Sutimlimab has demonstrated high efficacy in increasing hemoglobin levels and reducing markers of hemolysis in patients with cold agglutinin disease (CAD) in clinical trials. However, data on its real-world use are limited. We evaluated 54 patients treated with Sutimlimab through post-trial access or compassionate use programs in Austria, France, Germany, Italy, Japan, Spain, UK, and Australia. Past medical history, CAD treatments and laboratory parameters were collected at baseline and at weeks 2, 4, 8, 12, 16, and 24, and at months 12, 18, 24, after starting sutimlimab. Response was classified as partial (Hb >10 g/dL), complete (>12 g/dL), or no response. The median age was 66 years (range 45-88) and 31 (57%) patients were females. The direct antiglobulin test (DAT) was positive for C3 alone in 35 patients, for IgG and C3 in 8, and for IgM and C3 in 10. The median CA titer was 1:1,024 (range, 1:4 to >1:25,000). CAD bone marrow infiltrate was observed in 23 patients. Prior CAD treatments (median 3, 1-4) included corticosteroids (31), rituximab (40), and rituximab–bendamustine (12). Pre-Sutimlimab complications included 8 G≥ 3 infections and 12 thrombotic events. The median number of RBC transfusions in the 6 months pre-Sutimlimab was 2 (0–18). At baseline, median Hb was 8.4 g/dL (2.9–10.8), LDH 1.6× ULN (0.8–3.6), unconjugated bilirubin 1.9 mg/dL (0.8–7.49), and reticulocytes 107×10⁹/L (10–340). Sutimlimab was administered as monotherapy (n=42) or in combination with erythropoietin (8), corticosteroids (2), or both (2). The median follow-up was 28 months (6–81). Hb increased over time: +2.0 g/dL at 2 weeks, +2.7 g/dL at 4 weeks, and +3 g/dL at 8 weeks, followed by sustained levels up to 24 months. Overall response rates (PR + CR) were 78%, 75%, 84%, 85%, 86%, 80%, 77%, 84%, and 85% at the respective timepoints. An additional 14% of patients showed an Hb increase at week 2 and 18% at week 4. All but 7 patients became transfusion-independent. Sutimlimab was discontinued in 2 patients due to lack of response, in 1 due to overt Waldenström's macroglobulinemia, and in 7 based on their own decision with maintenance of good CAD control; 13 patients had an infection (G3 in 7, 1 H. influenzae pneumonia), 1 thrombosis (DVT of basilic vein), and 8 hemolytic exacerbations. At the last follow up only one patient had died due to progressive WM with sepsis. Sutimlimab confirmed to be an effective treatment in improving anemia and resolving transfusion dependence in more than 70% patients in the real world.

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2025
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