Summary

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• This summary is based on the review of 13 systematic review(s)/meta-analysis(es). ^[1-13]
• Tafamidis significantly reduced cardiovascular mortality (odds ratio (OR) 0.58; 95% confidence interval (CI): [0.41-0.83], P=0.003) and all-cause mortality (OR 0.45; 95% CI: [0.32-0.64], P≤0.00001) in patients with transthyretin amyloid cardiomyopathy (ATTR-CM), with notable effectiveness observed across various subgroups including age, weight, and bioavailable testosterone levels. However, mortality was statistically insignificant in patients with heart transplants and mechanical assist devices.
• The treatment showed stability in echocardiographic findings and cardiac biomarkers during the study, with no significant deterioration in left ventricular ejection fraction (LVEF) in wild-type transthyretin-related (ATTR) patients after tafamidis treatment.
• Significant differences in outcomes were noted between wild-type and hereditary ATTR, with the Val30Met genotype demonstrating the lowest survival rates. The analysis indicated that patients with wild-type ATTR were older and predominantly male compared to those with hereditary ATTR.
• Tafamidis was generally well-tolerated, with primarily mild to moderate adverse events reported, and no significant differences in mortality or severe adverse events between treatment and placebo groups.
• Common adverse events associated with tafamidis included nausea, flushing, and headache; however, no significant safety concerns were noted specifically among different population types or subgroups.
• Inotersen was highlighted as having a potentially higher risk profile, with increased mortality and severe adverse events compared to placebo, resulting in more participants discontinuing treatment due to adverse events.
• The studies indicate a focus on specific population types such as cardiac amyloidosis patients (including those with hypertrophic cardiomyopathy, heart failure with preserved ejection fraction, and elderly patients with aortic stenosis) and TTR-related familial amyloid polyneuropathy (FAP) populations, with relevant subgroup findings showing no significant deterioration in LVEF in wild-type ATTR patients post-tafamidis treatment, and varying survival estimates and mortality among different genotypes in hereditary ATTR; additionally, wild-type ATTR patients were found to be older and predominantly male compared to those with hereditary ATTR.