Umeå University study finds drug combination ineffective for cardiac amyloidosis

A large-scale study involving approximately 1,430 patients has found that combining two effective drugs for cardiac amyloidosis, also known as Skellefteå disease, does not reduce the risk of death from cardiovascular disease or the recurrence of worsening cardiovascular events.

The research, conducted by Umeå University and the Skellefteå Research Unit, examined the drug eplontersen, which works by reducing the production of the protein transthyretin. While eplontersen showed positive effects in patients not receiving other specific amyloidosis treatments, it failed to show a significant benefit when combined with the drug tafamidis regarding mortality or cardiovascular events.

Researchers expressed surprise at these results, as previous studies suggested that combining these medications might improve mortality rates. However, the combination did show some positive outcomes, including better preserved quality of life and improved performance in walking tests compared to the control group. Professor Emeritus Kurt Boman noted that the results indicate ongoing questions regarding the mechanisms and optimal treatment methods for the disease.