Pediatric Nephrology

, Volume 29, Issue 1, pp 75–83

Long-term remission of atypical HUS with anti-factor H antibodies after cyclophosphamide pulses

  • Gwenaëlle Sana
  • Marie-Agnès Dragon-Durey
  • Marina Charbit
  • Karim Bouchireb
  • Caroline Rousset-Rouvière
  • Etienne Bérard
  • Rémi Salomon
  • Véronique Frémeaux-Bacchi
  • Patrick Niaudet
  • Olivia Boyer
Original Article

DOI: 10.1007/s00467-013-2558-9

Cite this article as:
Sana, G., Dragon-Durey, MA., Charbit, M. et al. Pediatr Nephrol (2014) 29: 75. doi:10.1007/s00467-013-2558-9
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Abstract

Background

Anti-complement factor H (CFH) autoantibody (Ab)-associated atypical hemolytic uremic syndrome (aHUS) has a poor prognosis, but no consensus exists on its treatment.

Methods

We report the follow-up of four children with anti-CFH Ab (8,000 to >32,000 arbitrary units)-associated aHUS after plasma exchanges (PEs), prednisone, and cyclophosphamide pulse therapy with the evolution of anti-CFH Ab titers and kidney function.

Results

Patient 1 received PEs + prednisone + cyclophosphamide pulses after two relapses following PEs and then PEs + rituximab. The other three patients were treated with PEs + prednisone + cyclophosphamide pulses as a first-line therapy. In our four patients, the induction protocol combining PEs + prednisone + cyclophosphamide pulses led to a rapid and sustained remission up to 6 years, 4 years and 4 months without any maintenance therapy. Kidney function was normal and anti-CFH Ab titer decreased, but remained detectable during remission without any clinical or biological signs of relapse.

Conclusions

We demonstrate the long-term efficiency and safety of cyclophosphamide pulses combined with PEs and prednisone in anti-CFH Ab-associated aHUS leading to a prolonged decrease in anti-CFH Ab titers and prevention of relapses without the need for maintenance therapy.

Keywords

Acute kidney injury Anti-complement factor H autoantibody Atypical hemolytic uremic syndrome Children Cyclophosphamide 

Copyright information

© IPNA 2013

Authors and Affiliations

  • Gwenaëlle Sana
    • 1
  • Marie-Agnès Dragon-Durey
    • 2
  • Marina Charbit
    • 1
  • Karim Bouchireb
    • 1
  • Caroline Rousset-Rouvière
    • 3
  • Etienne Bérard
    • 4
  • Rémi Salomon
    • 1
  • Véronique Frémeaux-Bacchi
    • 2
  • Patrick Niaudet
    • 1
  • Olivia Boyer
    • 1
    • 5
  1. 1.Néphrologie Pédiatrique, Hôpital Necker – Enfants Malades, MARHEAAssistance Publique Hôpitaux de Paris (APHP), Inserm U983, IMAGINE, Université Paris Descartes-Sorbonne Paris-CitéParisFrance
  2. 2.Laboratoire d’Immunologie, Hôpital Européen Georges-Pompidou, APHPUniversité Paris Descartes-Sorbonne Paris-CitéParisFrance
  3. 3.Unité de Néphrologie Pédiatrique, Hôpital La Timone, AP-HMUniversité de la MéditerranéeMarseilleFrance
  4. 4.Néphrologie Pédiatrique, Hôpitaux Pédiatriques de Nice CHU-LenvalUniversité de Nice-Sophia-AntipolisNiceFrance
  5. 5.Service de Néphrologie PédiatriqueHôpital Necker – Enfants MaladesParis cedex 15France

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