This protocol applies to atypical hemolytic-uremic syndrome (aHUS) in patients without anti-factor H antibodies, where an initial course of plasma exchange (PEX) has not achieved the expected hematological remission targets.
Atypical HUS without anti-factor H (anti-FH) antibodies — a population in which inherited defects of the alternative complement pathway are a recognised underlying cause. Plasma exchange or plasma infusions represent the established first-line approach in this setting.
The preceding treatment was prompt initiation of plasma exchange (PEX), targeting hematological remission defined as all three of:
Failure to reach these targets is the criterion that triggers escalation to this next-line protocol.
In this situation, a humanized monoclonal antibody directed at complement component C5 is the intervention of choice. The complete indications, sequencing, and monitoring parameters are detailed in the full structured protocol.
Clinical goal: hematological remission is expected at a median of 7–8 days.
DOI: 10.1007/s00467-019-04233-7
Inherited defects of the alternative pathway are the chief cause of aHUS in Europe and North America.
Thus, PEX or plasma infusions remain the chief option for patients with aHUS, especially those with genetic defects in the complement pathway.
We recommend efforts to enable therapy with eculizumab in the following: (i) lack of remission despite 7–10 days of PEX, (ii) life-threatening features (seizures, cardiac dysfunction), (iii) complications due to PEX or vascular access, and (iv) inherited defect in complement regulation.
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