Heart failure & cardiomyopathies
Effect of Aficamten on Health Status Outcomes in Obstructive Hypertrophic Cardiomyopathy: Results From SEQUOIA-HCM
Authors' conclusion
In patients with symptomatic oHCM, treatment with aficamten resulted in markedly improved health status, including significant improvement in chest pain-related health status, than placebo.
Study context
- Population
- Symptomatic adults with obstructive hypertrophic cardiomyopathy (oHCM)
- Participants
- 282 adults
- Comparison
- Aficamten vs placebo
- Aficamtenn = 142
- Placebon = 140reference
- Primary endpoint
- Change in KCCQ-Overall Summary Score and SAQ7-Summary Score
- Follow-up
- 24 weeks
Methodological detailsplacebo-controlled, phase 3
- Placebo-controlled
- Phase 3
Primary result
As reported in the abstract
Change in KCCQ-OSS and SAQ7-SS
Aficamten
- KCCQ-Overall Summary Score (KCCQ-OSS) change
- 13.3 ± 16.3
- SAQ7-Summary Score (SAQ7-SS) change
- 11.6 ± 17.4
Placebo
- KCCQ-OSS change
- 6.1 ± 12.6
- SAQ7-SS change
- 3.8 ± 14.4
KCCQ-Overall Summary Score (KCCQ-OSS) change
Aficamten13.3 ± 16.3
Placebo6.1 ± 12.6
SAQ7-Summary Score (SAQ7-SS) change
Aficamten11.6 ± 17.4
Placebo3.8 ± 14.4
Aficamten vs PlaceboMean difference (95% CI)P value
KCCQ-OSS
7.9(4.8–11.0)
P < 0.001SAQ7-SS
7.8(4.7–11.0)
P < 0.001Further findings
Very large health status improvement (≥20 points)
KCCQ-OSS
SAQ7-SS
| Outcome | Aficamten | Placebo | P value |
|---|---|---|---|
| Change in health status after treatment withdrawal · 4-week washout | |||
| KCCQ-OSS change | −16.2 ± 19.0 | −3.0 ± 9.6 | P < 0.001 |
| SAQ7-SS change | −17.4 ± 21.4 | −2.5 ± 13.3 |
- Very large health status improvement (≥20 points) — NNT (KCCQ-OSS)
- 5.8
- Very large health status improvement (≥20 points) — NNT (SAQ7-SS)
- 5.8
Treatment effect across subgroups
- No heterogeneity in treatment effect across subgroups
Original publication
Effect of Aficamten on Health Status Outcomes in Obstructive Hypertrophic Cardiomyopathy: Results From SEQUOIA-HCM. J Am Coll Cardiol. Published 1 September 2024. doi:10.
Summarised from the published abstract; every number is checked against it