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Anti-CFH-associated hemolytic uremic syndrome: do we still need plasma exchange?

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An Editorial Commentary to this article was published on 18 April 2024

Abstract

Background

Between 5 and 50% of atypical hemolytic uremic syndrome (aHUS) cases in children are caused by autoantibodies against complement factor H (CFH). Given the acquired autoimmune nature of the disease, plasma exchange (PE) and various immunosuppressive treatments have been used. More recently, eculizumab has been proposed.

Methods

In this multicenter, retrospective study, we report outcomes of 12 children with anti-FH antibody-associated HUS treated with eculizumab associated with various immunosuppressive regimens.

Results

Patients were treated with eculizumab for 15.5 [9.5;23.0] months and 3 received PE or IgG adsorption. Three patients received mycophenolate mofetil (MMF) alone, 1 patient received MMF and steroids, 1 patient received MMF and rituximab, 3 patients received MMF/steroids and rituximab, and 4 patients did not receive any immunosuppression. Anti-FH antibody levels significantly decreased but no difference was observed based on the immunosuppressive regimen. Eculizumab was discontinued in 7/10 patients after 11 [7.5;15.5] months and MMF in 6/8 patients after 36 [35;40] months. Anti-FH titers at MMF discontinuation ranged from 257 to 3425 UI/L. None of these patients relapsed and eGFR at last follow-up was above 70 mL/min/1.73 m2 in all patients.

Conclusions

Eculizumab is effective and safe in inducing and maintaining remission in aHUS secondary to anti-FH antibodies and renders reduction of anti-FH titers less urgent. Anti-FH antibody titers decreased in most patients irrespective of the immunosuppressive treatment chosen, so that a strategy consisting of combining eculizumab with MMF monotherapy seems sufficient at least in non-Indian or less severe forms of anti-FH antibody-associated HUS.

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Data availability

The dataset generated during the current study is available from the corresponding author on reasonable request.

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Authors and Affiliations

  1. Pediatric Nephrology Department, Centre de référence des Maladies Rénales Rares MARHEA, Filières Maladies Rares ORKID et ERK-Net, Robert Debré Hospital, APHP, 48 bd Sérurier, 75019, Paris, France

    Marion Ferri, Claire Dossier, Charlotte Duneton, Thérésa Kwon & Julien Hogan

  2. Division of Nephrology, Laboratory of Nephrology, Ospedale Pediatrico Bambino Gesù IRCCS, Rome, Italy

    Federica Zotta & Marina Vivarelli

  3. Istituto di Ricerche Farmacologiche Mario Negri IRCCS, Ranica, Bergamo, Italy

    Roberta Donadelli & Lisa Quadri

  4. Assistance Publique-Hôpitaux de Paris, Department of Biological Immunology, Hôpital Européen Georges Pompidou, and INSERM UMR S1138, Complément et Maladies, Centre de Recherche des Cordeliers, Paris, France

    Carine El-Sissy & Véronique Fremeau-Bacchi

  5. Paediatric Clinic, IRCCS Ospedale Maggiore Policlinico Sant’Orsola, Department of Medicine and Surgery, University of Bologna, Bologna, Italy

    Andrea Pasini

  6. Department of Pediatric Nephrology, Centre de Référence des Maladies Rénales Rares Néphrogones, Filières Maladies Rares ORKID et ERK-Net, CHU de Lyon, Bron, France

    Anne-Laure Sellier-Leclerc

  7. INSERM, UMR-U970, PARCC, Paris Institute for Transplantation and Organ Regeneration, Université Paris Cité, Paris, France

    Julien Hogan

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  1. Marion Ferri
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  2. Federica Zotta
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Corresponding author

Correspondence to Julien Hogan.

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Conflict of interest

ALL received travel grants from Alexion and honoraria from Alexion. JH received travel grants from Alexion.

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Ferri, M., Zotta, F., Donadelli, R. et al. Anti-CFH-associated hemolytic uremic syndrome: do we still need plasma exchange?. Pediatr Nephrol (2024). https://doi.org/10.1007/s00467-024-06373-x

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