Summary

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• This summary is based on the review of one randomized controlled trial. ^[1]
• Eplontersen treatment led to a significant reduction in serum transthyretin concentration, with an adjusted mean percentage reduction of -81.7% at week 65, compared to -11.2% for the placebo group. The difference between the groups was -70.4% (95% confidence interval (CI), -75.2% to -65.7%; P < .001).
• Neuropathy impairment, measured by the modified neuropathy impairment score (mNIS)+7 composite score, showed an adjusted mean change of 0.3 for eplontersen from baseline to week 66, compared to 25.1 for placebo, resulting in a difference of -24.8 (95% CI, -31.0 to -18.6; P < .001).
• In terms of quality of life (Norfolk Quality of Life-Diabetic Neuropathy (QOL-DN) Total Score), eplontersen showed an adjusted mean change of -5.5 at week 66, compared to 14.2 for placebo, with a difference of -19.7 (95% CI, -25.6 to -13.8; P < .001).
• Adverse events leading to study drug discontinuation occurred in 6 patients (4%) in the eplontersen group and 2 patients (3%) in the placebo group.
• Two deaths were reported in the eplontersen group due to cardiac arrhythmia and intracerebral hemorrhage, both attributed to disease-related sequelae, while no deaths were reported in the placebo group.
• The study included 168 adults with hereditary transthyretin amyloidosis (ATTRv) polyneuropathy (Coutinho stage 1 or 2), with 144 patients in the eplontersen group (mean age 53.0 years; 69% male) and 60 patients in the placebo group (mean age 59.5 years; 68% male). No significant differences in effectiveness or safety outcomes were reported between different population types or subgroups.