Eculizumab treatment in severe pediatric STEC-HUS: a multicenter retrospective study

  • Lucas Percheron
  • Raluca Gramada
  • Stéphanie Tellier
  • Remi Salomon
  • Jérôme Harambat
  • Brigitte Llanas
  • Marc Fila
  • Emma Allain-Launay
  • Anne-Laure Lapeyraque
  • Valerie Leroy
  • Anne-Laure Adra
  • Etienne Bérard
  • Guylhène Bourdat-Michel
  • Hassid Chehade
  • Philippe Eckart
  • Elodie Merieau
  • Christine Piètrement
  • Anne-Laure Sellier-Leclerc
  • Véronique Frémeaux-Bacchi
  • Chloe Dimeglio
  • Arnaud Garnier
Original Article

Abstract

Background

Hemolytic uremic syndrome related to Shiga-toxin-secreting Escherichia coli infection (STEC-HUS) remains a common cause of acute kidney injury in young children. No specific treatment has been validated for this severe disease. Recently, experimental studies highlight the potential role of complement in STEC-HUS pathophysiology. Eculizumab (EC), a monoclonal antibody against terminal complement complex, has been used in severe STEC-HUS patients, mostly during the 2011 German outbreak, with conflicting results.

Methods

On behalf of the French Society of Pediatric Nephrology, we retrospectively studied 33 children from 15 centers treated with EC for severe STEC-HUS. Indication for EC was neurologic involvement in 20 patients, cardiac and neurologic involvement in 8, cardiac involvement in 2, and digestive involvement in 3. Based on medical status at last follow-up, patients were divided into two groups: favorable (n = 15) and unfavorable outcomes (n = 18).

Results

Among patients with favorable outcome, 11/14 patients (79%) displayed persistent blockade of complement activity before each EC reinjection. Conversely, in patients with unfavorable outcome, only 9/15 (53%) had persistent blockade (p = n.s.). Among 28 patients presenting neurological symptoms, 19 had favorable neurological outcome including 17 with prompt recovery following first EC injection. Only two adverse effects potentially related to EC treatment were reported.

Conclusions

Taken together, these results may support EC use in severe STEC-HUS patients, especially those presenting severe neurological symptoms. The study, however, is limited by absence of a control group and use of multiple therapeutic interventions in treatment groups. Thus, prospective, controlled trials should be undertaken.

Keywords

Hemolytic uremic syndrome Acute kidney injury Pediatric Complement 

Notes

Compliance with ethical standards

All parents gave informed consent for their children. The National Code on Clinical Trials has declared that ethics approval is not necessary for retrospective studies.

Conflict of interest

The authors declare that they have no conflict of interest.

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Copyright information

© IPNA 2018

Authors and Affiliations

  • Lucas Percheron
    • 1
  • Raluca Gramada
    • 2
  • Stéphanie Tellier
    • 1
  • Remi Salomon
    • 3
  • Jérôme Harambat
    • 4
  • Brigitte Llanas
    • 4
  • Marc Fila
    • 5
  • Emma Allain-Launay
    • 6
  • Anne-Laure Lapeyraque
    • 7
  • Valerie Leroy
    • 8
  • Anne-Laure Adra
    • 9
  • Etienne Bérard
    • 10
  • Guylhène Bourdat-Michel
    • 11
  • Hassid Chehade
    • 12
  • Philippe Eckart
    • 13
  • Elodie Merieau
    • 14
  • Christine Piètrement
    • 15
  • Anne-Laure Sellier-Leclerc
    • 16
  • Véronique Frémeaux-Bacchi
    • 17
  • Chloe Dimeglio
    • 18
  • Arnaud Garnier
    • 1
  1. 1.Service de Néphrologie PédiatriqueHôpital des enfants, CHU PurpanToulouseFrance
  2. 2.Service de Neuroradiologie Diagnostique et ThérapeutiqueCHU PurpanToulouseFrance
  3. 3.Service de Néphrologie PédiatriqueHôpital Necker Enfants Malades, Assistance Publique-Hôpitaux de ParisParisFrance
  4. 4.Service de Néphrologie PédiatriqueHôpital Pellegrin-Enfants, CHU BordeauxBordeauxFrance
  5. 5.Service de Néphrologie PédiatriqueHôpital Robert Debré-Paris, Assistance Publique-Hôpitaux de ParisParisFrance
  6. 6.Service de Néphrologie PédiatriqueHôpital Mère-Enfants, CHU NantesNantesFrance
  7. 7.Service de Néphrologie PédiatriqueCHU de Sainte-Justine à Montréal CanadaMontréalCanada
  8. 8.Service de Néphrologie PédiatriqueHôpital Jeanne de Flandre, CHU LilleLilleFrance
  9. 9.Service de Néphrologie PédiatriqueHôpital Arnaud de Villeneuve, CHU MontpellierMontpellierFrance
  10. 10.Service de Néphrologie PédiatriqueHôpital Archet 2, CHU NiceNiceFrance
  11. 11.Service de pédiatrieHôpital Couple-Enfants, CHU GrenobleGrenobleFrance
  12. 12.Service de Néphrologie PédiatriqueCHU de Lausanne SuisseLausanneSwitzerland
  13. 13.Service de Pédiatrie MédicaleHôpital côte de nacre, CHU CaenCaenFrance
  14. 14.Service de NéphrologieHôpital Clocheville, CHU ToursToursFrance
  15. 15.Service de PédiatrieHôpital Américain, CHU ReimsReimsFrance
  16. 16.Service de Néphrologie PédiatriqueHôpital femme mère enfant, Hospices Civils de LyonLyonFrance
  17. 17.Laboratoire d’immunologieHôpital Européen Georges Pompidou, Assistance Publique-Hôpitaux de ParisParisFrance
  18. 18.Biostatistiques, Informatique Médicale, UMR 1027 InsermUniversité Paul SabatierToulouseFrance

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