Summary

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• Onpattro (patisiran) is indicated for the treatment of polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.
• This summary is based on the review of four systematic review(s)/meta-analysis(es). ^[1-4]
• Survival Estimates in ATTR Subtypes: The 2-year survival rates for patients with ATTR subtypes were reported as 70.4% (95% CI 66.9-73.9) for non-subtyped ATTR, 76.0% (95% CI 73.0-78.9) for wild-type ATTR, and 77.2% (95% CI 74.0-80.4) for hereditary ATTR. Survival rates were higher in patients treated with tafamidis or patisiran compared to the natural disease course (79.9%, 95% CI 74.4-85.3 vs. 72.4%, 95% CI 69.8-74.9, p < 0.05).
• Efficacy in Polyneuropathy: Patisiran significantly reduced disability progression and improved quality of life compared to placebo, while tafamidis showed a slight reduction in peripheral neuropathy progression without significant improvement in quality of life. Diflunisal and inotersen were effective in reducing peripheral neuropathy progression, with inotersen also improving quality of life but associated with a higher dropout rate due to adverse events.
• Comparative Effectiveness: Patisiran showed superior efficacy in reducing disability progression and improving peripheral neuropathy and quality of life compared to placebo. Tafamidis demonstrated some efficacy in reducing peripheral neuropathy, but not in improving overall disability or quality of life.
• RCT Evidence on Safety: No significant differences in mortality or severe adverse events were observed for tafamidis, diflunisal, or patisiran compared to placebo. Patisiran had a lower dropout rate due to adverse events than placebo. Inotersen was associated with a possible increase in mortality, severe adverse events, and a higher dropout rate due to adverse events compared to placebo.
• Literature Review on Safety: No drug demonstrated superiority in safety, and pharmacoeconomic studies are required to determine the most efficient treatment.
• There is no population types or subgroups information available in the reviewed studies.