For children with a clinical diagnosis of aHUS, we propose eculizumab as first-line treatment, to avoid plasma exchange and the complications of central venous double line catheters.
In such cases, confirmation of a complement mutation is not required prior to treatment initiation. Undue treatment delay may affect ultimate renal recovery and increase the risk of early progression to end-stage renal disease.
When possible, eculizumab treatment should be initiated within 24–48 h of onset or admission.If eculizumab is not (or not immediately) available, plasma exchange (or plasma infusions if plasma exchange is not possible) should be started.
While the indication of complement blockade treatment is not limited to aHUS patients with a confirmed complement mutation as this treatment is also effective in patients without complement mutation, genetic screening is required for the longer-term management of patients.
Anti-CFH antibody testing is the only complement investigation urgently needed during the acute phase, as a positive result raises additional treatment options.
Comments by Evaluators:
Clear, easy to read, addresses specific management questions.
Authors preferred a narrative approach rather than a more quantifiable methodology
Little emphasis given to methodology description
No stakeholders other than physicians/scientists involved.