Title |
Eculizumab treatment in severe pediatric STEC-HUS: a multicenter retrospective study
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Published in |
Pediatric Nephrology, March 2018
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DOI | 10.1007/s00467-018-3903-9 |
Pubmed ID | |
Authors |
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 |
Abstract |
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. 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). 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. 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. |
X Demographics
Geographical breakdown
Country | Count | As % |
---|---|---|
United Kingdom | 2 | 14% |
Mexico | 1 | 7% |
United States | 1 | 7% |
France | 1 | 7% |
Canada | 1 | 7% |
Colombia | 1 | 7% |
Unknown | 7 | 50% |
Demographic breakdown
Type | Count | As % |
---|---|---|
Members of the public | 11 | 79% |
Scientists | 1 | 7% |
Practitioners (doctors, other healthcare professionals) | 1 | 7% |
Science communicators (journalists, bloggers, editors) | 1 | 7% |
Mendeley readers
Geographical breakdown
Country | Count | As % |
---|---|---|
Unknown | 46 | 100% |
Demographic breakdown
Readers by professional status | Count | As % |
---|---|---|
Student > Master | 8 | 17% |
Researcher | 7 | 15% |
Student > Postgraduate | 5 | 11% |
Student > Doctoral Student | 3 | 7% |
Student > Bachelor | 3 | 7% |
Other | 10 | 22% |
Unknown | 10 | 22% |
Readers by discipline | Count | As % |
---|---|---|
Medicine and Dentistry | 22 | 48% |
Nursing and Health Professions | 5 | 11% |
Pharmacology, Toxicology and Pharmaceutical Science | 3 | 7% |
Psychology | 2 | 4% |
Agricultural and Biological Sciences | 2 | 4% |
Other | 3 | 7% |
Unknown | 9 | 20% |