Медицинская библиотека · International journal of cardiology · 2026

Baseline clinical profile of patients with obstructive hypertrophic cardiomyopathy in the Italian Mavacamten early access program.

Bonacchi G, Del Franco A, Agostoni P, Agricola E, Ammirati E, Baratta P, Barocelli F, Bertero E, Biagini E, Boriani G, Calabrò P, Calò L, Calore C, Carigi S, Carluccio E, Cerrato N, Chimenti C, Chinaglia A, Chiriatti C, Crotti L, De Caterina R, Di Marco M, Di Spigno F, Evangelista A, Galasso G, Graziani F, Marra W Grosso, Guaricci A I, Guerra F, Leuzzi C, Limongelli G, Lofiego C, Losi M A, Mabritto B, Marazia S, Merlo M, Monte I, Moretti M, Murzilli R, Musumeci M B, Negri F, Pasqualucci D, Patti G, Perugini E, Piepoli M F, Pontone G, Quarta G, Raineri C, Re F, Ricci F, Ruggieri R, Saletti E, Santolamazza C, Scelsi L, Spoladore R, Todiere G, Pieroni M, Sinagra G, Olivotto I, partecipating Centers of the Early Access Program for Mavacamten, Italy

СтатьяМногоцентровое исследованиеПеревод в очереди

Аннотация

BACKGROUND

Obstructive hypertrophic cardiomyopathy (oHCM) has recently seen the introduction of mavacamten, a disease-specific therapy that alleviates obstruction and symptoms with potential effects on disease expression. Since most patients remain symptomatic with conventional therapies, mavacamten constitutes an efficacious treatment strategy

METHODS

The early access program (EAP) for mavacamten treatment ran from September 2023 to September 2024. We collected clinical and echocardiographic data prospectively

RESULTS

337 patients with oHCM (53% male, mean age 61.4 ± 12.3 years) were receiving mavacamten treatment from 54 centers in Italy. The EAP cohort was older and showed a more advanced cardiac phenotype, showing lower left ventricular ejection fraction (LVEF) (66 ± 6% vs 74 ± 6% in EXPLORER-HCM cohort, p < 0.0001) and higher left atrial volume index (51.1 ± 16.4 ml/m2 vs 40.5 ± 12.3 ml/m2, p < 0.0001. Only 74 (22%) patients in the EAP group were candidates for septal reduction therapy (SRT). Echocardiographic parameters like LVEF, maximal wall thickness and LAVI as well as pharmacologic therapy showed no difference between subgroups, while LVOT obstruction gradient was higher in SRT candidates (40 vs 63 mmHg, p < 0.0001)..

CONCLUSION

The compassionate use program for mavacamten in Italy demonstrated significant engagement. The Italian oHCM cohort were older and exhibited more advanced disease stages compared to those in the EXPLORER-HCM trial. Only one-quarter of the patients were eligible for SRT, they did not show a more severe biomarker or echocardiographic parameters compared to those without indication. Mavacamten provides an additional option for oHCM patients unresponsive to standard therapy, especially those symptomatic but ineligible for SRT.

Для цитирования: Bonacchi G, Del Franco A, Agostoni P, Agricola E, Ammirati E, Baratta P, Barocelli F, Bertero E, Biagini E, Boriani G, Calabrò P, Calò L, Calore C, Carigi S, Carluccio E, Cerrato N, Chimenti C, Chinaglia A, Chiriatti C, Crotti L, De Caterina R, Di Marco M, Di Spigno F, Evangelista A, Galasso G, Graziani F, Marra W Grosso, Guaricci A I, Guerra F, Leuzzi C, Limongelli G, Lofiego C, Losi M A, Mabritto B, Marazia S, Merlo M, Monte I, Moretti M, Murzilli R, Musumeci M B, Negri F, Pasqualucci D, Patti G, Perugini E, Piepoli M F, Pontone G, Quarta G, Raineri C, Re F, Ricci F, Ruggieri R, Saletti E, Santolamazza C, Scelsi L, Spoladore R, Todiere G, Pieroni M, Sinagra G, Olivotto I, partecipating Centers of the Early Access Program for Mavacamten, Italy. Baseline clinical profile of patients with obstructive hypertrophic cardiomyopathy in the Italian Mavacamten early access program.. International journal of cardiology. 2026. DOI: 10.1016/j.ijcard.2025.134152.

Предметные рубрики MeSH

Aged Female Humans Male Middle Aged Cardiomyopathy, Hypertrophic Cohort Studies Echocardiography Italy Prospective Studies Treatment Outcome Benzylamines Uracil

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