Long-term sutimlimab improves quality of life for patients with cold agglutinin disease: CARDINAL 2-year follow-up

Abstract

Cold agglutinin disease (CAD) is a rare form of autoimmune hemolytic anemia with a substantial burden on patient quality of life. CARDINAL (NCT03347396) was a two-part, open-label, single-arm, multicenter Phase 3 study evaluating the C1s inhibitor sutimlimab for treatment of CAD. Part A consisted of the pivotal study phase, with the Part B extension phase assessing long-term safety and durability of response including patient-reported outcomes, the focus of this report. Altogether, 22 patients continued from Part A to Part B, majority female (68.2%) with a median (range) age of 71.5 (55-85) years. Throughout treatment, score improvement on the Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue scale exceeded a pre-defined, group-level clinically important change of ≥5 points versus baseline, with a mean (standard error [SE]) change of 11.7 (3.7) points at Week 135. The 12-Item Short Form Health Survey physical and mental component scores remained above baseline, with Week 123 mean (SE) change exceeding clinically important changes of 3.9 for physical and 2.8 for mental component scores at 4.7 (2.8) and 3.8 (5.7) points, respectively. EuroQol Visual Analogue Scale, scoring patients' self-rated health, also remained above baseline with a change of 17.1 (5.6) points at Week 135. Patient Global Impression of (fatigue) Severity improved versus baseline, corroborating FACIT-Fatigue scores. Patient Global Impression of Change indicated a reduction in perceived disease burden. Data from CARDINAL Part B support sustained alleviation of CAD disease burden following long-term treatment with sutimlimab over 2 years, returning towards baseline upon treatment cessation.