Natural History of MYH7-Related Dilated Cardiomyopathy.

Variants in myosin heavy chain 7 (MYH7) are responsible for disease in 1% to 5% of patients with dilated cardiomyopathy (DCM); however, the clinical characteristics and natural history of MYH7-related DCM are poorly described. We sought to determine the phenotype and prognosis of MYH7-related DCM. W...

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Autores: de Frutos, Fernando, Ochoa, Juan Pablo, Navarro-Peñalver, Marina, Baas, Annette, Bjerre, Jesper Vandborg, Zorio, Esther, Méndez, Irene, Lorca, Rebeca, Verdonschot, Job A J, García-Granja, Pablo Elpidio, Bilinska, Zofia, Fatkin, Diane, Fuentes-Cañamero, M Eugenia, García-Pinilla, José M, García-Álvarez, María I, Girolami, Francesca, Barriales-Villa, Roberto, Díez-López, Carles, Lopes, Luis R, Wahbi, Karim, García-Álvarez, Ana, Rodríguez-Sánchez, Ibon, Rekondo-Olaetxea, Javier, Rodríguez-Palomares, José F, Gallego-Delgado, María, Meder, Benjamin, Kubanek, Milos, Hansen, Frederikke G, Restrepo-Córdoba, María Alejandra, Palomino-Doza, Julián, Ruiz-Guerrero, Luis, Sarquella-Brugada, Georgia, Perez-Perez, Alberto José, Bermúdez-Jiménez, Francisco José, Ripoll-Vera, Tomas, Rasmussen, Torsten Bloch, Jansen, Mark, Sabater-Molina, Maria, Elliot, Perry M, Garcia-Pavia, Pablo
Tipo de recurso: artículo
Fecha de publicación:2022
País:España
Institución:Instituto de Salud Carlos III (ISCIII)
Repositorio:Repisalud
Idioma:inglés
OAI Identifier:oai:repisalud.isciii.es:20.500.12105/15246
Acceso en línea:http://hdl.handle.net/20.500.12105/15246
Access Level:acceso abierto
Palabra clave:Cardiomyopathy, Dilated
Heart Failure
Myosin Heavy Chains
Adolescent
Adult
Arrhythmias, Cardiac
Cardiac Myosins
Female
Humans
Male
Middle Aged
Phenotype
Ventricular Remodeling
Young Adult
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repository_id_str
spelling Natural History of MYH7-Related Dilated Cardiomyopathy.de Frutos, FernandoOchoa, Juan PabloNavarro-Peñalver, MarinaBaas, AnnetteBjerre, Jesper VandborgZorio, EstherMéndez, IreneLorca, RebecaVerdonschot, Job A JGarcía-Granja, Pablo ElpidioBilinska, ZofiaFatkin, DianeFuentes-Cañamero, M EugeniaGarcía-Pinilla, José MGarcía-Álvarez, María IGirolami, FrancescaBarriales-Villa, RobertoDíez-López, CarlesLopes, Luis RWahbi, KarimGarcía-Álvarez, AnaRodríguez-Sánchez, IbonRekondo-Olaetxea, JavierRodríguez-Palomares, José FGallego-Delgado, MaríaMeder, BenjaminKubanek, MilosHansen, Frederikke GRestrepo-Córdoba, María AlejandraPalomino-Doza, JuliánRuiz-Guerrero, LuisSarquella-Brugada, GeorgiaPerez-Perez, Alberto JoséBermúdez-Jiménez, Francisco JoséRipoll-Vera, TomasRasmussen, Torsten BlochJansen, MarkSabater-Molina, MariaElliot, Perry MGarcia-Pavia, PabloCardiomyopathy, DilatedHeart FailureMyosin Heavy ChainsAdolescentAdultArrhythmias, CardiacCardiac MyosinsFemaleHumansMaleMiddle AgedPhenotypeVentricular RemodelingYoung AdultVariants in myosin heavy chain 7 (MYH7) are responsible for disease in 1% to 5% of patients with dilated cardiomyopathy (DCM); however, the clinical characteristics and natural history of MYH7-related DCM are poorly described. We sought to determine the phenotype and prognosis of MYH7-related DCM. We also evaluated the influence of variant location on phenotypic expression. We studied clinical data from 147 individuals with DCM-causing MYH7 variants (47.6% female; 35.6 ± 19.2 years) recruited from 29 international centers. At initial evaluation, 106 (72.1%) patients had DCM (left ventricular ejection fraction: 34.5% ± 11.7%). Median follow-up was 4.5 years (IQR: 1.7-8.0 years), and 23.7% of carriers who were initially phenotype-negative developed DCM. Phenotypic expression by 40 and 60 years was 46% and 88%, respectively, with 18 patients (16%) first diagnosed at <18 years of age. Thirty-six percent of patients with DCM met imaging criteria for LV noncompaction. During follow-up, 28% showed left ventricular reverse remodeling. Incidence of adverse cardiac events among patients with DCM at 5 years was 11.6%, with 5 (4.6%) deaths caused by end-stage heart failure (ESHF) and 5 patients (4.6%) requiring heart transplantation. The major ventricular arrhythmia rate was low (1.0% and 2.1% at 5 years in patients with DCM and in those with LVEF of ≤35%, respectively). ESHF and major ventricular arrhythmia were significantly lower compared with LMNA-related DCM and similar to DCM caused by TTN truncating variants. MYH7-related DCM is characterized by early age of onset, high phenotypic expression, low left ventricular reverse remodeling, and frequent progression to ESHF. Heart failure complications predominate over ventricular arrhythmias, which are rare.ElsevierInstituto de Salud Carlos IIIUnión Europea. Fondo Europeo de Desarrollo Regional (FEDER/ERDF)Ministerio de Ciencia e Innovación (España)Fundación ProCNICMinisterio de Ciencia e Innovación. Centro de Excelencia Severo Ochoa (España)European Reference Network for Rare and Low Prevalence Complex Diseases of the HeartUnión Europea. Comisión Europea. H2020. ERA-CVDDutch Heart Foundation (Holanda)Victor Chang Cardiac Research InstituteNSW HealthClinical Academic Research Partnerships (CARP)Deutsches Zentrum für Herz-Kreislauf-Forschung (German Center for Cardiovascular Research)Informatics for Life (Klaus Tschira Foundation)Ministry of Health (República Checa)Institute for Clinical and Experimental Medicine–IKEM20222022-11-2920222022-10-1120222022-10-11journal articlehttp://purl.org/coar/resource_type/c_6501VoRhttp://purl.org/coar/version/c_970fb48d4fbd8a85info:eu-repo/semantics/articleapplication/pdfhttp://hdl.handle.net/20.500.12105/15246reponame:Repisaludinstname:Instituto de Salud Carlos III (ISCIII)Inglésengopen accesshttp://purl.org/coar/access_right/c_abf2Atribución 4.0 Internacionalhttp://creativecommons.org/licenses/by/4.0/info:eu-repo/semantics/openAccessoai:repisalud.isciii.es:20.500.12105/152462026-06-12T12:43:37Z
dc.title.none.fl_str_mv Natural History of MYH7-Related Dilated Cardiomyopathy.
title Natural History of MYH7-Related Dilated Cardiomyopathy.
spellingShingle Natural History of MYH7-Related Dilated Cardiomyopathy.
de Frutos, Fernando
Cardiomyopathy, Dilated
Heart Failure
Myosin Heavy Chains
Adolescent
Adult
Arrhythmias, Cardiac
Cardiac Myosins
Female
Humans
Male
Middle Aged
Phenotype
Ventricular Remodeling
Young Adult
title_short Natural History of MYH7-Related Dilated Cardiomyopathy.
title_full Natural History of MYH7-Related Dilated Cardiomyopathy.
title_fullStr Natural History of MYH7-Related Dilated Cardiomyopathy.
title_full_unstemmed Natural History of MYH7-Related Dilated Cardiomyopathy.
title_sort Natural History of MYH7-Related Dilated Cardiomyopathy.
dc.creator.none.fl_str_mv de Frutos, Fernando
Ochoa, Juan Pablo
Navarro-Peñalver, Marina
Baas, Annette
Bjerre, Jesper Vandborg
Zorio, Esther
Méndez, Irene
Lorca, Rebeca
Verdonschot, Job A J
García-Granja, Pablo Elpidio
Bilinska, Zofia
Fatkin, Diane
Fuentes-Cañamero, M Eugenia
García-Pinilla, José M
García-Álvarez, María I
Girolami, Francesca
Barriales-Villa, Roberto
Díez-López, Carles
Lopes, Luis R
Wahbi, Karim
García-Álvarez, Ana
Rodríguez-Sánchez, Ibon
Rekondo-Olaetxea, Javier
Rodríguez-Palomares, José F
Gallego-Delgado, María
Meder, Benjamin
Kubanek, Milos
Hansen, Frederikke G
Restrepo-Córdoba, María Alejandra
Palomino-Doza, Julián
Ruiz-Guerrero, Luis
Sarquella-Brugada, Georgia
Perez-Perez, Alberto José
Bermúdez-Jiménez, Francisco José
Ripoll-Vera, Tomas
Rasmussen, Torsten Bloch
Jansen, Mark
Sabater-Molina, Maria
Elliot, Perry M
Garcia-Pavia, Pablo
author de Frutos, Fernando
author_facet de Frutos, Fernando
Ochoa, Juan Pablo
Navarro-Peñalver, Marina
Baas, Annette
Bjerre, Jesper Vandborg
Zorio, Esther
Méndez, Irene
Lorca, Rebeca
Verdonschot, Job A J
García-Granja, Pablo Elpidio
Bilinska, Zofia
Fatkin, Diane
Fuentes-Cañamero, M Eugenia
García-Pinilla, José M
García-Álvarez, María I
Girolami, Francesca
Barriales-Villa, Roberto
Díez-López, Carles
Lopes, Luis R
Wahbi, Karim
García-Álvarez, Ana
Rodríguez-Sánchez, Ibon
Rekondo-Olaetxea, Javier
Rodríguez-Palomares, José F
Gallego-Delgado, María
Meder, Benjamin
Kubanek, Milos
Hansen, Frederikke G
Restrepo-Córdoba, María Alejandra
Palomino-Doza, Julián
Ruiz-Guerrero, Luis
Sarquella-Brugada, Georgia
Perez-Perez, Alberto José
Bermúdez-Jiménez, Francisco José
Ripoll-Vera, Tomas
Rasmussen, Torsten Bloch
Jansen, Mark
Sabater-Molina, Maria
Elliot, Perry M
Garcia-Pavia, Pablo
author_role author
author2 Ochoa, Juan Pablo
Navarro-Peñalver, Marina
Baas, Annette
Bjerre, Jesper Vandborg
Zorio, Esther
Méndez, Irene
Lorca, Rebeca
Verdonschot, Job A J
García-Granja, Pablo Elpidio
Bilinska, Zofia
Fatkin, Diane
Fuentes-Cañamero, M Eugenia
García-Pinilla, José M
García-Álvarez, María I
Girolami, Francesca
Barriales-Villa, Roberto
Díez-López, Carles
Lopes, Luis R
Wahbi, Karim
García-Álvarez, Ana
Rodríguez-Sánchez, Ibon
Rekondo-Olaetxea, Javier
Rodríguez-Palomares, José F
Gallego-Delgado, María
Meder, Benjamin
Kubanek, Milos
Hansen, Frederikke G
Restrepo-Córdoba, María Alejandra
Palomino-Doza, Julián
Ruiz-Guerrero, Luis
Sarquella-Brugada, Georgia
Perez-Perez, Alberto José
Bermúdez-Jiménez, Francisco José
Ripoll-Vera, Tomas
Rasmussen, Torsten Bloch
Jansen, Mark
Sabater-Molina, Maria
Elliot, Perry M
Garcia-Pavia, Pablo
author2_role author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
dc.contributor.none.fl_str_mv Instituto de Salud Carlos III
Unión Europea. Fondo Europeo de Desarrollo Regional (FEDER/ERDF)
Ministerio de Ciencia e Innovación (España)
Fundación ProCNIC
Ministerio de Ciencia e Innovación. Centro de Excelencia Severo Ochoa (España)
European Reference Network for Rare and Low Prevalence Complex Diseases of the Heart
Unión Europea. Comisión Europea. H2020. ERA-CVD
Dutch Heart Foundation (Holanda)
Victor Chang Cardiac Research Institute
NSW Health
Clinical Academic Research Partnerships (CARP)
Deutsches Zentrum für Herz-Kreislauf-Forschung (German Center for Cardiovascular Research)
Informatics for Life (Klaus Tschira Foundation)
Ministry of Health (República Checa)
Institute for Clinical and Experimental Medicine–IKEM

dc.subject.none.fl_str_mv Cardiomyopathy, Dilated
Heart Failure
Myosin Heavy Chains
Adolescent
Adult
Arrhythmias, Cardiac
Cardiac Myosins
Female
Humans
Male
Middle Aged
Phenotype
Ventricular Remodeling
Young Adult
topic Cardiomyopathy, Dilated
Heart Failure
Myosin Heavy Chains
Adolescent
Adult
Arrhythmias, Cardiac
Cardiac Myosins
Female
Humans
Male
Middle Aged
Phenotype
Ventricular Remodeling
Young Adult
description Variants in myosin heavy chain 7 (MYH7) are responsible for disease in 1% to 5% of patients with dilated cardiomyopathy (DCM); however, the clinical characteristics and natural history of MYH7-related DCM are poorly described. We sought to determine the phenotype and prognosis of MYH7-related DCM. We also evaluated the influence of variant location on phenotypic expression. We studied clinical data from 147 individuals with DCM-causing MYH7 variants (47.6% female; 35.6 ± 19.2 years) recruited from 29 international centers. At initial evaluation, 106 (72.1%) patients had DCM (left ventricular ejection fraction: 34.5% ± 11.7%). Median follow-up was 4.5 years (IQR: 1.7-8.0 years), and 23.7% of carriers who were initially phenotype-negative developed DCM. Phenotypic expression by 40 and 60 years was 46% and 88%, respectively, with 18 patients (16%) first diagnosed at <18 years of age. Thirty-six percent of patients with DCM met imaging criteria for LV noncompaction. During follow-up, 28% showed left ventricular reverse remodeling. Incidence of adverse cardiac events among patients with DCM at 5 years was 11.6%, with 5 (4.6%) deaths caused by end-stage heart failure (ESHF) and 5 patients (4.6%) requiring heart transplantation. The major ventricular arrhythmia rate was low (1.0% and 2.1% at 5 years in patients with DCM and in those with LVEF of ≤35%, respectively). ESHF and major ventricular arrhythmia were significantly lower compared with LMNA-related DCM and similar to DCM caused by TTN truncating variants. MYH7-related DCM is characterized by early age of onset, high phenotypic expression, low left ventricular reverse remodeling, and frequent progression to ESHF. Heart failure complications predominate over ventricular arrhythmias, which are rare.
publishDate 2022
dc.date.none.fl_str_mv 2022
2022-11-29
2022
2022-10-11
2022
2022-10-11
dc.type.none.fl_str_mv journal article
http://purl.org/coar/resource_type/c_6501
VoR
http://purl.org/coar/version/c_970fb48d4fbd8a85
dc.type.openaire.fl_str_mv info:eu-repo/semantics/article
format article
dc.identifier.none.fl_str_mv http://hdl.handle.net/20.500.12105/15246
url http://hdl.handle.net/20.500.12105/15246
dc.language.none.fl_str_mv Inglés
eng
language_invalid_str_mv Inglés
language eng
dc.rights.none.fl_str_mv open access
http://purl.org/coar/access_right/c_abf2
Atribución 4.0 Internacional
http://creativecommons.org/licenses/by/4.0/
dc.rights.openaire.fl_str_mv info:eu-repo/semantics/openAccess
rights_invalid_str_mv open access
http://purl.org/coar/access_right/c_abf2
Atribución 4.0 Internacional
http://creativecommons.org/licenses/by/4.0/
eu_rights_str_mv openAccess
dc.format.none.fl_str_mv application/pdf
dc.publisher.none.fl_str_mv Elsevier
publisher.none.fl_str_mv Elsevier
dc.source.none.fl_str_mv reponame:Repisalud
instname:Instituto de Salud Carlos III (ISCIII)
instname_str Instituto de Salud Carlos III (ISCIII)
reponame_str Repisalud
collection Repisalud
repository.name.fl_str_mv
repository.mail.fl_str_mv
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