Follow-up of children with infection-associated haemolytic uraemic syndrome 1979-1995: Would eculizumab have improved prognosis?

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 23 years 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 23 years 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.