BACKGROUND: In 2009, the European Paediatric Study Group for Haemolytic Uraemic Syndrome (HUS) published a clinical practice guideline for the investigation and initial therapy of diarrhea-negative HUS (now more widely referred to as atypical HUS, aHUS). The therapeutic component of the guideline (comprising early, high-volume plasmapheresis) was derived from anecdotal evidence and expert consensus, and the authors committed to auditing outcome. METHODS: Questionnaires were distributed to pediatric nephrologists across Europe, North America, and the Middle East, who were asked to complete one questionnaire per patient episode of aHUS between July 1, 2009 and December 31, 2010. Comprehensive, anonymous demographic and clinical data were collected. RESULTS: Seventy-one children were reported with an episode of aHUS during the audit period. Six cases occurred on a background of influenza A H1N1 infection. Of 71 patients, 59 (83 %) received plasma therapy within the first 33 days, of whom ten received plasma infusion only. Complications of central venous catheters occurred in 16 out of 51 patients with a catheter in-situ (31 %). Median time to enter hematological remission was 11.5 days, and eight of 71 (11 %) patients did not enter hematological remission by day 33. Twelve patients (17 %) remained dialysis dependent at day 33. CONCLUSIONS: This audit provides a snapshot of the early outcome of a group of children with aHUS in the months prior to more widespread use of eculizumab.
BACKGROUND: In 2009, the European Paediatric Study Group for Haemolytic Uraemic Syndrome (HUS) published a clinical practice guideline for the investigation and initial therapy of diarrhea-negative HUS (now more widely referred to as atypical HUS, aHUS). The therapeutic component of the guideline (comprising early, high-volume plasmapheresis) was derived from anecdotal evidence and expert consensus, and the authors committed to auditing outcome. METHODS: Questionnaires were distributed to pediatric nephrologists across Europe, North America, and the Middle East, who were asked to complete one questionnaire per patient episode of aHUS between July 1, 2009 and December 31, 2010. Comprehensive, anonymous demographic and clinical data were collected. RESULTS: Seventy-one children were reported with an episode of aHUS during the audit period. Six cases occurred on a background of influenza A H1N1 infection. Of 71 patients, 59 (83 %) received plasma therapy within the first 33 days, of whom ten received plasma infusion only. Complications of central venous catheters occurred in 16 out of 51 patients with a catheter in-situ (31 %). Median time to enter hematological remission was 11.5 days, and eight of 71 (11 %) patients did not enter hematological remission by day 33. Twelve patients (17 %) remained dialysis dependent at day 33. CONCLUSIONS: This audit provides a snapshot of the early outcome of a group of children with aHUS in the months prior to more widespread use of eculizumab.
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Authors: C M Legendre; C Licht; P Muus; L A Greenbaum; S Babu; C Bedrosian; C Bingham; D J Cohen; Y Delmas; K Douglas; F Eitner; T Feldkamp; D Fouque; R R Furman; O Gaber; M Herthelius; M Hourmant; D Karpman; Y Lebranchu; C Mariat; J Menne; B Moulin; J Nürnberger; M Ogawa; G Remuzzi; T Richard; R Sberro-Soussan; B Severino; N S Sheerin; A Trivelli; L B Zimmerhackl; T Goodship; C Loirat Journal: N Engl J Med Date: 2013-06-06 Impact factor: 91.245
Authors: Mathieu Lemaire; Véronique Frémeaux-Bacchi; Franz Schaefer; Murim Choi; Wai Ho Tang; Moglie Le Quintrec; Fadi Fakhouri; Sophie Taque; François Nobili; Frank Martinez; Weizhen Ji; John D Overton; Shrikant M Mane; Gudrun Nürnberg; Janine Altmüller; Holger Thiele; Denis Morin; Georges Deschenes; Véronique Baudouin; Brigitte Llanas; Laure Collard; Mohammed A Majid; Eva Simkova; Peter Nürnberg; Nathalie Rioux-Leclerc; Gilbert W Moeckel; Marie Claire Gubler; John Hwa; Chantal Loirat; Richard P Lifton Journal: Nat Genet Date: 2013-03-31 Impact factor: 41.307
Authors: Chantal Loirat; Fadi Fakhouri; Gema Ariceta; Nesrin Besbas; Martin Bitzan; Anna Bjerre; Rosanna Coppo; Francesco Emma; Sally Johnson; Diana Karpman; Daniel Landau; Craig B Langman; Anne-Laure Lapeyraque; Christoph Licht; Carla Nester; Carmine Pecoraro; Magdalena Riedl; Nicole C A J van de Kar; Johan Van de Walle; Marina Vivarelli; Véronique Frémeaux-Bacchi Journal: Pediatr Nephrol Date: 2015-04-11 Impact factor: 3.714