What are the treatment attributes preferred by UK patients with transthyretin amyloid cardiomyopathy?

Background

Prior to 2024 there were no disease-modifying therapies available on the NHS in the UK for patients with either wild-type or hereditary transthyretin amyloid cardiomyopathy (ATTR-CM). Since then, multiple treatment options have been reimbursed, each differing in formulation, characteristics, and route of administration.1,2,3 As the landscape of available therapies expands, understanding patient preferences has become increasingly important to ensure that care is truly patient-centric.

Methods

An international patient advisory board was convened in November 2024 with English-speaking patients (n=13), including four participants from the UK. Before the meeting, patients completed an online survey featuring three object-case, best-worst scaling (BWS) exercises covering 11 hypothetical treatment benefits, 8 side effects, and 5 modes of administration. Higher scores represented more important, burdensome and bothersome attributes. Following the advisory board, a confirmatory online survey was administered to patients in the UK, recruited through Cardiomyopathy UK and Amyloidosis UK, to confirm the findings from the advisory board.

Results

36 patients completed the online survey: median age, 73.5; 67% were male; 69% had wild­type ATTR­CM; 41% had a university degree. In the week prior, 44% of patients had experienced mild heart failure (HF) symptoms, 36% experienced moderate HF symptoms and 8% experienced severe HF symptoms. 94% of patients were taking, or had taken, treatment for their ATTR-CM.

Among the 10 benefits of treatment, the 3 most important were a lesser risk of dying due to HF (59% above average(aa)), reduce heart-related hospital admissions (44% aa) and reduced need for a heart transplant or implanted device (26% aa). The least important benefits were related to completing specific tasks of daily living (fig. 1). Among the 8 side effects, the 2 most bothersome were fatigue (41% aa) and dizziness (37% aa); the 2 least bothersome were injection pain (60% below average (ba)) and injection site reactions (56% ba; fig. 2). The group strongly preferred administration as a once daily (OD) pill taken at home (64% ba burden). The least preferred modes were IV infusion (47% aa) and subcutaneous injections (30% aa; fig. 3).

Conclusion

As the ATTR-CM treatment landscape expands to include treatments with distinct attributes, patient preferences should inform care teams in making treatment decisions that prioritise patient goals.

NB: results are reported on an index where 0 is the lowest possible score (e.g., least bothersome) and 200 is the highest possible score (e.g., most bothersome). A score of 100 is considered average; scores above 100 are considered above average, and scores below 100 are considered below average. The degree to which each item is above or below 100 shows how much better or worse the item is performing relative to the average. For example, an item with a score of 120 is 20% better than average.Figure 1 & Figure 2For image description, please refer to the figure legend and surrounding text.Figure 3For image description, please refer to the figure legend and surrounding text.