Heart failure & cardiomyopathies
Long-Term Impact of Vutrisiran in Patients With TTR Amyloidosis With Cardiomyopathy: 1-Year Efficacy and Safety Outcomes From the HELIOS-B Open-Label Extension
Main finding · First cardiovascular event or ACMResult — go to the resultHazard ratio 0.63 95% CI 0.51–0.79Authors' conclusion
Long-term vutrisiran resulted in clinically meaningful benefits across mortality, cardiovascular events, cardiac biomarkers, functional status, and quality of life in patients with TTR amyloidosis with cardiomyopathy through month 48. Patients initially treated with vutrisiran had better outcomes than those first receiving placebo, highlighting the importance of early diagnosis and treatment in TTR amyloidosis with cardiomyopathy.
Study context
- Population
- Patients with TTR (transthyretin) amyloidosis with cardiomyopathy who completed HELIOS-Bentered the open-label extension, of 654 participants in HELIOS-B (vutrisiran, n=326, placebo, n=328), 478 completed double-blind treatment
- Participants
- 466 patients
- Comparison
- Continuous vutrisiran group versus the placebo/vutrisiran group
- Continuous vutrisirann = 243
- Placebo/vutrisirann = 223reference
- Outcome
- First cardiovascular event or all-cause mortality (ACM)
- Follow-up
- Through month 48
Main finding
As reported in the abstract
Hazard ratio
0.63
95% CI 0.51–0.79
P = 0.0001
Continuous vutrisiranvs placebo/vutrisiran
hazard ratio 0.63 (95% CI 0.51–0.79); P = 0.0001
Further findings
| Outcome | Estimate | P value | |
|---|---|---|---|
| ACM · through month 48Risk reduced by 37% in the continuous vutrisiran group | hazard ratio 0.63 (95% CI 0.46–0.84) | P = 0.0023 | |
| Improved or stable New York Heart Association class · through month 48 | odds ratio 1.6 (95% CI 1.1–2.2) | P = 0.0084 | |
| 0.512Ratio (log scale) |
| Outcome | Least squares mean difference (95% CI) | P value | |
|---|---|---|---|
| Kansas City Cardiomyopathy Questionnaire-Overall Summary score · through month 48 | 9.0 (5.3–12.6) | P < 0.0001 | |
| −1001020Difference (linear scale) |
| Outcome | Adjusted geometric mean fold change ratio (95% CI) | P value |
|---|---|---|
| NT-proBNP · through month 48 | 0.66 (0.58–0.74) | P < 0.0001 |
| Troponin I · through month 48 | 0.71 (0.64–0.79) | P < 0.0001 |
- Kansas City Cardiomyopathy Questionnaire-Overall Summary score: favouring continuous vutrisiran: preserved health status and quality of life
- Improved or stable New York Heart Association class: more patients with improved or stable class in the continuous vutrisiran group
- NT-proBNP: ratio of continuous vutrisiran to placebo/vutrisiran, favouring continuous vutrisiran: benefits maintained
- Troponin I: ratio of continuous vutrisiran to placebo/vutrisiran, favouring continuous vutrisiran: benefits maintained
Vutrisiran monotherapy population
- Similar benefits were observed in the vutrisiran monotherapy population
Safety
- No new safety signals were observed
Original publication
Long-Term Impact of Vutrisiran in Patients With TTR Amyloidosis With Cardiomyopathy: 1-Year Efficacy and Safety Outcomes From the HELIOS-B Open-Label Extension. Circulation. Published 3 November 2026. doi:10.
Composed by AI from the published abstract and checked against it. How Briefs are made →