Real-world treatment patterns and preferences in patients with transthyretin amyloid cardiomyopathy from Germany and Spain
R Knapp, L Michel, J Garratt-Wheeldon, M S Ares Gomez, J Wright, A Braid, T Evers, J Horswill, F Dominguez Rodriguez, M L Pena PenaAbstract
Background/Introduction
Early treatment is key to stabilise amyloid deposition in transthyretin amyloid cardiomyopathy (ATTR-CM).
Purpose
This study describes the real-world use of disease modifying therapy (DMT) in patients with ATTR-CM.
Methods
We descriptively analysed treatment patterns and preferences using data from the Adelphi Real World ATTR Disease Specific Programme, a real-world, cross-sectional survey of cardiologists and patients with ATTR-CM conducted in Germany and Spain between September 2024 and January 2025.
Results
Among 120 patients included in each country, 93% (Germany) and 56% (Spain) were undergoing treatment at the time of survey completion. Three patients had discontinued; 24% were never treated (Germany: 5%, Spain: 43%), mostly due to ineligibility. In patients receiving DMT or symptomatic treatment, tafamidis was prescribed most (96%). Delaying disease progression was the most common driver of treatment selection (97%). Patient preferences around hypothetical treatments are summarised in Figure 1.
Providers reported feeling very satisfied with the effects of DMT in 66% of cases (Germany: 78%; Spain: 48%). Poor outcomes related to disease progression (35%), quality of life (30%), and symptom management (30%) were the most common reasons for dissatisfaction with DMT in both countries.
Conclusions
Treatment varied in Germany and Spain, with delays in disease progression driving DMT choice. More patients felt completely or somewhat comfortable with oral medications as compared with regular injections or intravenous infusions, reinforcing the value of oral therapeutics.For image description, please refer to the figure legend and surrounding text.