Heart failure & cardiomyopathies
Impact of Vutrisiran on Exploratory Cardiac Parameters in Hereditary Transthyretin-Mediated Amyloidosis with Polyneuropathy
Authors' conclusion
Vutrisiran demonstrated evidence of potential benefit on cardiac manifestations in patients with ATTRv amyloidosis with polyneuropathy, with an acceptable safety profile.
Study context
- Population
- Patients with hereditary transthyretin (ATTRv) amyloidosis with polyneuropathyModified intent-to-treat (mITT)
- Participants
- 122 patients
- Comparison
- Vutrisiran vs external placebo; patisiran reference group
- Primary endpoint
- Exploratory cardiac endpoints: change from baseline in NT-proBNP and echocardiographic parameters vs external placebo
- Follow-up
- Month 18
Methodological detailsPhase 3, open-label, external placebo comparator (APOLLO), prespecified cardiac subpopulation
- Phase 3
- Open-label
- External placebo comparator (APOLLO)
- Prespecified cardiac subpopulation
Primary result
As reported in the abstract
NT-proBNP change from baseline
Adjusted geometric mean fold change ratio
0.480
95% CI 0.383–0.600P = 9.606 × 10-10
mITT
Adjusted geometric mean fold change ratio
0.491
95% CI 0.337–0.716P = 0.0004
cardiac subpopulation
Further findings
Echocardiographic parameters vs external placebo (mITT and cardiac subpopulation)
- Benefits or trends towards benefit in echocardiographic parameters vs external placebo in both populations
99mTc scintigraphy vs baseline (vutrisiran-treated cohort at select sites)
- Reduced normalized LV total uptake
- 32/47 (68.1%)
- Reduced heart-to-contralateral lung ratio
- 31/48 (64.6%)
- Perugini grade reduced or unchanged vs baseline (evaluable patients)
- 55/57 (96.5%)
- Compared with baseline
Safety
Cardiac adverse events
- No increase in cardiac AEs with vutrisiran vs external placebo
Original publication
Impact of Vutrisiran on Exploratory Cardiac Parameters in Hereditary Transthyretin-Mediated Amyloidosis with Polyneuropathy. Eur J Heart Fail. Published 6 February 2024. doi:10.
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