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Case Reports
. 2025 Apr;40(4):1041-1047.
doi: 10.1007/s00467-024-06530-2. Epub 2024 Oct 9.

Eculizumab as first-line treatment for patients with severe presentation of complement factor H antibody-mediated hemolytic uremic syndrome

Paula A Coccia  1 , Laura F Alconcher  2 , Veronica Ferraris  3 , Lucas I Lucarelli  2 , Maria A Grillo  3 , Maria Andrea Arias  4 , Mariana Saurit  5 , Viviana M Ratto  6 , Celia Dos Santos  7 , Analía Sánchez-Luceros  7
Affiliations
Case Reports

Eculizumab as first-line treatment for patients with severe presentation of complement factor H antibody-mediated hemolytic uremic syndrome

Paula A Coccia et al. Pediatr Nephrol. 2025 Apr.

Abstract

Background: Complement factor H (FH) antibody-mediated hemolytic uremic syndrome (HUS) has varying prevalence globally. Plasmapheresis and immunosuppressive drugs are the standard treatment. Recently, eculizumab has been reported as an effective alternative. This study aims to report four children with FH antibody-mediated HUS managed with eculizumab plus immunosuppression as first-line therapy.

Methods: A retrospective chart review was conducted for children aged ≤ 18 years old with complement-mediated HUS in two referral centers. Patients with FH antibody-mediated HUS treated with eculizumab as first-line therapy were included.

Results: Four children (aged 6-11 years old) were included. Dialysis was necessary in three patients. Eculizumab was administered 5-23 days after onset. None of them received plasmapheresis. Prednisone and mycophenolate mofetil were added after receiving positive FH antibody results. Hematological signs and kidney function improved after the second eculizumab dose. Eculizumab was discontinued in three patients after 6 months. One patient required rituximab due to persistent high FH antibody titers; discontinuation of eculizumab occurred after 15 months without recurrence. No treatment-related complications were observed. During a mean 12-month follow-up (range 6-24 months), no relapses were recorded and all patients ended with normal GFR.

Conclusion: Our data suggest that a short course of 6 months of C5 inhibitor might be sufficient to reverse thrombotic microangiopathy symptoms and improve kidney function in patients with severe FH antibody-mediated HUS. Simultaneously, adding immunosuppressive agents might reduce the risk of relapse and allow cessation of C5 inhibition in a shorter period of time.

Keywords: Anti-factor H antibodies; Atypical HUS; Complement factor H; Complement-mediated HUS; Eculizumab; Thrombotic microangiopathy.

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Conflict of interest statement

Declarations. Ethical considerations: Ethical approval was obtained via the ethics committee of Hospital Italiano de Buenos Aires. Due to the retrospective nature and because the study involved chart review of children admitted within the study period to the institutions who have been already discharged, the study received a waiver for informed consent. Conflict of interest: The authors declare no competing interests.

References

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