Patients with cold agglutinin disease (CAD) are twice as likely to die of their disease within the first five years after being diagnosed, when compared with the general population, a study says.
The findings announced by Bioverativ — a pharmaceutical company now part of Sanofi that is focused on developing new therapies for patients with rare blood disorders — were presented for the first time last year at the 23rd Annual Congress of the European Hematology Association (EHA) in Stockholm, Sweden.
CAD is a rare autoimmune disease in which autoantibodies attack and destroy red blood cells at low temperatures. For this reason, patients with CAD are affected by chronic hemolytic anemia — a form of anemia caused by the destruction of red blood cells — which is associated with extreme fatigue, overall poor quality of life, and the occurrence of life-threatening thromboembolic events (TEs or blood clots that block blood circulation), such as stroke or heart attack.
In the retrospective population-based survival study, researchers set out to assess the risk of mortality and the frequency of TEs in a group of Danish CAD patients compared with the general population.
A total of 72 CAD patients and 720 individuals from the general population — who were matched for age, sex, and region of residence — were included in the analysis. Data from the Danish National Patient Registry was used to estimate the prevalence of CAD and TEs in Denmark, and compare the overall survival of CAD patients with individuals from the general population.
Results showed that CAD patients had a risk of mortality 2.27 times higher than the general population during the first five years after receiving their diagnosis. In addition, analysis showed that five years after diagnosis, CAD patients had a 61 percent chance of surviving, while individuals from the general population had an 82 percent chance of survival.
“The results of our survival analysis contribute to the growing body of evidence that indicate that CAD is a more severe disease than previously thought,” Sigbjørn Berentsen, MD, PhD, Consultant Hematologist at Haugesund Hospital in Norway and corresponding author of the study, said in a press release. “The increased mortality from CAD appears to start at disease onset, and the possible effect of earlier treatment in reducing mortality and complications should be explored in future studies.”
Moreover, findings showed that the overall rate of TEs was 55 per cent higher in CAD patients compared with controls.
“These data show that greater understanding is required about the risks associated with cold agglutinin disease, and they reinforce the pressing need for an approved and targeted treatment for this life-threatening condition,” said Jaime Morales, MD, FAAP, an executive director at Bioverativ. “That is why we are conducting Phase 3 trials of sutimlimab, a novel treatment that has been designed to directly and specifically target CAD.”
Sutimlimab (formerly known as BIVV009), is an investigational antibody that blocks the complement C1, one of the molecules that is involved in the destruction of red blood cells in CAD patients. After receiving the designation of Breakthrough Therapy by the U.S. Food and Drug Administration, the efficacy and safety of Sutimlimab is being tested in Phase 3 clinical trials (NCT03347396; NCT03347422, which is still recruiting participants).