Targeted ATTR-CM therapies reduced all-cause mortality by 31% (RR 0.69) and cardiovascular hospitalizations by 31%, with NNT of 12 at 2 years.
Do targeted ATTR-CM therapies reduce all-cause mortality and cardiovascular hospitalizations in patients with transthyretin amyloid cardiomyopathy?
Targeted therapies for ATTR-CM significantly reduce the risk of all-cause mortality and cardiovascular hospitalizations, with an estimated number needed to treat of 12 at 2 years to prevent one death.
Absolute Event Rate: 0% vs 0%
Abstract Introduction Transthyretin amyloid cardiomyopathy (ATTR-CM) is a progressive disease that leads to heart failure and has a poor prognosis. Recent advancements in targeted therapies, such as transthyretin (TTR) stabilizers and gene silencers, have improved patient outcomes. However, a comprehensive meta-analysis evaluating the overall efficacy of these treatments on clinical outcomes is still lacking. Aim This meta-analysis aims to evaluate the pooled effects of targeted ATTR-CM therapies on all-cause mortality and cardiovascular hospitalizations, providing a quantitative synthesis of current evidence to guide clinical decision-making. Methods A systematic literature search was conducted in PubMed up to August 31, 2024. After screening 635 records, seven randomized and non-randomized controlled studies involving 3,138 patients were included. Data extraction followed the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines. The primary outcome was all-cause mortality, and the secondary outcome was cardiovascular hospitalizations. A random-effects meta-analysis was performed to determine the pooled relative risk (RR) estimates for each outcome. Results Targeted ATTR-CM treatments demonstrated a significant reduction in all-cause mortality by 31% (RR 0.69, 95% CI: 0.58–0.82, I² = 11%, p = 0.35, Figure 1). A sensitivity analysis including only randomized controlled trials confirmed a 28% mortality reduction (RR 0.72, 95% CI: 0.60–0.86, I² = 0%, p = 0.56). Similarly, cardiovascular hospitalizations were reduced by 31% (RR 0.69, 95% CI: 0.53–0.89, I² = 68%, p = 0.01). Applying these estimates to large-scale epidemiological data suggested a number needed to treat (NNT) of 12 at 2 years and 7 at 5 years to prevent one death (Figure 2). Conclusions This meta-analysis provides estimates of reduction in mortality and cardiovascular hospitalizations risk wth targeted ATTR-CM therapies. These findings highlight the importance of early diagnosis and treatment initiation to maximize patient survival. Future research should explore optimal therapeutic strategies, including potential combination approaches, to enhance treatment efficacy and accessibility.Figure 1 Figure 2
Tsampras et al. (Sat,) reported a other. Targeted ATTR-CM therapies reduced all-cause mortality by 31% (RR 0.69) and cardiovascular hospitalizations by 31%, with NNT of 12 at 2 years.