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Clinical Trial
. 2024 Jan;26(1):59-64.
doi: 10.1002/ejhf.3120. Epub 2024 Jan 2.

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

Edoardo Bertero  1 , Chiara Chiti  2 , Maria Alessandra Schiavo  3   4 , Giacomo Tini  5 , Paolo Costa  1 , Giancarlo Todiere  6 , Barbara Mabritto  7 , Lorenzo-Lupo Dei  8   9 , Alessia Giannattasio  10 , Davide Mariani  11 , Carla Lofiego  12 , Caterina Santolamazza  13 , Emanuele Monda  14 , Giovanni Quarta  15 , Davide Barbisan  4   16 , Giulia Elena Mandoli  17 , Massimo Mapelli  18   19 , Maurizio Sguazzotti  20 , Francesco Negri  21 , Simona De Vecchi  22 , Michele Ciabatti  23 , Daniela Tomasoni  24 , Andrea Mazzanti  25   26 , Francesca Marzo  27 , Cesare de Gregorio  28 , Claudia Raineri  29 , Pier Filippo Vianello  30 , Alberto Marchi  2 , Giulia Biagioni  2 , Eleonora Insinna  2 , Vanda Parisi  3   4 , Raffaello Ditaranto  3   4 , Andrea Barison  6 , Andrea Giammarresi  15 , Gaetano Maria De Ferrari  29 , Silvia Priori  25   26 , Marco Metra  24 , Maurizio Pieroni  23 , Giuseppe Patti  22 , Massimo Imazio  21 , Enrica Perugini  20 , Piergiuseppe Agostoni  18   19 , Matteo Cameli  17 , Marco Merlo  16 , Gianfranco Sinagra  16 , Michele Senni  15 , Giuseppe Limongelli  14 , Enrico Ammirati  13 , Fabio Vagnarelli  12 , Lia Crotti  11   31 , Luigi Badano  11 , Chiara Calore  10 , Domenico Gabrielli  8 , Federica Re  8 , Giuseppe Musumeci  7 , Michele Emdin  6 , Emanuele Barbato  5 , Beatrice Musumeci  5 , Camillo Autore  32 , Elena Biagini  3 , Italo Porto  1   30 , Iacopo Olivotto  2 , Marco Canepa  1   30
Affiliations
Clinical Trial

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

Edoardo Bertero et al. Eur J Heart Fail. 2024 Jan.

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.

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References

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