Cold agglutinin disease (CAD) causes predominantly extravascular hemolysis and 
anemia via complement activation. Sutimlimab is a novel humanized monoclonal 
antibody directed against classical pathway complement factor C1s. We aimed to 
evaluate the safety and efficacy of long-term maintenance treatment with 
sutimlimab in patients with CAD. Seven CAD patients treated with sutimlimab as 
part of a phase 1B study were transitioned to a named patient program. After a 
loading dose, patients received biweekly (once every 2 weeks) infusions of 
sutimlimab at various doses. When a patient's laboratory data showed signs of 
breakthrough hemolysis, the dose of sutimlimab was increased. Three patients 
started with a dose of 45 mg/kg, another 3 with 60 mg/kg, and 1 with a fixed 
dose of 5.5 g every other week. All CAD patients responded to re-treatment, and 
sutimlimab increased hemoglobin from a median initial level of 7.7 g/dL to a 
median peak of 12.5 g/dL (P = .016). Patients maintained near normal hemoglobin 
levels except for a few breakthrough events that were related to underdosing and 
which resolved after the appropriate dose increase. Four of the patients 
included were eventually treated with a biweekly 5.5 g fixed-dose regimen of 
sutimlimab. None of them had any breakthrough hemolysis. All patients remained 
transfusion free while receiving sutimlimab. There were no treatment-related 
serious adverse events. Overlapping treatment with erythropoietin, rituximab, or 
ibrutinib in individual patients was safe and did not cause untoward drug 
interactions. Long-term maintenance treatment with sutimlimab was safe, 
effectively inhibited hemolysis, and significantly increased hemoglobin levels 
in re-exposed, previously transfusion-dependent CAD patients.