Long-term impact of aficamten on patient-reported outcome measures in obstructive hypertrophic cardiomyopathy: results from FOREST-HCM.
Shepard D Weiner, Lusha W Liang, Ahmad Masri et al.
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In 30 seconds
This open-label extension study assessed longer-term patient-reported outcomes with aficamten in people with obstructive hypertrophic cardiomyopathy who had completed an aficamten trial. Among 172 participants with at least 48 weeks of follow-up, improvements across all measured outcomes were evident by week 12 and persisted through week 48. At 48 weeks, the mean Kansas City Cardiomyopathy Questionnaire Overall Summary Score had improved by 18.8 (16.9-20.6) points (P < .001). Patient-reported improvements were also significantly correlated with clinical markers of disease severity.
Key findings
- At 48 weeks, the mean KCCQ Overall Summary Score improved by 18.8 (16.9-20.6) points (P < .001).
- The largest KCCQ gain was in Quality of Life: 25.8 points (23.2-28.5; P < .001).
- Seattle Angina Questionnaire 7-item Summary Scores increased by 17.2 (15.1-19.4; P < .001).
- EuroQol index and visual analogue scale scores improved by 0.11 and 12.7 points, respectively (P < .001); PRO improvements correlated with clinical markers of oHCM.
Why it matters
Patient-reported outcomes capture how symptoms and disease affect daily life, complementing clinical measures in obstructive hypertrophic cardiomyopathy. Sustained improvements across several health-status instruments over 48 weeks suggest that the observed benefit was not limited to a single questionnaire or early assessment. The correlations with clinical markers provide additional context for interpreting these reported changes.
What to keep in mind
FOREST-HCM was an open-label extension, and the abstract does not report a concurrent comparator; the findings therefore describe longer-term outcomes without establishing comparative effects.