Drugs that target the transthyretin (TTR) protein have life-extending potential for patients with transthyretin amyloid cardiomyopathy (ATTR-CM) and are safe to use, found a recent review published in the journal BMC Cardiovascular Disorders.
To function properly, protein molecules (including TTR) must fold into complex three-dimensional shapes. In ATTR-CM, misfolded TTR proteins build up in the heart, stiffening the walls of the heart. Over time, this weakens the heart muscle and leads to heart failure.
Drugs called TTR stabilizers (like tafamidis and acoramidis) help the TTR protein maintain its correct structure. Another type of drug known as TTR knock-down therapies (such as patisiran, vutrisiran, inotersen and eplontersen) reduce how much TTR protein the body produces.
To examine how safe and effective both types of drugs are for treating ATTR-CM, researchers analyzed data from nine clinical trials with a total of 2,713 patients, of whom 1,160 (59%) received TTR-targeting therapies.
They found that these therapies significantly lowered the risk of early death from any cause by approximately 30% compared to patients who received a placebo. Both stabilizers and knock-down drugs were found to be equally effective in this regard.
Read more about treatment and care for ATTR-CM
In addition to improving survival, these treatments helped preserve heart function. Patients receiving therapy showed better left ventricular longitudinal strain measurements and a reduction in left ventricular mass.
However, the drugs did not significantly reduce hospitalizations due to heart failure or overall hospital visits compared to the placebo.
Importantly, the treatments were also safe. Patients on TTR-specific drugs did not experience more side effects or serious complications than those who received a placebo.
Together, these studies “provide strong evidence supporting the use of TTR-specific therapies to manage ATTR-CM,” the authors said. “These therapies significantly improve all-cause mortality and have demonstrated a favorable safety profile in patients with ATTR-CM.”
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