrôlée

Abstract : Background: treatment by eculizumab, a monoclonal anti-C5 antibody in shigatoxin-associated hemolytic and uremic syndrome (STEC-HUS) remains controversial despite of an increasing utilization in STEC-HUS with severe organ involvement since 2011. Methods: data were retrospectively collected from medical records of children diagnosed with STEC-HUS treated in Bordeaux University Children's Hospital between 1995 and 2017. The outcomes of patients treated with eculizumab for a STEC-HUS were compared to those of a control group of untreated patients matched for age, sex, and severity of acute kidney injury with a 1:2 matching scheme. Results: a total of 18 children (median age 40.6 months, 39% boys) with STEC-HUS were treated by eculizumab and compared with 36 matched control patients (median 36.4 months, 33% boys) who had not received eculizumab. All patients survived in the two groups. During one month after HUS onset, the evolution of hematological parameters did not differ between the two groups. Median duration of renal replacement therapy was 6 days (IQR 4-12) in eculizumab group and 7 days (IQR 4-15) in controls. At 1 and 12 months of follow-up, there was no statistically significant difference between the two groups regarding the proportion of patients with high blood pressure, abnormal proteinuria or decreased glomerular filtration rate (GFR). After a median follow-up of 2.5 years in patients treated by eculizumab and 5.0 years in controls, the prevalence of decreased GFR tended to be lower in eculizumab group (5/18, 27%) than in controls (14/36, 38%), and the prevalence of proteinuria tended to be lower in eculizumab group (3/18, 17%) than in controls (10/36, 27%). These differences were not statistically significant. Five children (27%) who received eculizumab and two from the control group (5%) had extra-renal sequaelae during follow-up. Conclusion: this single-center study did not show any signal for harm of eculizumab as a treatment of STEC-HUS in children. No differences were seen in the kinetics of biological markers over one year between the two groups. The benefit of eculizumab on renal and extra-renal outcomes in STEC-HUS could not be established based on our results. Larger prospective randomized trials are required to assess the efficacy of eculizumab in this indication.
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Master Thesis
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Audrey Chevallier. Traitement par eculizumab dans le syndrome hémolytique et urémique typique de l’enfant : étude rétrospective contrôlée. Médecine humaine et pathologie. 2018. ⟨dumas-02066675⟩

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