Ronald Witteles, MD, is a professor of cardiovascular medicine at Stanford University and co-director of the Stanford Amyloid Center and the Stanford Multidisciplinary Sarcoidosis Program

Ronald Witteles, MD

Future Directions in ATTR-CM

The panel concludes by discussing the future of ATTR-CM care. They highlight continued progress in disease-modifying therapies, with growing interest in determining how best to sequence or potentially combine treatments as evidence evolves. The group notes that as patients are diagnosed earlier, the goals of therapy may increasingly shift from slowing decline to preserving long-term function and quality of life.

They emphasize the importance of expanding awareness and education across specialties so that more patients are recognized before advanced cardiac dysfunction develops. Improving access to therapy remains an essential area of focus, as cost and insurance barriers continue to influence treatment decisions. The panel also anticipates advances in monitoring disease progression, including the use of biomarkers and imaging to better assess response over time.

Ultimately, the experts express optimism that ongoing research, broader recognition, and more refined care pathways will continue to improve outcomes for individuals living with ATTR-CM.

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The panel concludes by discussing the future of ATTR-CM care. They highlight continued progress in disease-modifying therapies, with growing interest in determining how best to sequence or potentially combine treatments as evidence evolves. The group notes that as patients are diagnosed earlier, the goals of therapy may increasingly shift from slowing decline to preserving long-term function and quality of life.
They emphasize the importance of expanding awareness and education across specialties so that more patients are recognized before advanced cardiac dysfunction develops. Improving access to therapy remains an essential area of focus, as cost and insurance barriers continue to influence treatment decisions. The panel also anticipates advances in monitoring disease progression, including the use of biomarkers and imaging to better assess response over time.
Ultimately, the experts express optimism that ongoing research, broader recognition, and more refined care pathways will continue to improve outcomes for individuals living with ATTR-CM.


Patient Engagement and Shared Decision-Making in ATTR-CM

The panel discusses the importance of shared decision-making in the care of patients with ATTR-CM. They emphasize that treatment conversations are often ongoing rather than single moments, as patients and families may need time to understand the diagnosis, the goals of therapy, and what to expect over time. The clinicians highlight the role of clear, honest communication, particularly when addressing uncertainty around disease progression or response to therapy.

The group describes how patient preferences can influence treatment selection, including the choice between oral therapies and periodic injections, the desire to minimize medication burden, and the importance of maintaining independence and daily routines. They also note that caregivers are crucial partners in supporting adherence, appointment planning, and monitoring changes in function or symptoms. Ultimately, the panel agrees that successful care depends on making decisions with patients, ensuring they understand their options, and aligning treatment plans with their values, goals, and quality-of-life priorities.

The panel discusses the importance of shared decision-making in the care of patients with ATTR-CM. They emphasize that treatment conversations are often ongoing rather than single moments, as patients and families may need time to understand the diagnosis, the goals of therapy, and what to expect over time. The clinicians highlight the role of clear, honest communication, particularly when addressing uncertainty around disease progression or response to therapy.
The group describes how patient preferences can influence treatment selection, including the choice between oral therapies and periodic injections, the desire to minimize medication burden, and the importance of maintaining independence and daily routines. They also note that caregivers are crucial partners in supporting adherence, appointment planning, and monitoring changes in function or symptoms. Ultimately, the panel agrees that successful care depends on making decisions with patients, ensuring they understand their options, and aligning treatment plans with their values, goals, and quality-of-life priorities.


Quality of Life and GI Tolerability in ATTR-CM Management

The panel focuses on how quality of life considerations shape ATTR-CM management. Beyond survival and hospitalization outcomes, many patients most value being able to remain mobile and independent—continuing activities such as walking, gardening, or caring for grandchildren. Even subjective concerns like weight loss or appearing “frail” can meaningfully affect how patients feel about their health. Managing symptoms of congestion and reducing the need for frequent diuretic adjustments also plays an important role in daily well-being.

The discussion then highlights recent data from HFSA showing that vutrisiran was associated with fewer gastrointestinal symptoms, such as diarrhea and constipation. The panel found this particularly interesting in wild-type ATTR-CM, where GI involvement is not always recognized. Improvements in GI comfort can support better nutrition, energy, and overall day-to-day function. Across the conversation, the experts emphasize that maintaining function and stability over time is often the clinical outcome patients value most.

The panel focuses on how quality of life considerations shape ATTR-CM management. Beyond survival and hospitalization outcomes, many patients most value being able to remain mobile and independent—continuing activities such as walking, gardening, or caring for grandchildren. Even subjective concerns like weight loss or appearing “frail” can meaningfully affect how patients feel about their health. Managing symptoms of congestion and reducing the need for frequent diuretic adjustments also plays an important role in daily well-being.
The discussion then highlights recent data from HFSA showing that vutrisiran was associated with fewer gastrointestinal symptoms, such as diarrhea and constipation. The panel found this particularly interesting in wild-type ATTR-CM, where GI involvement is not always recognized. Improvements in GI comfort can support better nutrition, energy, and overall day-to-day function. Across the conversation, the experts emphasize that maintaining function and stability over time is often the clinical outcome patients value most.


Clinical Considerations for Monotherapy vs Dual Therapy in ATTR-CM Management

The panel discusses decision-making around monotherapy versus dual therapy in ATTR-CM. They explain that while there is not definitive, statistically significant evidence supporting routine dual therapy for all patients, there is also no evidence of harm when stabilizers and silencers are used together. Because the two therapeutic classes work through different mechanisms, dual therapy is considered reasonable in selected patients, particularly when the goal is to slow progression as much as possible.

The panel emphasizes that treatment decisions should be individualized, guided by disease stage, symptom burden, neurologic involvement, patient priorities, and access considerations. They stress that ATTR-CM is not a uniform disease, and two patients with the same diagnosis may have completely different clinical needs. Ultimately, the group agrees that the only clearly inappropriate choice is providing no disease-modifying therapy at all.

The panel discusses decision-making around monotherapy versus dual therapy in ATTR-CM. They explain that while there is not definitive, statistically significant evidence supporting routine dual therapy for all patients, there is also no evidence of harm when stabilizers and silencers are used together. Because the two therapeutic classes work through different mechanisms, dual therapy is considered reasonable in selected patients, particularly when the goal is to slow progression as much as possible.
The panel emphasizes that treatment decisions should be individualized, guided by disease stage, symptom burden, neurologic involvement, patient priorities, and access considerations. They stress that ATTR-CM is not a uniform disease, and two patients with the same diagnosis may have completely different clinical needs. Ultimately, the group agrees that the only clearly inappropriate choice is providing no disease-modifying therapy at all.


Ronald Witteles, MD

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