Pediatric Nephrology

, Volume 29, Issue 12, pp 2415–2419

Eculizumab in neonatal hemolytic uremic syndrome with homozygous factor H deficiency

Authors

    • Centre de Référence des Maladies Rénales Rares, Hôpital Femme Mère EnfantHospices Civils de Lyon
  • Justine Bacchetta
    • Centre de Référence des Maladies Rénales Rares, Hôpital Femme Mère EnfantHospices Civils de Lyon
    • Université Lyon 1
  • Etienne Javouhey
    • Réanimation Pédiatrique, Hôpital Femme Mère EnfantHospices Civils de Lyon
    • Université Lyon 1
  • Pierre Cochat
    • Centre de Référence des Maladies Rénales Rares, Hôpital Femme Mère EnfantHospices Civils de Lyon
    • Université Lyon 1
  • Véronique Frémaux-Bacchi
    • Laboratoire d’immunologie, Hôpital Georges PompidouAssistance publique–Hôpitaux de Paris (AP-HP)
  • Anne-Laure Sellier-Leclerc
    • Centre de Référence des Maladies Rénales Rares, Hôpital Femme Mère EnfantHospices Civils de Lyon
Brief Report

DOI: 10.1007/s00467-014-2933-1

Cite this article as:
Michaux, K., Bacchetta, J., Javouhey, E. et al. Pediatr Nephrol (2014) 29: 2415. doi:10.1007/s00467-014-2933-1

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).

Keywords

Atypical hemolytic uremic syndromeHomozygousFactor H deficiencyEculizumabInfant

Copyright information

© IPNA 2014