ESPN 51th Annual Meeting

ESPN 2018


 
Eculizumab treatment in STEC-HUS: a retrospective matched-controlled study
DIDAILLER CATHERINE 1 CHEVALLIER AUDREY 1 DUBRASQUET ASTRID 1 DELMAS YAHSOU 2 BORDES C├ęCILE 3 LLANAS BRIGITTE 1 HARAMBAT JEROME 1

1- PEDIATRIC NEPHROLOGY UNIT, BORDEAUX UNIVERSITY HOSPITAL, FRANCE
2- NEPHROLOGY DEPARTMENT, BORDEAUX UNIVERSITY HOSPITAL, FRANCE
3- LABORATORY OF IMMUNOLOGY, BORDEAUX UNIVERSITY HOSPITAL, FRANCE
 
Introduction:

Treatment by eculizumab in shigatoxin-associated hemolytic uremic syndrome (STEC-HUS) remains controversial despite of an increasing utilization in STEC-HUS with severe organ involvement.

Material and methods:

Data were retrospectively collected from children diagnosed with STEC-HUS treated in a single center. The outcomes of patients wo received eculizumab for STEC-HUS were compared to a control group of untreated patients matched for age, sex, and severity of acute kidney injury.

Results:

A total of 18 children (median age 40 months, 39% boys) with STEC-HUS were treated by eculizumab and compared with 36 matched control patients (median age 36 months, 33% boys) who had not received eculizumab. All patients survived. Within 1 month of HUS onset, hematological parameters kinetics was similar between the two groups. Median duration of renal replacement therapy was 6 days in eculizumab group and 7 days 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, proteinuria or decreased GFR. After a median follow-up of 2.5 years in eculizumab treated patients and 5.0 years in controls, the prevalence of decreased GFR (27% in eculizumab group vs. 38% in controls), and of proteinuria (17% in eculizumab vs. 27% in controls) were not statistically significantly different. Five children (27%) who received eculizumab and two from the control group (5%) had extra-renal sequaelae during follow-up.

Conclusions:

This study did not show any signal for harm of eculizumab as a treatment of STEC-HUS. The benefit of eculizumab on renal and extra-renal outcomes in STEC-HUS could not be established based on our results. Larger prospective trials are required to assess the efficacy of eculizumab in this indication.