Heart failure & cardiomyopathiesRandomised controlled trial
Patisiran Treatment in Patients with Transthyretin Cardiac Amyloidosis
- Randomised controlled trial
- phase 3
- double-blind
- hierarchical testing
Authors' conclusion
In this trial, administration of patisiran over a period of 12 months resulted in preserved functional capacity in patients with ATTR cardiac amyloidosis.
Study context
- Population
- Patients with hereditary (variant) or wild-type ATTR cardiac amyloidosis
- Participants
- 360 patients
- Comparison
- Patisiran vs placebo
- Patisirann = 181
- Placebon = 179reference
- Primary endpoint
- Change from baseline in 6-minute walk test distance
- Follow-up
- 12 months
Primary result
Change in 6-minute walk distance · Month 12
Hodges-Lehmann estimate of median difference
14.69 m
95% CI 0.69–28.69
P = 0.02
Patisiran vs Placebo
Hodges-Lehmann estimate of median difference 14.69 m (95% CI 0.69–28.69); P = 0.02
As reported in the abstract
Secondary outcomes
| Outcome | least-squares mean difference (points) (95% CI) | P value | |
|---|---|---|---|
| KCCQ-OS score change (higher = better) · Month 12 · increased with patisiran, declined with placebo · first secondary end point | 3.7 (0.2–7.2) | P = 0.04 | |
| −50510Difference, points (linear scale) |
Composite of all-cause death, CV events and change in 6-minute walk distance · 12 months
- Significant benefits not observed
- second secondary end point
Safety
Infusion-related reactions, arthralgia, muscle spasms
- Occurred more often with patisiran than placebo
Original publication
Patisiran Treatment in Patients with Transthyretin Cardiac Amyloidosis. N Engl J Med. Published 26 October 2023. doi:10.1056/nejmoa2300757
Summarised from the published abstract; every number is checked against it