Ask Your Doctor About Your Options for ARRT-CM Treatment
Transthyretin amyloid cardiomyopathy (ATTR-CM) is a progressive disease where abnormal transthyretin proteins build up and deposit in the heart muscle. These deposits make the heart walls stiff, and it becomes more difficult to pump blood effectively. Over time, the condition can lead to heart failure, arrhythmias and other complications. Thankfully there are options for ATTR-CM treatment and symptom management. Keep reading to learn more.
Options for Various Medications
TTR Stabilizers
TTR stabilizers work by binding to the transthyretin protein and preventing it from breaking apart and forming amyloid fibrils. This approach addresses the root cause of the disease rather than just managing symptoms.
Tafamidis, sold under the names Vyndaqel and Vyndamax, was the first FDA-approved drug for ATTR-CM. Clinical trials have shown that tafamidis reduces the risk of death and lowers cardiovascular hospitalizations over a 30-month period. It is generally well tolerated and is considered a first-line treatment for eligible patients.
Diflunisal, a nonsteroidal anti-inflammatory drug, is sometimes prescribed off-label as a stabilizer. While it has shown some ability to slow disease progression, it is not as extensively studied in ATTR-CM as tafamidis or acoramidis, and long-term safety for this specific use is still being evaluated.
TTR Silencers
TTR silencers take a different approach by reducing the body’s production of transthyretin protein at the genetic level. They use small interfering RNA or antisense oligonucleotides to target the liver, where most transthyretin is made.
Patisiran and inotersen are approved for hereditary ATTR-polyneuropathy and are in advanced trials for ATTR-CM. Vutrisiran, which is similar to patisiran but requires fewer injections, was originally approved for polyneuropathy and received an expanded indication for ATTR-CM in March 2025. These therapies can be especially useful when stabilizers are not suitable due to intolerance or insurance barriers. Research suggests they may slow heart muscle thickening and improve exercise capacity.
Emerging Therapies
Several innovative ATTR-CM treatment options are being developed. Monoclonal antibodies designed to target and remove amyloid deposits from the heart could eventually complement stabilizers or silencers. Gene-editing technologies, such as CRISPR-based approaches, aim to permanently shut down production of abnormal transthyretin. These therapies are still in early clinical stages but hold promise for transforming long-term outcomes.
Symptom Management
Even with disease-modifying drugs, many patients need additional ATTR-CM treatment to manage heart failure symptoms and maintain daily functioning.
Managing Heart Failure
Diuretics are often prescribed to reduce fluid retention and ease swelling in the legs or abdomen. They can also relieve shortness of breath caused by fluid buildup in the lungs. However, traditional heart failure medications like ACE inhibitors, angiotensin receptor blockers and beta blockers are not always well tolerated in ATTR-CM due to the unique way the disease affects heart function.
Controlling Arrhythmias
Irregular heart rhythms, such as atrial fibrillation, are common in ATTR-CM. These may require antiarrhythmic drugs, electrical cardioversion or devices like pacemakers and implantable cardioverter-defibrillators. Anticoagulants are often prescribed to reduce the risk of stroke in patients with atrial fibrillation.
Advanced Cardiac Interventions
In advanced cases where medications no longer control symptoms, more invasive options may be considered. Heart transplantation can provide a new start for eligible patients, though donor availability and underlying health can limit candidacy. In hereditary ATTR, a combined liver-heart transplant has been used to address both the source of abnormal transthyretin and the damaged heart, though this approach is less common today. Left ventricular assist devices may also help select patients as a bridge to transplant or as a long-term therapy.
Complementary and Alternative Approaches
Some patients explore adjunctive therapies to support heart health and potentially influence disease progression. Green tea extract, which contains epigallocatechin gallate (EGCG), has been studied in small groups and may help reduce amyloid deposits and improve heart structure. While promising, this approach is not a substitute for medical therapy and should be discussed with a healthcare provider before use.
Tolcapone, a drug with orphan designation for ATTR-CM, acts as a stabilizer but is still in early research phases. Its safety profile and effectiveness remain under review.
Lifestyle and Supportive Care
Lifestyle measures play an important role alongside medical treatment. Reducing dietary sodium helps limit fluid retention and reduces the workload on the heart. Maintaining a healthy weight and staying as physically active as symptoms allow can help preserve function and improve energy levels.
Regular follow-up with a cardiologist experienced in amyloidosis is essential. This ensures that medication regimens are adjusted as needed, emerging symptoms are addressed quickly and disease progression is tracked using imaging and laboratory tests.
For hereditary ATTR-CM, genetic counseling is highly recommended. Counselors can explain the inheritance pattern, arrange genetic testing for family members, and discuss reproductive options such as preimplantation genetic diagnosis to prevent passing on the mutation.
The information on this website is for general educational purposes only and is not a substitute for professional medical advice. Always consult your doctor or qualified healthcare provider before making changes to your health, diet or treatment plan.