Patients with transthyretin amyloid cardiomyopathy (ATTR-CM) may benefit from RNA interference (RNAi) therapies, which appear to reduce the risk of serious heart complications and may even help some patients live longer, according to a new meta-analysis published recently in Current Problems in Cardiology.
ATTR-CM is a rare but serious condition in which abnormal transthyretin proteins build up in the heart, making it stiff and less able to pump blood. RNAi therapies work by shutting down the liver’s production of transthyretin, limiting the amount of harmful protein circulating in the blood. The transthyretin silencer vutrisiran is currently the only RNAi therapy approved to treat ATTR-CM, though additional therapies are being tested in clinical trials.
This meta-analysis of clinical trials examined how well RNAi therapies work and how safe they are.
“This systematic review and meta-analysis provide compelling evidence that RNAi therapies offer significant benefits in reducing cardiac adverse events in patients with ATTR-CM, with a potential mortality advantage when excluding confounding data,” explained the authors. They continued, “These findings reinforce the importance of RNA-targeting approaches in modifying disease progression and highlight the need for further research to refine treatment protocols, optimize patient selection, and explore combination strategies.”
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In this analysis, researchers pooled results from three major clinical trials that included nearly 1,200 patients. They found that RNAi therapies lowered the chances of heart-related complications by about 28% overall. While the treatment did not show a clear survival benefit when all three trials were included, excluding one trial — ENDEAVOR, which was stopped early due to safety concerns — revealed a meaningful improvement in survival for patients on RNAi therapy.
Safety is a key concern with any new treatment. In this case, patients receiving RNAi therapies were not more likely to experience side effects compared with those taking a placebo. Serious heart-related side effects also occurred at similar rates in both groups.
This finding is important for patients because fewer cardiac complications may mean less time in the hospital, better quality of life and potentially longer survival — especially if doctors can determine which patients will respond best. The research also hints that RNAi might help the body clear out existing protein buildup in the heart, although more long-term studies are needed.
For now, patients and caregivers can be cautiously optimistic. RNAi therapies may offer a new and more targeted way to treat ATTR-CM, especially when other options are limited. Future research will continue to clarify how these treatments fit into care plans and who stands to gain the most.
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