Patients with transthyretin amyloid cardiomyopathy (ATTR-CM) are faced with different possibilities for treating their symptoms and underlying disease. However, treatment options may vary depending on the severity of the disease, the cost of the treatment, and other factors.
Transthyretin amyloid cardiomyopathy (ATTR-CM) is a rare, progressive disease that primarily affects the heart, although it can affect different organs of the body. It is caused by the misfolding of transthyretin proteins that aggregate in the myocardial tissue. If left untreated, ATTR-CM can progress rapidly and cause death. Different treatment options are available and these are reviewed in detail in an analysis published in the Journal of Clinical Medicine.
ATTR-CM is typically characterized by restrictive ventricular filling and reduced stroke volume, which often manifests with symptoms of heart failure. As such, loop diuretics are an option for relieving cardiac congestion and edema in the lungs. Amiodarone is an option for patients with atrial fibrillation or flutter. Anticoagulants are also important for patients with atrial fibrillation to prevent thrombotic events.
While tafamidis is the only approved drug for ATTR-CM at the moment, other pharmacologic agents may be considered for off-label use. Tolcapone and diflunisal have been shown to stabilize the TTR tetramer and improve the prognosis of the disease. However, tolcapone typically needs to be taken multiple times throughout the day, and diflunisal causes typical NSAID side effects, such as cardiovascular and gastrointestinal problems.
Live transplantation is an effective treatment strategy for many patients, as TTR production occurs primarily in the liver. Targeting the gene responsible for TTR production has been a potential treatment strategy in some studies. Certain drugs, such as patisiran, inotersen, and revusiran may be potential treatment options that suppress the TTR production in the liver.
Despite the availability of treatments for ATTR-CM, it is difficult to determine the best treatment option due to a lack of comparative studies and high costs. In addition, some drugs require a high frequency of dosing. As an orphan disease, ATTR-CM affects a small subset of the population, which can make it difficult to generalize the effects of the available treatments. Healthcare providers with ATTR-CM patients should approach treatment options with a sound cost-benefit analysis to determine the best treatment option for their patients.
Tschöpe, C., & Elsanhoury, A. (2022). Treatment of Transthyretin Amyloid Cardiomyopathy: The Current Options, the Future, and the Challenges. J Clin Med, 11(8). https://doi.org/10.3390/jcm11082148