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.
