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Eculizumab in neonatal hemolytic uremic syndrome with homozygous factor H deficiency

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Abstract

Background

Neonatal atypical hemolytic uremic syndrome (aHUS) is a rare but severe disease that is mainly due to methylmalonic aciduria or genetic complement abnormalities. Traditional management of aHUS includes plasma infusion/exchange, but in small or unstable infants, plasma exchange can be challenging because of high extracorporeal volume and difficulty to obtain an adequate venous access. The C5 complement blocker eculizumab has become a cornerstone of first-line management of aHUS due to complement deregulation in older patients. However, little data are available on its use in neonatal aHUS.

Case-diagnosis/treatment

We report on an 11-day-old neonate with severe aHUS (myocardial impairment, respiratory failure, acute kidney disease requiring hemodiafiltration) due to homozygous factor-H deficiency. She received early treatment with eculizumab as first-line therapy and completely recovered within 5 days. A second dose of eculizumab was administered 7 days after the first infusion, followed by a dose every 2 weeks for 2 months and then every 3 weeks, at the same dosage (300 mg). With more than 24 months of follow-up, renal function remains normal.

Conclusions

We report on the long-term efficacy and safety of eculizumab as first-line therapy in neonatal aHUS. However its use still requires optimization in terms of indications and administration (frequency, dosage).

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Acknowledgments

The authors would like to thank Thabo Mahendiran, Dr Agnès Veyradier (unité INSERM, Hôpital Kremlin-Bicêtre, Paris) and Pr Chantal Loirat (Hôpital Robert Debré, Paris) for their help.

Financial disclosure statement

none

Conflicts of interest

Veronique Fremeaux-Bacchi had received fees from Alexion Pharmaceuticals for invited lectures and participation on advisory boards

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Authors and Affiliations

  1. Centre de Référence des Maladies Rénales Rares, Hôpital Femme Mère Enfant, Hospices Civils de Lyon, 59 boulevard Pinel Bron, 69677, Bron Cedex, France

    Katell Michaux, Justine Bacchetta, Pierre Cochat & Anne-Laure Sellier-Leclerc

  2. Réanimation Pédiatrique, Hôpital Femme Mère Enfant, Hospices Civils de Lyon, Bron Cedex, France

    Etienne Javouhey

  3. Université Lyon 1, Lyon, France

    Justine Bacchetta, Etienne Javouhey & Pierre Cochat

  4. Laboratoire d’immunologie, Hôpital Georges Pompidou, Assistance publique–Hôpitaux de Paris (AP-HP), Paris, France

    Véronique Frémaux-Bacchi

Authors
  1. Katell Michaux
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  2. Justine Bacchetta
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  3. Etienne Javouhey
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  4. Pierre Cochat
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  5. Véronique Frémaux-Bacchi
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  6. Anne-Laure Sellier-Leclerc
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Corresponding author

Correspondence to Katell Michaux.

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Michaux, K., Bacchetta, J., Javouhey, E. et al. Eculizumab in neonatal hemolytic uremic syndrome with homozygous factor H deficiency. Pediatr Nephrol 29, 2415–2419 (2014). https://doi.org/10.1007/s00467-014-2933-1

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  • DOI: https://doi.org/10.1007/s00467-014-2933-1

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