Cold agglutinin disease (CAD) exacts a heavy long-term burden on patients and health care systems due to the lack of effective treatments to manage the disease. This was the conclusion of a recent retrospective, observational study published in the Journal of Managed Care + Specialty Pharmacy.

According to the authors of the publication, “these results provide evidence of the unmet medical need to treat and control the disease burden of CAD, particularly in patients with severe anemia, and may prompt further investigation into treatments and management of this rare disease.”

Most (65.6%) patients in the study cohort used corticosteroids to manage the disease. During the full follow-up period, 68.7% and 12.6% of patients with severe and moderate anemia received a blood transfusion, respectively. None of the patients with mild anemia required blood transfusion. The mean number of blood transfusions per patient per year was 3.26 and 5.47 over the full follow-up period.

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At 12 months, the vast majority (97.7%) of patients needed outpatient services, whereas 34.1% and 29.3% required 1 or more hospitalizations or emergency department visits, respectively. These proportions increased to 99%, 52.5%, and 53.9% over the full follow-up.

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Nearly one-half (51%) of patients had moderate/severe anemia at baseline. This proportion increased to 57.7% at the 12-month follow-up and 66.6% over the full follow-up period. Moreover, during the full follow-up period, about one-half of the patients showed elevated bilirubin and lactate dehydrogenase levels.

The study also found that health care resource utilization was greater in patients with severe anemia compared with patients with mild or moderate anemia.

The study enrolled 610 adults with CAD (median age, 72 years, 65.4% female) from the US Optum Electronic Health Record database. The median duration of follow-up was 42.8 months. The study was sponsored by Sanofi.


Pham HP, Wilson A, Adeyemi A, et al. An observational analysis of disease burden in patients with cold agglutinin disease: results from a large US electronic health record database. J Manag Care Spec Pharm. 2022;28(12):1419-1428. doi:10.18553/jmcp.2022.28.12.1419