Key result
Vutrisiran reduces gastrointestinal events by ~42% versus placebo in patients with ATTR-CM.
Why the study?
Does vutrisiran reduce gastrointestinal adverse events compared to placebo in patients with transthyretin amyloidosis with cardiomyopathy?
Population
654 patients with transthyretin amyloidosis (ATTR) with cardiomyopathy (CM)
Comparison
Vutrisiran 25 mg every 12 weeks for up to 36… vs Placebo every 12 weeks for up to 36 months
Design
RCT, double-blind
Follow-up
up to 36 months
Authors
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May reduce GI burden in ATTR-CM; extends RCT evidence for vutrisiran to extracardiac manifestations.
RCT (n=654)
Double-blind
Does vutrisiran reduce gastrointestinal adverse events compared to placebo in patients with transthyretin amyloidosis with cardiomyopathy?
Relative Risk: 0.58
Absolute Event Rate: 23.4% vs 40.6%
p-value: p=< 0.0001
Vutrisiran significantly reduces the burden of extracardiac gastrointestinal manifestations in patients with ATTR-CM compared to placebo.
Urey et al. (2026) conducted an RCT in Transthyretin amyloidosis (ATTR) with cardiomyopathy (CM) (n=654). Vutrisiran vs. Placebo was evaluated on Gastrointestinal events (MedDRA GI disorders system organ class) (rate ratio 0.58, p=< 0.0001). Vutrisiran significantly reduced the rate of gastrointestinal events compared to placebo in patients with ATTR-CM (23.4 vs 40.6 events/100 patient-years; rate ratio 0.58; P<0.0001).
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