Abstract
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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Declaration of interests
The following authors declare interests. CMT and MB were scientific advisers to Thallion Pharmaceuticals. SJ, NvdK, GA, JVW, C. Licht, and MR have served on Advisory Boards for Alexion Pharmaceuticals. DK, C. Licht, and C. Loirat were the national coordinators of the multi-center Eculizumab trials in aHUS in Sweden, Canada, and France, respectively.
The European Paediatric Study Group for HUS is now known as HUS International and comprises pediatric nephrologists from Europe, North America, and the Middle East.
Contributors
Anne-Laure Adra1, Klaus Arbeiter2, Yelda Bilginer3, Heiko Billing4, Maria Bitsori5, Bernard Boudailliez6, Francisco de la Cerda – Ojeda7, Gérard Champion8, Amit Dagan9, Bradley Dixon, MD10, Philippe Eckart11, Israel Eisenstein12, Arnaud Garnier13, Clara Gomes14, Karsten Häffner15, Jerome Harambat16, Carol Inward17, Rafael Krmar18, Theresa Kwon19, Anne-Laure Lapeyraque20, Sebastian Loos21, Meeta Malik22, Mignon McCulloch23, David Milford24, Danko Milosevic25, Carla Nester26, François Nobili27, Pedro J Ortega28, Fotios Papachristou29, Antonia Peña - Carrión30, Ludmila Podracká31, Nikoleta Printza29, Bruno Ranchin32, Christopher Reid23, Caroline Rousset-Rouvière33, Rémi Salomon34, Anne-Laure Sellier-Leclerc35, James Springate36, Sophie Taque37, Despoina Tramma38, Sibylle Tschumi39, Ulrike Walden40, Kay Tyerman41, Aoife Waters42, Donald J Weaver Jr43
1Pediatric nephrology department, Hôpital A. de Villeneuve, Montpellier, France
2Medical University Vienna, Pediatric Nephrology, Vienna, Austria
3Hacettepe University Faculty of Medicine Pediatric Nephrology, Ankara, Turkey
4University Hospital Heidelberg, Pediatric Nephrology, Heidelberg, Germany
5Department of Pediatrics, University Hospital of Heraklion, Greece
6Department of Pediatrics, Centre Hospitalier Universitaire Nord, Amiens , France
7Virgen del Rocío Children´s Hospital, Division of Pediatric Nephrology, Seville, Spain
8Department of Pediatrics, Centre Hospitalier Universitaire, Angers, France
9Schneider Children's Hospital, Petach Tikvah (Tel Aviv University), Israel
10Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, USA
11Department of Pediatrics, Centre Hospitalier Universitaire, Caen, France
12Myers Children's Hospital (Rambam Medical Center), Haifa (Technion University), Israel
13Pediatric nephrology department, Centre Hospitalier Universitaire Purpan, Hôpital des enfants, Toulouse, France
14Coimbra Pediatric Hospital, Division of Pediatric Nephrology, Coimbra, Portugal
15University Hospital Freiburg, Department of Pediatrics, Freiburg, Germany
16Department of Pediatrics, Centre Hospitalier Universitaire, Bordeaux, France
17Department of Paediatric Nephrology, Bristol Royal Hospital for Children, Bristol, UK
18Karolinska University Hospital Huddinge, Stockholm, Sweden
19Pediatric nephrology department Hôpital Robert Debré, Paris, France
20Centre Hospitalier Universitaire Sainte Justine, Montreal Quebec
21University Hospital Hamburg, Pediatric Nephrology, Hamburg, Germany
22Children's Renal & Urology Unit, Nottingham University Hospitals, Nottingham, UK
23Paediatric Nephro-urology Unit, Evelina Children’s Hospital, London, UK
24Birmingham Children’s Hospital, Birmingham, UK
25University Hospital Center Zagreb, Croatia
26The University of Iowa, Iowa City, Iowa, USA
27Pediatric Nephrology, Centre Hospitalier Universitaire, Besançon, France
28La Fe Hospital, Division of Pediatric Nephrology, Valencia, Spain
291st Department of Paediatrics, Hippokration General Hospital, Aristotle University of Thessaloniki, Thessaloniki, Greece
30La Paz Universitary Hospital, Division of Pediatric Nephrology, Madrid, Spain
31Children's Hospital of P.J. Safarik's University, 1st Pediatric Department, Košice, Slovakia
32Pediatric nephrology department, Hospices Civils de Lyon, Lyon, France
33Pediatric nephrology department, Hôpital de la Timone, Marseille, France
34Pediatric nephrology department, Hôpital Necker-Enfants Malades, Paris, France
35Pediatric nephrology department, Hôpital Trousseau, Paris, France
36Division of Nephrology, Women & Children's Hospital, Buffalo, New York, USA
37Department of Pediatrics, Centre Hospitalier Universitaire, Rennes, France
38Aristotle University of Thessaloniki, Thessaloniki, Greece
39Paediatric Nephrology, University Children's Hospital Bern, Switzerland
40Childrens Hospital Augsburg, Augsburg, Germany
41Department of Paediatric Nephrology, Leeds General Infirmary, Leeds, UK
42Department of Paediatric Nephrology, Great Ormond Street Hospital, London, UK
43Levine Children's Hospital at Carolinas Medical Center, Charlotte, North Carolina, USA
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Johnson, S., Stojanovic, J., Ariceta, G. et al. An audit analysis of a guideline for the investigation and initial therapy of diarrhea negative (atypical) hemolytic uremic syndrome. Pediatr Nephrol 29, 1967–1978 (2014). https://doi.org/10.1007/s00467-014-2817-4
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DOI: https://doi.org/10.1007/s00467-014-2817-4