title: Long-term remission of atypical HUS with anti-factor H antibodies after cyclophosphamide pulses.
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.
authors.affiliation: Néphrologie Pédiatrique, Hôpital Necker - Enfants Malades, MARHEA, Assistance Publique Hôpitaux de Paris (APHP), Inserm U983, IMAGINE, Université Paris Descartes-Sorbonne Paris-Cité, Paris, France.
authors.forename: Gwenaëlle
authors.initials: G
authors.lastname: Sana
authors.forename: Marie-Agnès
authors.initials: MA
authors.lastname: Dragon-Durey
authors.forename: Marina
authors.initials: M
authors.lastname: Charbit
authors.forename: Karim
authors.initials: K
authors.lastname: Bouchireb
authors.forename: Caroline
authors.initials: C
authors.lastname: Rousset-Rouvière
authors.forename: Etienne
authors.initials: E
authors.lastname: Bérard
authors.forename: Rémi
authors.initials: R
authors.lastname: Salomon
authors.forename: Véronique
authors.initials: V
authors.lastname: Frémeaux-Bacchi
authors.forename: Patrick
authors.initials: P
authors.lastname: Niaudet
authors.forename: Olivia
authors.initials: O
authors.lastname: Boyer
mesh_terms: D000893:Anti-Inflammatory Agents / Q000008:administration & dosage*; D065766:Atypical Hemolytic Uremic Syndrome; D001323:Autoantibodies / Q000097:blood / Q000276:immunology*; D001324:Autoantigens / Q000276:immunology; D002648:Child; D002675:Child, Preschool; D017242:Complement Factor H / Q000276:immunology*; D003520:Cyclophosphamide / Q000008:administration & dosage*; D005260:Female; D006463:Hemolytic-Uremic Syndrome / Q000097:blood / Q000188:drug therapy* / Q000276:immunology; D006801:Humans; D007223:Infant; D007677:Kidney Function Tests; D008297:Male; D010951:Plasma Exchange; D011241:Prednisone / Q000008:administration & dosage; D012074:Remission Induction; D013995:Time; D016896:Treatment Outcome
chemical_list: D000893:Anti-Inflammatory Agents; D001323:Autoantibodies; D001324:Autoantigens; D017242:Complement Factor H; D003520:Cyclophosphamide; D011241:Prednisone