Efficacy of aficamten in patients with obstructive hypertrophic cardiomyopathy and mild symptoms: results from the SEQUOIA-HCM trial
Jazyk angličtina Země Anglie, Velká Británie Médium print-electronic
Typ dokumentu klinické zkoušky, fáze III, časopisecké články, multicentrická studie, randomizované kontrolované studie
Grantová podpora
Cytokinetics, Incorporated
PubMed
41120118
PubMed Central
PMC12539928
DOI
10.1093/eurheartj/ehaf364
PII: 8133640
Knihovny.cz E-zdroje
- Klíčová slova
- Clinical trial, Hypertrophic cardiomyopathy, Obstructive hypertrophic cardiomyopathy, Therapy, aficamten,
- MeSH
- hypertrofická kardiomyopatie * krev diagnóza farmakoterapie patofyziologie MeSH
- lidé středního věku MeSH
- lidé MeSH
- natriuretický peptid typu B krev metabolismus MeSH
- peptidové fragmenty krev metabolismus MeSH
- pyrazoly * aplikace a dávkování škodlivé účinky MeSH
- senioři MeSH
- výsledek terapie MeSH
- Check Tag
- lidé středního věku MeSH
- lidé MeSH
- mužské pohlaví MeSH
- senioři MeSH
- ženské pohlaví MeSH
- Publikační typ
- časopisecké články MeSH
- klinické zkoušky, fáze III MeSH
- multicentrická studie MeSH
- randomizované kontrolované studie MeSH
- Názvy látek
- aficamten MeSH Prohlížeč
- natriuretický peptid typu B MeSH
- peptidové fragmenty MeSH
- pro-brain natriuretic peptide (1-76) MeSH Prohlížeč
- pyrazoly * MeSH
BACKGROUND AND AIMS: Patients with obstructive hypertrophic cardiomyopathy (oHCM) treated with aficamten in SEQUOIA-HCM (NCT05186818) demonstrated marked improvement in symptoms and functional capacity. This analysis explores whether oHCM and mild symptoms patients experience similar clinical benefits with aficamten as patients with more advanced limitations. METHODS: Patients in SEQUOIA-HCM (N = 282) were grouped at baseline according to symptom severity. Mild symptoms (n = 118) were defined as New York Heart Association (NYHA) class II and Kansas City Cardiomyopathy Questionnaire Clinical Summary Score (KCCQ-CSS) ≥ 80, and moderate to severe symptoms (n = 150) as NYHA class II/III/IV and KCCQ-CSS <80. Primary endpoint was change in peak oxygen uptake (pVO2) from baseline to Week 24; secondary endpoints included change in NYHA class, KCCQ-CSS, outflow tract gradients, and N-terminal pro-B-type natriuretic peptide (NT-proBNP). RESULTS: In aficamten-treated patients, change at Week 24 was not different between moderate to severe (1.8 mL/kg/min; n = 71) and mild (1.6 mL/kg/min; n = 62) symptom groups (P = .8). Likewise, the change in secondary endpoints (NYHA class, resting or Valsalva gradients, and NT-proBNP) did not differ significantly between the two symptom groups. Both groups experienced statistically significant improvements in KCCQ-CSS, but the extent of improvement was greater in the advanced symptom group (P = .02 for interaction). Treatment-emergent serious adverse events were infrequent in both groups. CONCLUSIONS: 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. Less severely symptomatic patients could be considered for aficamten treatment.
Baim Institute for Clinical Research Boston MA USA
Brigham and Women's Hospital Boston MA USA
Cardiac Department University Hospital Virgen Arrixaca CIBERCV ERN Guard Heart Murcia Spain
Cardiovascular Division Brigham and Women's Hospital Harvard Medical School Boston MA USA
Cardiovascular Division Oregon Health and Science University Portland OR USA
Complexo Hospitalario Universitario A Coruña INIBIC CIBERCV ISCIII A Coruña Spain
Cytokinetics Incorporated South San Francisco CA USA
Institute of Health Information and Statistics Prague Czech Republic
Meyer Children's Hospital Instituto di Ricovero e Cura a Carattere Scientifico Florence Italy
Saint Luke's Mid America Heart Institute Kansas City MO USA
School of Cardiovascular and Metabolic Health University of Glasgow Glasgow Scotland
doi: 10.1093/eurheartj/ehaf507 PubMed
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