Sutimlimab improves quality of life in patients with cold agglutinin disease: results of patient-reported outcomes from the CARDINAL study.

Patients with cold agglutinin disease (CAD) experience fatigue and poor quality of life. However, previous CAD-related studies have not explored patient-reported outcomes such as the Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue. Sutimlimab, a C1s complement inhibitor, has been shown to halt haemolysis in CAD. Here, we present 26-weeks' patient-reported data from CARDINAL Part A (ClinicalTrials.gov, NCT03347396), which assessed efficacy and safety of sutimlimab in patients with CAD and recent history of transfusion. Aside from measuring changes in haemolytic markers, FACIT-Fatigue was measured at the treatment assessment timepoint (TAT; average of weeks 23, 25, and 26). Exploratory endpoints included the change in EuroQol 5-dimension 5-level questionnaire (EQ-5D-5L) and the 12-Item Short Form Health Survey (SF-12) at TAT, and Patient Global Impression of Change (PGIC), and Patient Global Impression of (fatigue) Severity (PGIS) at week 26. Mean (range) FACIT-Fatigue scores increased from 32.5 (14.0-47.0) at baseline (a score indicative of severe fatigue) to 44.3 (28.0-51.0) at TAT. Considerable improvements were reported for EQ-5D-5L at TAT, SF-12 scores at TAT, and PGIC and PGIS scores at week 26. Sutimlimab treatment resulted in sustained improvements in symptoms of fatigue and overall quality of life in patients with CAD. NCT03347396. Registered 20 November, 2017.

Occurrence, thromboembolic risk, and mortality in Danish patients with cold agglutinin disease.

Cold agglutinin disease (CAD) is a rare form of autoimmune hemolytic anemia with limited epidemiological and clinical data. We used the Danish National Patient Registries to examine CAD occurrence and risk of thromboembolic events (TEs) and mortality in CAD patients compared with a matched cohort from the general population in Denmark. We identified 72 patients diagnosed with CAD and 720 matched controls between 1999 and 2013. For 2013, the most recent year of study, crude incidence of CAD was 0.18 per 100 000 inhabitants per year and prevalence was 1.26 per 100 000 inhabitants. Risk of TEs was higher in the CAD patient cohort than in the comparison cohort at 1 year (7.2% of CAD patients had TEs vs 1.9% of comparisons), 3 years (9.0% vs 5.3%), and 5 years (11.5% vs 7.8%) after the index date. The median survival was 8.5 years. CAD patients had increased mortality compared with the general population cohort (adjusted hazard ratio [aHR], 1.84; 95% confidence interval [CI], 1.10-3.06; P = .020), with the highest mortality observed during the first 5 years after diagnosis (aHR, 2.27; 95% CI, 1.32-3.89; P = .003). Mortality rates 1 and 5 years after diagnosis were 17% and 39% in the CAD group vs 3% and 18% in the comparison cohort, respectively. CAD is a rare illness characterized by increased risk of TEs and mortality.