Follow-up of children with infection-associated haemolytic uraemic syndrome 1979-1995: Would eculizumab have improved prognosis?
JOURNAL OF PAEDIATRICS AND CHILD HEALTH(2020)
Abstract
The late outcome in 55 children with infection-mediated haemolytic uremic syndrome (Shiga Toxin E Coli (STEC)-HUS and pneumococcal HUS) observed in 1979-1995 was followed up 23years after disease onset. Of these, two were later confirmed to have atypical HUS (aHUS). Furthermore, of this population, five children had impaired kidney function at 3-months follow-up, which continued to deteriorate. These children had significant oliguria/anuria and hypertension during their illness requiring early dialysis and antihypertensive therapy. At 23years post-disease onset, all five (100%) of these children have developed end-stage kidney disease or chronic kidney disease. Eculizumab is a monoclonal antibody that binds with high affinity to the C5 protein of the complement pathway, a major component of the pathophysiology of infection-mediated HUS. There are no long-term randomised controlled trials in the literature to support its use in such cases. Our 23-year follow-up of a population of severely affected children with infection-mediated HUS demonstrates a high percentage of chronic kidney disease and end-stage kidney disease (19%). Randomised controlled trials with eculizumab are now being conducted in this affected cohort.
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Key words
eculizumab,haemolytic uremic syndrome,paediatrics
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