New Data Highlight Unmet Needs Over Time of Primary CAD Patients

The burden of disease among people with primary cold agglutinin disease (CAD) remains high multiple years after diagnosis, illustrating the unmet needs of these patients, new data from a large U.S. Electronic Health Record (EHR) database shows.

The data were shared in a presentation, titled “Longitudinal Analysis of Anemia Severity, Treatment and Healthcare Resource Utilization Among Patients with Primary Cold Agglutinin Disease in a Large US Database,” given at the recent annual meeting of the American Society of Hematology.

There is not much published data on the impact of CAD, either on individuals or on society as a whole. However, such data is important for guiding decisions related to researching the acquired autoimmune disorder, providing effective care, and enacting helpful policies.

Now, researchers from Sanofi and other institutions conducted an analysis of insurance data, from the Optum EHR database, in order to better understand the impact of CAD.

By assessing diagnostic codes, the researchers identified 610 adults with primary CAD who were included in the database between 2007 and 2019. These patients’ mean age was 67.9 years, and 65.4% were female.

Nearly all of the patients (90%) had a follow-up time of at least one year. The mean follow-up time was 48.1 months, or just over four years.

At baseline — up to six months prior to the first documented instance of CAD in the database — more than a quarter (26.9%) of the patients had severe anemia, defined as hemoglobin levels below 8 g/dL. A further 24.1% had moderate anemia at baseline, with hemoglobin levels between 8-10 g/dL.

For context, hemoglobin levels of 12 g/dL or higher were considered normal (i.e., no anemia), which was reported in 26.9% of CAD patients at baseline.

Severe and moderate anemia were similarly common in the six months immediately following baseline. At subsequent timepoints, the proportion of patients with severe anemia decreased, but it was still relatively high — at all timepoints assessed, more than one in 10 patients had severe anemia.

In the U.S., no approved therapies exist to treat CAD, leading to high unmet needs among patients. However, some medications — such as corticosteroids or antibody-based therapies that reduce inflammation — are used off-label.

Indeed, at six months after baseline, CAD patients in the database had received a median of four disease-related treatments.

“The median number of CAD-related treatments per patient was high in all CAD patients at 6 months [after baseline] and remained high during the follow-up period,” the researchers wrote.

The frequency of blood transfusions, a treatment option usually used only in severe cases, was similarly high, with an average of 2.94 transfusions in the six months post-baseline. That average remained high at subsequent timepoints.

CAD patients also had frequent hospital and emergency room visits, especially among those who had more severe anemia. For example, in the 19-to-24 month period post-baseline, the mean number of emergency room visits was nearly four for patients with severe anemia; in the entire group, it was 0.85 visits during this period.

“The results highlight the long-term substantial burden of CAD on patients and healthcare systems, which generally increased with higher severity of anemia,” the researchers concluded.

The team said this analysis “illustrates the unmet medical needs in primary CAD.”