ATTR-CM Patients Prioritize Longevity and Enjoyable Activities Over Basic Self-Care

A multinational study published in Cardiology and Therapy reveals that individuals living with transthyretin amyloid cardiomyopathy (ATTR-CM) prioritize treatments that extend their lives and allow them to continue engaging in meaningful activities, often valuing these outcomes over basic self-care routines. The research, conducted through a patient advisory board and pre-advisory board survey, provides detailed insights into the preferences of patients regarding the attributes of current and emerging therapies for this progressive heart condition. By exploring these perspectives, the study aims to inform shared decision-making processes between patients and healthcare providers as the treatment landscape for ATTR-CM continues to evolve.

ATTR-CM is a serious and progressive disease characterized by the accumulation of misfolded transthyretin amyloid proteins in the myocardium, which frequently leads to heart failure. The condition exists in two subtypes: wild-type, which is more common and typically affects individuals older than 60 years, and variant ATTR-CM, which involves inherited genetic mutations and is associated with higher all-cause mortality. Patients often present with symptoms such as congestive heart failure, atrial fibrillation, and heart conduction disturbances, which limit daily activities and functioning. The study sought to understand which features of treatments are most important to patients, given the lack of in-depth evaluation of their experiences and treatment priorities.

The study involved 13 adults living with ATTR-CM from the United States, Canada, Switzerland, the United Kingdom, and Australia. Participants were recruited through local amyloid patient advocacy groups to ensure diversity in ATTR-CM subtype, nationality, gender, and race. The mean age of the participants was 73 years, with 77% being men and 85% being retired. Nearly two-thirds of the participants had wild-type ATTR-CM, while 38% had the variant form. All participants were receiving therapy for ATTR-CM, and the median time since diagnosis was four years. This sample provided a foundation for qualitative exploration of patient preferences through a combination of survey and advisory board discussions.

Before the advisory board, participants completed an online survey that included benefit-risk scaling questions to gauge the importance of treatment side effects, benefits, and modes of administration. The survey results informed the development of the advisory board discussion guide, which was used to facilitate in-depth conversations about treatment preferences. The advisory board took place virtually in November 2024, with participants split into breakout groups to discuss their priorities. The discussions focused on understanding the drivers of treatment choices, including side effects, benefits, mode of administration, and experiences with diagnosis and monitoring.

Regarding treatment side effects, participants identified tiredness and lack of energy as the most bothersome, selecting this as the most bothersome side effect 65.4% of the time it appeared in the survey. In contrast, injection site pain was considered the least bothersome, with many participants explaining that it had become routine and did not significantly impact their daily lives. The advisory board discussions highlighted that tiredness affected how participants enjoyed their lives and experienced activities they valued. Participants noted that distinguishing between tiredness associated with treatment and that associated with ATTR-CM itself was challenging, and that their fatigue had worsened as their disease progressed.

In terms of treatment benefits, lowering the risk of death was the most important attribute, selected as such 60% of the time it was shown in the survey. Participants emphasized the importance of longevity and being able to engage in the activities they enjoyed, such as spending time with family and participating in hobbies. Being able to easily take a bath or shower was considered the least important benefit, with many participants indicating that they had found ways to manage daily tasks with the help of care partners. The study revealed that patients valued quality of life and the ability to live fully, prioritizing these over basic functioning and self-care routines.

With respect to mode of administration, once-daily oral administration was the most preferred, with participants citing convenience, independence, and the reduced need for travel to treatment centers. This preference was consistent across both the survey and advisory board discussions. Infusions were the least preferred mode of administration, with participants citing the time involved, the need for premedication, and the disruptions caused by steroid treatments. The study also noted significant heterogeneity in preferences, with some participants preferring injections over infusions due to the shorter time required and less travel. Individual-level variability was observed for various side effects and treatment benefits, driven by patients’ circumstances and previous experiences.

The study also included a confirmatory survey with 169 patients from Australia, the UK, Spain, Belgium, Canada, and the US, which broadly supported the findings from the advisory board. The most bothersome side effect, tiredness and lack of energy, and the least bothersome, injection pain, were consistent across both samples. Lowering the risk of death remained the most important treatment benefit, while being able to easily take a bath or shower was among the least important. The confirmatory survey reinforced the importance of shared decision-making, highlighting the need for care teams to consider individual patient preferences and goals when selecting treatments.

Limitations of the study include the small sample size of the advisory board and the potential for selection bias, as participants were recruited through advocacy groups. The sample was highly educated, with three-quarters having a university degree or higher, which may not be representative of the broader ATTR-CM population. Additionally, the study did not evaluate preferences associated with heart failure of other origins or other health conditions. Despite these limitations, the confirmatory survey demonstrated that the key findings were consistent in a larger and more diverse sample, supporting the generalizability of the results.

As the ATTR-CM treatment landscape expands to include therapies with distinct attributes, the study underscores the importance of shared decision-making between patients and care teams. Factors such as caregiver support, distance from treatment centers, and daily pill burden may influence treatment preferences, and these should be considered in individualized treatment decisions. The findings highlight the need for nuanced discussions that incorporate patient priorities and goals, ensuring that treatment choices align with what matters most to individuals living with ATTR-CM.

The study identified specific therapeutic agents currently available or under investigation for ATTR-CM, noting that no head-to-head trials have yet been conducted to compare them directly. Existing disease-modifying therapies include transthyretin silencer agents, which suppress the synthesis of transthyretin protein, and transthyretin stabilizers, which bind to the protein to prevent the formation of amyloid fibrils. Among the silencer agents, patisiran is administered intravenously every three weeks, while vutrisiran is given as a subcutaneous injection in a clinic every three months. Both are also indicated for polyneuropathy associated with variant ATTR. The antisense oligonucleotide eplontersen, another transthyretin knockdown agent, is administered subcutaneously and is currently under investigation for use in ATTR-CM. Oral stabilizers include tafamidis, taken as one pill once daily, and acoramidis, taken as two pills twice daily. Diflunisal, a nonsteroidal anti-inflammatory agent, is used off-label to stabilize transthyretin. These distinct administration routes and dosing schedules create a complex landscape where individualized treatment decisions are critical, as the lack of comparative efficacy data means patient preferences become a significant factor in selecting the most suitable therapy.

Participants in the advisory board provided detailed insights into how specific side effects impacted their daily lives, particularly regarding visual disturbances. Trouble seeing in darker settings was generally considered less bothersome than other side effects, but perspectives varied significantly based on individual circumstances. Some participants had adapted to this side effect or relied on support from care partners to manage it, whereas for others, particularly those living alone without a care partner, it posed a significant challenge. This variability underscores the importance of considering individual living situations and support systems when evaluating treatment tolerability. Similarly, participants noted that while injection pain was ranked as the least bothersome side effect on average, one participant with a history of numerous injections for both ATTR-CM and a previous organ transplant found injections to be more worrisome than most other participants. This individual experience highlights how prior medical history and personal tolerance levels can diverge from group averages, reinforcing the need for personalized discussions rather than relying solely on population-level preference data.

The advisory board discussions also revealed that participants had developed various strategies to monitor their disease progression and manage their health. Some individuals prioritized keeping detailed records of their health status, using spreadsheets to track test results and clinical queries, reflecting a highly engaged approach to managing their condition. Others found physical self-check-ins to be more useful, such as running on a treadmill or monitoring their health during normal activities. These diverse monitoring strategies indicate that patients are actively involved in understanding their disease trajectory, which may influence their preferences for treatment attributes that allow for consistent self-management. The ability to maintain these routines without excessive disruption from treatment logistics, such as travel for infusions or complex dosing schedules, was a recurring theme in the discussions about quality of life and independence.

While the study focused on patient preferences, it also acknowledged that factors not explored in the research, such as treatment availability, ease of access, cost, and reimbursement pathways, are likely drivers of treatment decisions in the real world. The authors noted that although no theoretical framework exists to measure the alignment of preferences and adherence, evidence suggests that treatment adherence correlates with patient preferences in populations living with chronic disease. Real-world data indicates that patients with ATTR-CM are generally adherent to and persist with treatment regimens for cardiomyopathy. Ensuring that treatment decisions reflect the outcomes and modes of administration that patients value may help support adherence and ultimately improve clinical outcomes. This connection between preference and adherence suggests that shared decision-making conversations are not only about respecting patient autonomy but also about optimizing the likelihood that patients will continue their prescribed therapies effectively.

The findings from this study align with broader trends observed in preference research for other forms of heart failure. Previous studies on heart failure with reduced ejection fraction found that patients valued quality of life and longevity, prioritizing these features over tolerability and convenience. Similarly, a scoping review of preference studies in heart failure found that mortality risk was consistently the most important attribute, while symptom improvement and hospitalization risk were also highly ranked. These similarities suggest that the preferences of people with ATTR-CM may be similar to those of people with heart failure of any origin. However, the specific context of ATTR-CM, with its unique treatment options and disease progression, necessitates dedicated research to understand the nuances of patient priorities in this specific population. The study’s findings contribute to this body of evidence by providing detailed insights into the relative importance of specific treatment attributes for ATTR-CM patients.

Subject of Research: Patient preferences for treatment attributes in transthyretin amyloid cardiomyopathy

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

Article References: Kumar, N., Coulter, J., Haddad-Angulo, A., Baig, L., Chung, H., Chandrasekar, S. M., Vass, C., Galinsky, J., Carr, A. M., McIntosh, K., & Hauber, B. (2026). Patient Perspectives on Transthyretin Amyloid Cardiomyopathy (ATTR-CM) Treatment Attributes. Cardiology and Therapy, 15(2), 231-253. https://doi.org/10.1007/s40119-026-00452-w

Image Credits: AI Generated

DOI: 10.1007/s40119-026-00452-w

Keywords: ATTR-CM, patient preferences, cardiology, treatment attributes, shared decision-making, heart failure, transthyretin, advisory board, quality of life, mode of administration, Patient, Perspectives