Heart failure & cardiomyopathies

Efficacy of aficamten in patients with obstructive hypertrophic cardiomyopathy and mild symptoms: results from the SEQUOIA-HCM trial

Eur Heart JPublished Secondary analysis of the SEQUOIA-HCM randomised trial

Authors' conclusion

Patients with oHCM and mild symptoms treated with aficamten achieved significant improvement across a range of clinically relevant outcomes and generally similar to patients with more advanced symptoms.

Study context

Population
Patients with obstructive hypertrophic cardiomyopathy (oHCM) in SEQUOIA-HCM
Participants
282 patients
Comparison
Aficamten in mild vs moderate to severe symptoms
  • Mild symptomsn = 118
  • Moderate to severe symptomsn = 150reference
Primary endpoint
Change in peak oxygen uptake (pVO2) from baseline
Follow-up
Week 24
Methodological detailssubgroup analysis by baseline symptom severity
  • Subgroup analysis by baseline symptom severity

Primary result

As reported in the abstract

Change in pVO2, aficamten-treated

Moderate to severe symptoms

1.8

mL/kg/min · n = 71

Mild symptoms

1.6

mL/kg/min · n = 62

P = 0.8

Secondary outcomes

New York Heart Association (NYHA) class, resting or Valsalva gradients, NT-proBNP

Change did not differ significantly between the two symptom groups

Kansas City Cardiomyopathy Questionnaire Clinical Summary Score (KCCQ-CSS)

Significant improvement in both groups; greater in the advanced symptom group
P = 0.02 for interaction
  • Interaction P = .02

Safety

Treatment-emergent serious adverse events

Infrequent in both groups

Original publication

Efficacy of aficamten in patients with obstructive hypertrophic cardiomyopathy and mild symptoms: results from the SEQUOIA-HCM trial. Eur Heart J. Published 17 May 2025. doi:10.1093/eurheartj/ehaf364

Summarised from the published abstract; every number is checked against it

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