Summary

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• This summary is based on the review of eight systematic review(s)/meta-analysis(es). ^[1-8]
• Patisiran demonstrated significant improvements in neuropathy impairment scores (mNIS + 7) with a standardized mean difference of -0.18 (95% CI: -0.32 to -0.03, p=0.018) and in quality of life (Norfolk Quality of Life-Diabetic Neuropathy) with a mean difference of -21.10 points (95% CI: -27.20 to -15.00; p<0.001), indicating superior outcomes over placebo.
• Vutrisiran consistently showed significant improvements in neuropathy and quality of life, with indirect comparisons indicating its superiority over Tafamidis, which reduced the progression of peripheral neuropathy as evidenced by a mean difference of -3.21 points (95% CI: -5.63 to -0.79; p=0.009) in the Neuropathy Impairment Score (NIS).
• Inotersen reduced the progression of peripheral neuropathy with a mean difference of -19.73 points (95% CI: -26.50 to -12.96; p<0.001) in modified NIS plus 7 nerve tests but was associated with higher rates of severe adverse events and increased mortality compared to placebo.
• Patisiran was associated with 413 adverse events (AEs), affecting 84.8% of patients, predominantly mild to moderate, and showed a reduced dropout rate due to AEs compared to placebo (RR: 0.33; 95% CI: 0.13 to 0.82; p=0.017), with no deaths attributed to its use.
• Inotersen exhibited a higher rate of severe AEs and increased mortality rate compared to placebo, with a relative risk of 5.94 (95% CI: 0.33 to 105.60; p=0.22), indicating potential safety concerns.
• Patisiran demonstrated effectiveness in reducing disability and neuropathy in patients with variant transthyretin amyloidosis, with significant improvements in quality of life measures, while Tafamidis showed significant improvements in all-cause mortality and cardiovascular hospitalizations in both variant and wild-type transthyretin cardiac amyloidosis. Inotersen was effective in reducing neuropathy progression but was associated with higher rates of severe adverse events and increased mortality.