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Pediatric atypical hemolytic–uremic syndrome due to auto-antibodies against factor H: is there an interest to combine eculizumab and mycophenolate mofetil?

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Abstract

Background

Atypical hemolytic and uremic syndrome (aHUS), a thrombotic micro-angiopathy (TMA) caused by deregulation in the complement pathway, is sometimes due to the presence of anti-complement factor H (CFH) auto-antibodies. The “standard” treatment for such aHUS combines plasma exchange therapy and immunosuppressive drugs. Eculizumab, a monoclonal antibody that blocks the terminal pathway of the complement cascade, could be an interesting alternative in association with an immunosuppressive treatment for maintenance regimen.

Case–diagnosis/treatment

We report on two children, diagnosed with mildly severe aHUS due to anti-CFH antibodies, who were treated with the association eculizumab–mycophenolate mofetil (MMF). Neither side effects nor relapses were observed during the 3 years of follow-up; MMF was even progressively tapered and withdrawn successfully in one patient.

Conclusions

The association of eculizumab and MMF appears to be an effective and safe option in pediatric cases of aHUS due to anti-CFH antibodies of mild severity.

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Acknowledgements

The authors would like to thank Dr Véronique Frémeaux-Bacchi for the genetic explorations performed in the patients.

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Authors and Affiliations

  1. Centre de Référence des Maladies Rénales Rares, Service de Néphrologie Rhumatologie Dermatologie Pédiatriques, Hôpital Femme Mère Enfant, 59 Boulevard Pinel, 69677, Bron Cedex, France

    Lucie Matrat, Justine Bacchetta, Bruno Ranchin, Corentin Tanné & Anne-Laure Sellier-Leclerc

  2. Faculté de Médecine Lyon Est, Université Lyon 1, Lyon, France

    Justine Bacchetta & Corentin Tanné

  3. Service de pédiatrie et néonatalogie, médecine du sport, hôpitaux du pays du Mont-Blanc, Sallanches, France

    Corentin Tanné

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  1. Lucie Matrat
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  2. Justine Bacchetta
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  3. Bruno Ranchin
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  4. Corentin Tanné
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  5. Anne-Laure Sellier-Leclerc
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Corresponding authors

Correspondence to Lucie Matrat or Anne-Laure Sellier-Leclerc.

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Conflict of interest

JB received travel grants and honoraria from Alexion.

ALL received travel grants from Alexion and honararia from Alexion (2013, 2014).

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Matrat, L., Bacchetta, J., Ranchin, B. et al. Pediatric atypical hemolytic–uremic syndrome due to auto-antibodies against factor H: is there an interest to combine eculizumab and mycophenolate mofetil?. Pediatr Nephrol 36, 1647–1650 (2021). https://doi.org/10.1007/s00467-021-05025-8

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  • DOI: https://doi.org/10.1007/s00467-021-05025-8

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