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Outcome of children with Shiga toxin-associated haemolytic uraemic syndrome treated with eculizumab: a matched cohort study

dc.rights.licenseopenen_US
dc.contributor.authorMONET-DIDAILLER, C.
dc.contributor.authorCHEVALLIER, A.
dc.contributor.authorGODRON-DUBRASQUET, A.
dc.contributor.authorALLARD, L.
dc.contributor.authorDELMAS, Y.
dc.contributor.authorCONTIN-BORDES, C.
dc.contributor.authorBRISSAUD, O.
dc.contributor.authorLLANAS, B.
hal.structure.identifierBordeaux population health [BPH]
dc.contributor.authorHARAMBAT, Jerome
IDREF: 110567358
dc.date.accessioned2021-03-09T15:27:57Z
dc.date.available2021-03-09T15:27:57Z
dc.date.issued2020-12-04
dc.identifier.issn1460-2385 (Electronic) 0931-0509 (Linking)en_US
dc.identifier.urihttps://oskar-bordeaux.fr/handle/20.500.12278/26471
dc.description.abstractEnBACKGROUND: Treatment with eculizumab in Shiga toxin-associated haemolytic and uraemic syndrome (STEC-HUS) remains controversial despite its increasing utilization. The aim of our study was to evaluate the outcomes of children treated with eculizumab for STEC-HUS in a single-centre matched cohort study. METHODS: Data were retrospectively collected from medical records of children diagnosed with STEC-HUS. The outcomes of patients treated with eculizumab for STEC-HUS were compared with those of a control group of untreated patients matched for age, sex and severity of acute kidney injury with a 1:2 matching scheme. RESULTS: Eighteen children (median age 40.6 months) with STEC-HUS treated with eculizumab were compared with 36 matched control patients (median age 36.4 months) who did not receive eculizumab. All patients survived in the two groups. Within 1 month of HUS onset, the evolution of haematological and renal parameters did not differ between the two groups. At 12 months of follow-up, renal outcome was not significantly different between the two groups. At the last follow-up, the prevalence of decreased glomerular filtration rate in the eculizumab group (27%) was not statistically different from that in controls (38%), as was the prevalence of proteinuria and high blood pressure. Children who received eculizumab more often had extrarenal sequelae during follow-up. Eculizumab treatment appeared to be safe in children with STEC-HUS. CONCLUSION: The benefit of eculizumab on renal and extrarenal outcomes in STEC-HUS could not be established based on our findings. However, efficacy and safety are not best assessed by the observational design and small sample size of our study. Randomized controlled trials are thus required to determine the efficacy of eculizumab in this indication.
dc.language.isoENen_US
dc.title.enOutcome of children with Shiga toxin-associated haemolytic uraemic syndrome treated with eculizumab: a matched cohort study
dc.title.alternativeNephrol Dial Transplanten_US
dc.typeArticle de revueen_US
dc.identifier.doi10.1093/ndt/gfz158en_US
dc.subject.halSciences du Vivant [q-bio]/Santé publique et épidémiologieen_US
dc.identifier.pubmed31411695en_US
bordeaux.journalNephrology Dialysis Transplantationen_US
bordeaux.page2147-2153en_US
bordeaux.volume35en_US
bordeaux.hal.laboratoriesBordeaux Population Health Research Center (BPH) - U1219en_US
bordeaux.issue12en_US
bordeaux.institutionUniversité de Bordeauxen_US
bordeaux.teamLEHA_BPH
bordeaux.peerReviewedouien_US
bordeaux.inpressnonen_US
hal.identifierhal-03164090
hal.version1
hal.date.transferred2021-03-09T15:28:00Z
hal.exporttrue
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