Vutrisiran treatment reduced outpatient worsening heart failure (HF) in patients with transthyretin amyloid cardiomyopathy (ATTR-CM) who participated in the HELIOS-B trial, according to a paper published in the Journal of the American College of Cardiology.
The results showed that patients on vutrisiran had a reduced risk of death and cardiovascular events and experienced a significant increase in functional capacity and quality of life.
What is ATTR-CM?
Transthyretin amyloidosis cardiomyopathy (ATTR-CM) is a rare progressive disease of the heart muscle that leads to congestive heart failure. It occurs when the transthyretin protein produced by the liver is unstable. Symptoms include fatigue; shortness of breath; irregular heart rate or palpitations; swelling of the legs, ankles and stomach; brain fog; wheezing; and dizziness. It often goes underdiagnosed because of a lack of awareness and knowledge of the disease. There is currently no cure for ATTR-CM.
“The benefits of vutrisiran on outpatient worsening HF are consistent with its impact on all-cause mortality, recurrent [cardiovascular] events, functional capacity, health status, and quality of life,” study authors said.
Read more about ATTR-CM therapies
Vutrisiran is an experimental drug for ATTR-CM that blocks the production of transthyretin in the liver, preventing its harmful deposition in heart muscle. The HELIOS-B trial assessed the efficacy of vutrisiran by administering it to more than 300 patients with ATTR-CM and comparing results with a similar population receiving a placebo.
The study aimed to assess the effect of vutrisiran therapy in a subgroup of patients with ATTR-CM. It analyzed the effects of the drugs in patients with outpatient worsening HF, defined as oral diuretic initiation or intensification.
Out of more than 600 patients participating in the trial, 321 patients had outpatient worsening HF. Researchers observed that it correlated with an increased risk of all-cause mortality and the occurrence of cardiovascular events, as close to 50% of patients in this subgroup experienced cardiovascular events or died.
About half of the population received vutrisiran in the HELIOS-B trial, while the other half received a placebo. Patients who received vutrisiran were less likely to experience outpatient worsening HF than those on placebo, independent of the concomitant use of tafamidis.
“While the prognostic value of outpatient worsening HF is established in patients with HF,8-10 it is only in this contemporary ATTR-CM patient population that a similar effect can also be found, as patients in older ATTR-CM studies had more severe cardiomyopathy making assessment of diuretic dose increases challenging,” the authors said.
