Real-world candidacy to mavacamten in a contemporary hypertrophic obstructive cardiomyopathy population.

Autor:	Bertero E; Cardiovascular Unit, Department of Internal Medicine, University of Genova, Genova, Italy., Chiti C; Cardiomyopathy Unit, Careggi University Hospital, Florence, Italy., Schiavo MA; Cardiology Unit, Cardio-Thoraco-Vascular Department, IRCCS Azienda Ospedaliero Universitaria di Bologna, Bologna, Italy.; European Reference Network for Rare, Low Prevalence, and Complex Diseases of the Heart (ERN GUARD-Heart)., Tini G; Department of Clinical and Molecular Medicine, Sapienza University of Rome, Rome, Italy., Costa P; Cardiovascular Unit, Department of Internal Medicine, University of Genova, Genova, Italy., Todiere G; Cardiothoracic Department, Fondazione Toscana Gabriele Monasterio Pisa, Pisa, Italy., Mabritto B; Division of Cardiology, Azienda Sanitaria Ospedaliera Ordine Mauriziano, Torino, Italy., Dei LL; Cardiology Division, Cardiomyopathies Unit, St. Camillo Hospital, Rome, Italy.; Cardiology Unit, Department of Life, Health and Environmental Sciences, University of L'Aquila, L'Aquila, Italy., Giannattasio A; Department of Cardiac, Thoracic, and Vascular Sciences and Public Health, University of Padova, Padova, Italy., Mariani D; IRCCS, Istituto Auxologico Italiano, Department of Cardiology, San Luca Hospital, Cardiomyopathy Unit, Milan, Italy., Lofiego C; Department of Cardiology, Lancisi Cardiovascular Center, Marche University Hospital, Ancona, Italy., Santolamazza C; De Gasperis Cardio Center, ASST Grande Ospedale Metropolitano Niguarda, Milan, Italy., Monda E; Inherited and Rare Cardiovascular Diseases, Department of Translational Medical Sciences, University of Campania 'Luigi Vanvitelli', Monaldi Hospital, Naples, Italy., Quarta G; SC Cardiology 1, Cardiovascular Department, ASST Papa Giovanni XXIII, Bergamo, Italy., Barbisan D; European Reference Network for Rare, Low Prevalence, and Complex Diseases of the Heart (ERN GUARD-Heart).; Cardiothoracovascular Department, Azienda Sanitaria Universitaria Giuliano Isontina (ASUGI) and University of Trieste, Trieste, Italy., Mandoli GE; Division of Cardiology, Department of Medical Biotechnologies, University of Siena, Siena, Italy., Mapelli M; Centro Cardiologico Monzino IRCCS, Milan, Italy.; Department of Clinical Sciences and Community Health, Cardiovascular Section, University of Milan, Milan, Italy., Sguazzotti M; Cardiology Department, Ospedale Maggiore, Bologna, Italy., Negri F; Cardiology Department, University Hospital 'Santa Maria della Misericordia', Azienda Sanitaria Universitaria Integrata Friuli Centrale (ASUFC), Udine, Italy., De Vecchi S; Division of Cardiology, Maggiore della Carità Hospital, University of Eastern Piedmont, Novara, Italy., Ciabatti M; Cardiovascular Department, San Donato Hospital, Arezzo, Italy., Tomasoni D; Cardiology and Cardiac Catheterization Laboratory, Cardio-Thoracic Department, Civil Hospitals; Department of Medical and Surgical Specialties, Radiological Sciences, and Public Health, University of Brescia, Brescia, Italy., Mazzanti A; Department of Molecular Medicine, University of Pavia, Pavia, Italy.; Molecular Cardiology, IRCCS Istituti Clinici Scientifici Maugeri, Pavia, Italy., Marzo F; Cardiology Unit, Infermi Hospital, Rimini, Italy., de Gregorio C; Department of Clinical and Experimental Medicine, University Hospital of Messina, Messina, Italy., Raineri C; Division of Cardiology, Cardiovascular and Thoracic Department, 'Città della Salute e della Scienza, Hospital, Turin, Italy., Vianello PF; Cardiovascular Unit, IRCCS Ospedale Policlinico San Martino, Genoa, Italy., Marchi A; Cardiomyopathy Unit, Careggi University Hospital, Florence, Italy., Biagioni G; Cardiomyopathy Unit, Careggi University Hospital, Florence, Italy., Insinna E; Cardiomyopathy Unit, Careggi University Hospital, Florence, Italy., Parisi V; Cardiology Unit, Cardio-Thoraco-Vascular Department, IRCCS Azienda Ospedaliero Universitaria di Bologna, Bologna, Italy.; European Reference Network for Rare, Low Prevalence, and Complex Diseases of the Heart (ERN GUARD-Heart)., Ditaranto R; Cardiology Unit, Cardio-Thoraco-Vascular Department, IRCCS Azienda Ospedaliero Universitaria di Bologna, Bologna, Italy.; European Reference Network for Rare, Low Prevalence, and Complex Diseases of the Heart (ERN GUARD-Heart)., Barison A; Cardiothoracic Department, Fondazione Toscana Gabriele Monasterio Pisa, Pisa, Italy., Giammarresi A; SC Cardiology 1, Cardiovascular Department, ASST Papa Giovanni XXIII, Bergamo, Italy., De Ferrari GM; Division of Cardiology, Cardiovascular and Thoracic Department, 'Città della Salute e della Scienza, Hospital, Turin, Italy., Priori S; Department of Molecular Medicine, University of Pavia, Pavia, Italy.; Molecular Cardiology, IRCCS Istituti Clinici Scientifici Maugeri, Pavia, Italy., Metra M; Cardiology and Cardiac Catheterization Laboratory, Cardio-Thoracic Department, Civil Hospitals; Department of Medical and Surgical Specialties, Radiological Sciences, and Public Health, University of Brescia, Brescia, Italy., Pieroni M; Cardiovascular Department, San Donato Hospital, Arezzo, Italy., Patti G; Division of Cardiology, Maggiore della Carità Hospital, University of Eastern Piedmont, Novara, Italy., Imazio M; Cardiology Department, University Hospital 'Santa Maria della Misericordia', Azienda Sanitaria Universitaria Integrata Friuli Centrale (ASUFC), Udine, Italy., Perugini E; Cardiology Department, Ospedale Maggiore, Bologna, Italy., Agostoni P; Centro Cardiologico Monzino IRCCS, Milan, Italy.; Department of Clinical Sciences and Community Health, Cardiovascular Section, University of Milan, Milan, Italy., Cameli M; Division of Cardiology, Department of Medical Biotechnologies, University of Siena, Siena, Italy., Merlo M; Cardiothoracovascular Department, Azienda Sanitaria Universitaria Giuliano Isontina (ASUGI) and University of Trieste, Trieste, Italy., Sinagra G; Cardiothoracovascular Department, Azienda Sanitaria Universitaria Giuliano Isontina (ASUGI) and University of Trieste, Trieste, Italy., Senni M; SC Cardiology 1, Cardiovascular Department, ASST Papa Giovanni XXIII, Bergamo, Italy., Limongelli G; Inherited and Rare Cardiovascular Diseases, Department of Translational Medical Sciences, University of Campania 'Luigi Vanvitelli', Monaldi Hospital, Naples, Italy., Ammirati E; De Gasperis Cardio Center, ASST Grande Ospedale Metropolitano Niguarda, Milan, Italy., Vagnarelli F; Department of Cardiology, Lancisi Cardiovascular Center, Marche University Hospital, Ancona, Italy., Crotti L; IRCCS, Istituto Auxologico Italiano, Department of Cardiology, San Luca Hospital, Cardiomyopathy Unit, Milan, Italy.; Department of Medicine and Surgery, University of Milano-Bicocca, Milan, Italy., Badano L; IRCCS, Istituto Auxologico Italiano, Department of Cardiology, San Luca Hospital, Cardiomyopathy Unit, Milan, Italy., Calore C; Department of Cardiac, Thoracic, and Vascular Sciences and Public Health, University of Padova, Padova, Italy., Gabrielli D; Cardiology Division, Cardiomyopathies Unit, St. Camillo Hospital, Rome, Italy., Re F; Cardiology Division, Cardiomyopathies Unit, St. Camillo Hospital, Rome, Italy., Musumeci G; Division of Cardiology, Azienda Sanitaria Ospedaliera Ordine Mauriziano, Torino, Italy., Emdin M; Cardiothoracic Department, Fondazione Toscana Gabriele Monasterio Pisa, Pisa, Italy., Barbato E; Department of Clinical and Molecular Medicine, Sapienza University of Rome, Rome, Italy., Musumeci B; Department of Clinical and Molecular Medicine, Sapienza University of Rome, Rome, Italy., Autore C; Department of Cardiology and Respiratory Sciences, San Raffaele Cassino, Cassino, Italy., Biagini E; Cardiology Unit, Cardio-Thoraco-Vascular Department, IRCCS Azienda Ospedaliero Universitaria di Bologna, Bologna, Italy., Porto I; Cardiovascular Unit, Department of Internal Medicine, University of Genova, Genova, Italy.; Cardiovascular Unit, IRCCS Ospedale Policlinico San Martino, Genoa, Italy., Olivotto I; Cardiomyopathy Unit, Careggi University Hospital, Florence, Italy., Canepa M; Cardiovascular Unit, Department of Internal Medicine, University of Genova, Genova, Italy.; Cardiovascular Unit, IRCCS Ospedale Policlinico San Martino, Genoa, Italy.
Jazyk:	angličtina
Zdroj:	European journal of heart failure [Eur J Heart Fail] 2024 Jan; Vol. 26 (1), pp. 59-64. Date of Electronic Publication: 2024 Jan 02.
DOI:	10.1002/ejhf.3120
Abstrakt:	Aims: In the EXPLORER-HCM trial, mavacamten reduced left ventricular outflow tract obstruction (LVOTO) and improved functional capacity of symptomatic hypertrophic obstructive cardiomyopathy (HOCM) patients. We sought to define the potential use of mavacamten by comparing real-world HOCM patients with those enrolled in EXPLORER-HCM and assessing their eligibility to treatment. Methods and Results: We collected information on HOCM patients followed up at 25 Italian HCM outpatient clinics and with significant LVOTO (i.e. gradient ≥30 mmHg at rest or ≥50 mmHg after Valsalva manoeuvre or exercise) despite pharmacological or non-pharmacological therapy. Pharmacological or non-pharmacological therapy resolved LVOTO in 1044 (61.2%) of the 1706 HOCM patients under active follow-up, whereas 662 patients (38.8%) had persistent LVOTO. Compared to the EXPLORER-HCM trial population, these real-world HOCM patients were older (62.1 ± 14.3 vs. 58.5 ± 12.2 years, p = 0.02), had a lower body mass index (26.8 ± 5.3 vs. 29.7 ± 4.9 kg/m 2 , p < 0.0001) and a more frequent history of atrial fibrillation (21.5% vs. 9.8%, p = 0.027). At echocardiography, they had lower left ventricular ejection fraction (LVEF, 66 ± 7% vs. 74 ± 6%, p < 0.0001), higher left ventricular outflow tract gradients at rest (60 ± 27 vs. 52 ± 29 mmHg, p = 0.003), and larger left atrial volume index (49 ± 16 vs. 40 ± 12 ml/m 2 , p < 0.0001). Overall, 324 (48.9%) would have been eligible for enrolment in the EXPLORER-HCM trial and 339 (51.2%) for treatment with mavacamten according to European guidelines. Conclusions: Real-world HOCM patients differ from the EXPLORER-HCM population for their older age, lower LVEF and larger atrial volume, potentially reflecting a more advanced stage of the disease. About half of real-world HOCM patients were found eligible to mavacamten. (© 2023 The Authors. European Journal of Heart Failure published by John Wiley & Sons Ltd on behalf of European Society of Cardiology.)
Databáze:	MEDLINE
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