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

Edoardo Bertero; Chiara Chiti; Maria Alessandra Schiavo; Giacomo Tini; Paolo Costa; Giancarlo Todiere; Barbara Mabritto; Lorenzo Lupo Dei; Alessia Giannattasio; Davide Mariani; Carla Lofiego; Caterina Santolamazza; Emanuele Monda; Giovanni Quarta; Davide Barbisan; Giulia Elena Mandoli; Massimo Mapelli; Maurizio Sguazzotti; Francesco Negri; Simona De Vecchi; Michele Ciabatti; Daniela Tomasoni; Andrea Mazzanti; Francesca Marzo; Cesare de Gregorio; Claudia Raineri; Pier Filippo Vianello; Alberto Marchi; Giulia Biagioni; Eleonora Insinna; Vanda Parisi; Raffaello Ditaranto; Andrea Barison; Andrea Giammarresi; Gaetano Maria De Ferrari; Silvia Priori; Marco Metra; Maurizio Pieroni; Giuseppe Patti; Massimo Imazio; Enrica Perugini; Piergiuseppe Agostoni; Matteo Cameli; Marco Merlo; Gianfranco Sinagra; Michele Senni; Giuseppe Limongelli; Enrico Ammirati; Fabio Vagnarelli; Lia Crotti; Luigi Badano; Chiara Calore; Domenico Gabrielli; Federica Re; Giuseppe Musumeci; Michele Emdin; Emanuele Barbato; Beatrice Musumeci; Camillo Autore; Elena Biagini; Italo Porto; Iacopo Olivotto; Marco Canepa

doi:10.1002/ejhf.3120

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

Edoardo Bertero, Chiara Chiti, Maria Alessandra Schiavo, Giacomo Tini, Paolo Costa, Giancarlo Todiere, Barbara Mabritto, Lorenzo Lupo Dei, Alessia Giannattasio, Davide Mariani, Carla Lofiego, Caterina Santolamazza, Emanuele Monda, Giovanni Quarta, Davide Barbisan, Giulia Elena Mandoli, Massimo Mapelli, Maurizio Sguazzotti, Francesco Negri, Simona De VecchiMichele Ciabatti, Daniela Tomasoni, Andrea Mazzanti, Francesca Marzo, Cesare de Gregorio, Claudia Raineri, Pier Filippo Vianello, Alberto Marchi, Giulia Biagioni, Eleonora Insinna, Vanda Parisi, Raffaello Ditaranto, Andrea Barison, Andrea Giammarresi, Gaetano Maria De Ferrari, Silvia Priori, Marco Metra, Maurizio Pieroni, Giuseppe Patti, Massimo Imazio, Enrica Perugini, Piergiuseppe Agostoni, Matteo Cameli, Marco Merlo, Gianfranco Sinagra, Michele Senni, Giuseppe Limongelli, Enrico Ammirati, Fabio Vagnarelli, Lia Crotti, Luigi Badano, Chiara Calore, Domenico Gabrielli, Federica Re, Giuseppe Musumeci, Michele Emdin, Emanuele Barbato, Beatrice Musumeci, Camillo Autore, Elena Biagini, Italo Porto, Iacopo Olivotto, Marco Canepa

Risultato della ricerca: Contributo su rivista › Articolo in rivista › peer review

Abstract

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/m², 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/m², 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.

Lingua originale	Inglese
pagine (da-a)	59-64
Numero di pagine	6
Rivista	European Journal of Heart Failure
Volume	26
Numero di pubblicazione	1
DOI	https://doi.org/10.1002/ejhf.3120
Stato di pubblicazione	Pubblicato - gen 2024
Pubblicato esternamente	Sì

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10.1002/ejhf.3120

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Bertero, E., Chiti, C., Schiavo, M. A., Tini, G., Costa, P., Todiere, G., Mabritto, B., Dei, L. L., Giannattasio, A., Mariani, D., Lofiego, C., Santolamazza, C., Monda, E., Quarta, G., Barbisan, D., Mandoli, G. E., Mapelli, M., Sguazzotti, M., Negri, F., ... Canepa, M. (2024). Real-world candidacy to mavacamten in a contemporary hypertrophic obstructive cardiomyopathy population. European Journal of Heart Failure, 26(1), 59-64. https://doi.org/10.1002/ejhf.3120

@article{7d14388913354bffab5028607482bce8,

title = "Real-world candidacy to mavacamten in a contemporary hypertrophic obstructive cardiomyopathy population",

abstract = "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/m2, 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/m2, 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.",

keywords = "EXPLORER-HCM trial, Hypertrophic obstructive cardiomyopathy, Mavacamten, Myosin inhibitors",

author = "Edoardo Bertero and Chiara Chiti and Schiavo, {Maria Alessandra} and Giacomo Tini and Paolo Costa and Giancarlo Todiere and Barbara Mabritto and Dei, {Lorenzo Lupo} and Alessia Giannattasio and Davide Mariani and Carla Lofiego and Caterina Santolamazza and Emanuele Monda and Giovanni Quarta and Davide Barbisan and Mandoli, {Giulia Elena} and Massimo Mapelli and Maurizio Sguazzotti and Francesco Negri and {De Vecchi}, Simona and Michele Ciabatti and Daniela Tomasoni and Andrea Mazzanti and Francesca Marzo and {de Gregorio}, Cesare and Claudia Raineri and Vianello, {Pier Filippo} and Alberto Marchi and Giulia Biagioni and Eleonora Insinna and Vanda Parisi and Raffaello Ditaranto and Andrea Barison and Andrea Giammarresi and {De Ferrari}, {Gaetano Maria} and Silvia Priori and Marco Metra and Maurizio Pieroni and Giuseppe Patti and Massimo Imazio and Enrica Perugini and Piergiuseppe Agostoni and Matteo Cameli and Marco Merlo and Gianfranco Sinagra and Michele Senni and Giuseppe Limongelli and Enrico Ammirati and Fabio Vagnarelli and Lia Crotti and Luigi Badano and Chiara Calore and Domenico Gabrielli and Federica Re and Giuseppe Musumeci and Michele Emdin and Emanuele Barbato and Beatrice Musumeci and Camillo Autore and Elena Biagini and Italo Porto and Iacopo Olivotto and Marco Canepa",

note = "Publisher Copyright: {\textcopyright} 2023 The Authors. European Journal of Heart Failure published by John Wiley & Sons Ltd on behalf of European Society of Cardiology.",

year = "2024",

month = jan,

doi = "10.1002/ejhf.3120",

language = "English",

volume = "26",

pages = "59--64",

journal = "European Journal of Heart Failure",

issn = "1388-9842",

publisher = "John Wiley and Sons Ltd",

number = "1",

}

Bertero, E, Chiti, C, Schiavo, MA, Tini, G, Costa, P, Todiere, G, Mabritto, B, Dei, LL, Giannattasio, A, Mariani, D, Lofiego, C, Santolamazza, C, Monda, E, Quarta, G, Barbisan, D, Mandoli, GE, Mapelli, M, Sguazzotti, M, Negri, F, De Vecchi, S, Ciabatti, M, Tomasoni, D, Mazzanti, A, Marzo, F, de Gregorio, C, Raineri, C, Vianello, PF, Marchi, A, Biagioni, G, Insinna, E, Parisi, V, Ditaranto, R, Barison, A, Giammarresi, A, De Ferrari, GM, Priori, S, Metra, M, Pieroni, M, Patti, G, Imazio, M, Perugini, E, Agostoni, P, Cameli, M, Merlo, M, Sinagra, G, Senni, M, Limongelli, G, Ammirati, E, Vagnarelli, F, Crotti, L, Badano, L, Calore, C, Gabrielli, D, Re, F, Musumeci, G, Emdin, M, Barbato, E, Musumeci, B, Autore, C, Biagini, E, Porto, I, Olivotto, I & Canepa, M 2024, 'Real-world candidacy to mavacamten in a contemporary hypertrophic obstructive cardiomyopathy population', European Journal of Heart Failure, vol. 26, n. 1, pagg. 59-64. https://doi.org/10.1002/ejhf.3120

TY - JOUR

T1 - Real-world candidacy to mavacamten in a contemporary hypertrophic obstructive cardiomyopathy population

AU - Bertero, Edoardo

AU - Chiti, Chiara

AU - Schiavo, Maria Alessandra

AU - Tini, Giacomo

AU - Costa, Paolo

AU - Todiere, Giancarlo

AU - Mabritto, Barbara

AU - Dei, Lorenzo Lupo

AU - Giannattasio, Alessia

AU - Mariani, Davide

AU - Lofiego, Carla

AU - Santolamazza, Caterina

AU - Monda, Emanuele

AU - Quarta, Giovanni

AU - Barbisan, Davide

AU - Mandoli, Giulia Elena

AU - Mapelli, Massimo

AU - Sguazzotti, Maurizio

AU - Negri, Francesco

AU - De Vecchi, Simona

AU - Ciabatti, Michele

AU - Tomasoni, Daniela

AU - Mazzanti, Andrea

AU - Marzo, Francesca

AU - de Gregorio, Cesare

AU - Raineri, Claudia

AU - Vianello, Pier Filippo

AU - Marchi, Alberto

AU - Biagioni, Giulia

AU - Insinna, Eleonora

AU - Parisi, Vanda

AU - Ditaranto, Raffaello

AU - Barison, Andrea

AU - Giammarresi, Andrea

AU - De Ferrari, Gaetano Maria

AU - Priori, Silvia

AU - Metra, Marco

AU - Pieroni, Maurizio

AU - Patti, Giuseppe

AU - Imazio, Massimo

AU - Perugini, Enrica

AU - Agostoni, Piergiuseppe

AU - Cameli, Matteo

AU - Merlo, Marco

AU - Sinagra, Gianfranco

AU - Senni, Michele

AU - Limongelli, Giuseppe

AU - Ammirati, Enrico

AU - Vagnarelli, Fabio

AU - Crotti, Lia

AU - Badano, Luigi

AU - Calore, Chiara

AU - Gabrielli, Domenico

AU - Re, Federica

AU - Musumeci, Giuseppe

AU - Emdin, Michele

AU - Barbato, Emanuele

AU - Musumeci, Beatrice

AU - Autore, Camillo

AU - Biagini, Elena

AU - Porto, Italo

AU - Olivotto, Iacopo

AU - Canepa, Marco

PY - 2024/1

Y1 - 2024/1

N2 - 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/m2, 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/m2, 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.

AB - 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/m2, 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/m2, 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.

KW - EXPLORER-HCM trial

KW - Hypertrophic obstructive cardiomyopathy

KW - Mavacamten

KW - Myosin inhibitors

UR - http://www.scopus.com/inward/record.url?scp=85181192117&partnerID=8YFLogxK

U2 - 10.1002/ejhf.3120

DO - 10.1002/ejhf.3120

M3 - Article

SN - 1388-9842

VL - 26

SP - 59

EP - 64

JO - European Journal of Heart Failure

JF - European Journal of Heart Failure

IS - 1

ER -

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

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