Specific therapy for transthyretin cardiac amyloidosis: A systematic literature review and evidence‐based recommendations
Journal of the American Heart Association Oct 16, 2020
Marques N, Azevedo O, Almeida AR, et al. - Researchers analyzed relevant randomized controlled trials (RCTs), non‐RCTs, or observational studies to evaluate specific therapies for transthyretin cardiac amyloidosis (CA). They followed PRISMA (Preferred Reporting Items for Systematic Reviews and Meta‐Analyses) guidelines and performed a systematic review of the literature. The included studies involved adult patients with variant/wild‐type transthyretin‐CA and documented cardiovascular outcomes. Per findings, a significant improvement in all‐cause mortality and cardiovascular hospitalizations as well as decrease in worsening of 6‐minute walk test, Kansas City Cardiomyopathy Questionnaire—Overall Summary score, and N‐terminal pro‐B‐type natriuretic peptide were achieved with tafamidis in cases with variant/wild‐type transthyretin‐CA. In a subgroup analysis of patients having variant transthyretin‐CA, promising outcomes were afforded by patisiran. Conflicting results on cardiac imaging parameters were demonstrated by inotersen. Overall, the use of tafamidis in wild‐type and variant transthyretin‐CA is supported by the findings. New treatment targets including transthyretin gene silencers are currently under inquiry.
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