Eculizumab treatment in severe pediatric STEC-HUS: a multicenter retrospective study

Abstract

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.

Verotoxin Receptor-Based Pathology and Therapies

“…Since VT can activate complement, this approach has been tested for efficacy in eHUS (Rasa et al, 2017;Walsh and Johnson, 2019). Therapeutic effect has been shown, particularly for patients with neurological symptoms (Percheron et al, 2018) but results overall are, as yet, highly variable (Loos et al, 2018;Monet-Didailler et al, 2019).…”

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

“…Haemolytic uraemic syndrome (HUS) was previously the most common cause of acute kidney injury in childhood . It is characterised by microangiopathic haemolytic anaemia, thrombocytopenia and acute renal insufficiency . HUS has historically been classified into two forms: (i) typical following a diarrhoeal illness and (ii) atypical, which often has an inherited and/or acquired complement abnormality .…”

“…TMA can manifest with a diverse range of signs and symptoms; however, acute kidney injury is a prominent feature due to the propensity of glomerular circulation to endothelial damage and occlusion . HUS caused by the shiga toxin‐producing Escherichia coli (most frequently E. coli serotype O157:H7) is defined by the presence of shiga toxin‐producing bacteria (STEC‐HUS) . There is evidence of complement activation via an alternate pathway in STEC‐HUS patients .…”

“…In STEC‐HUS, short‐term studies in children show a 10–15% risk of permanent renal impairment. This incidence is the highest in patients with prolonged anuria or longer duration of dialysis . Brenner et al .…”

“…Eculizumab may reduce cortical necrosis in the acute stage and subsequent hyperfiltration damage. In STEC‐HUS, there have been some small limited studies with eculizumab but no firm support for its administration in such cases . In Australia, eculizumab is an expensive agent, and its use is restricted to patients with a confirmed diagnosis of aHUS.…”