Patient Perspectives on Transthyretin Amyloid Cardiomyopathy (ATTR-CM) Treatment Attributes

Transthyretin amyloid cardiomyopathy (ATTR-CM), a progressive disease characterized by the accumulation of misfolded transthyretin amyloid proteins in the myocardium, often leads to heart failure. The experiences and treatment priorities of individuals living with ATTR-CM have not been evaluated in depth. This multinational patient advisory board qualitatively explored patients’ experiences with ATTR-CM and their preferences for the attributes of current and emerging treatments for heart failure caused by ATTR-CM. In a pre-advisory board survey, participants answered benefit–risk scaling questions to gauge the importance of treatment side effects, treatment benefits, and modes of administration. The results of the survey were then discussed during the advisory board. Of the 13 participants who completed the survey (two in Australia, two in Canada, one in Switzerland, four in the United Kingdom, and four in the United States), 11 participated in the advisory board discussion and two in follow-up discussions. The survey results and advisory board discussion revealed tiredness and fatigue to be the most bothersome and injection site pain to be the least bothersome of the side effects evaluated. Lowering the risk of death was the most important and being able to easily take a bath or a shower was the least important of the treatment benefits evaluated. On average, once-daily oral administration was the most preferred mode of administration tested, over twice-daily oral administration, subcutaneous injection, and infusion, although participants’ preferences were heterogeneous. Although the length of life was important, quality of life was also important to participants: they especially valued spending time doing the activities they enjoyed and prioritized these activities over basic functioning and self-care routines. Individuals living with ATTR-CM have heterogeneous preferences. As the ATTR-CM treatment landscape expands to include treatments with distinct attributes, patients and care teams should participate in shared decision-making to prioritize patients’ preferences and goals. Researchers conducted a multinational study in November 2024 to understand what patients with the heart condition called cardiac amyloidosis related to transthyretin (TTR) thought were the most important from a set of potential medication features. Thirteen patients completed a survey and later joined a discussion about their priorities. Participants answered questions that allowed the researchers to calculate the importance of different potential medication benefits, side effects, and ways they take their medicine. The options for how to take medicine were as a pill once daily; as pills twice daily; as an injection in the clinic every 3 months; as an injection every month (self-administered or in the clinic); and as an infusion at the hospital every 3 months. Individual patients had different priorities when thinking about the features of a medicine for cardiac amyloidosis. When thinking about benefits, patients in general were most concerned with a medicine’s ability to increase their chance of living longer. Their least concern was making it easier to bathe or take a shower. When considering side effects, tiredness was the most concerning, whereas pain from an injection was the least concern. Patients preferred to take their medicine as a pill over a monthly injection or a quarterly infusion. Patients also value a treatment that fits easily into daily life, allowing them to continue doing the things they enjoy most. As more treatments for cardiac amyloidosis become available, patients and care teams should work together to make shared treatment decisions and incorporate patient preferences wherever possible.