Cold agglutinin disease is a haemolytic anaemia that requires active management when symptoms are present. This protocol addresses the treatment step indicated when a prior complement inhibition regimen has not met its haematological goals, necessitating escalation to the next line of therapy.
Complement inhibition with sutimlimab (intravenous infusion on day 0, day 7, then every two weeks) or with eculizumab (in patients with primarily intravascular haemolysis) is considered to have failed when the following targets are not reached:
Non-achievement of these targets is the indication for escalation to the protocol described here.
The regimen at this stage involves a combination of agents and is reserved for carefully selected patients. It represents a more intensive approach taken after prior complement-directed therapy has been insufficient. The complete eligibility criteria, full sequence, and all dosing details are available in the full protocol.
Primary treatment goal: Increase in haemoglobin levels, including achievement of transfusion independence.