Formin Homology 2 Domain Containing 3 (FHOD3) Is a Genetic Basis for Hypertrophic Cardiomyopathy

BACKGROUND The genetic cause of hypertrophic cardiomyopathy remains unexplained in a substantial proportion of cases. Formin homology 2 domain containing 3 (FHOD3) may have a role in the pathogenesis of cardiac hypertrophy but has not been implicated in hypertrophic cardiomyopathy. OBJECTIVES This s...

Descripción completa

Detalles Bibliográficos
Autores: Pablo Ochoa, Juan, Sabater-Molina M, Manuel Garcia-Pinilla, Jose, Mogensen J, Restrepo-Córdoba A, Palomino-Doza J, Villacorta E, Martinez-Moreno M, Ramos-Maqueda J, Zorio E, Pena-Pena, Maria L., García-Granja PE, Rodríguez-Palomares JF, Cardenas-Reyes, Ivonne J., de la Torre-Carpente, Maria M., Bautista-Paves, Alicia, Akhtar, Mohammed M., Cicerchia, Marcos N., Bilbao-Quesada, Raquel, Victoria Mogollon-Jimenez, Maria, Salazar-Mendiguchia, Joel, Mesa Latorre, Jose M., Arnaez, Blanca, Olavarri-Miguel, Ivan, Fuentes-Canamero, Maria E., Lamounier, Jr., Arsonval, Perez Ruiz, Jose Maria, Climent-Paya, Vicente, Perez-Sanchez, Inmaculada, Trujillo-Quintero, Juan P., Lopes, Luis R., Reparaz-Andrade, Alfredo, Marin-Iglesias, Rosario, Rodriguez-Vilela, Alejandro, Sandin-Fuentes, Maria, Garrote, Jose A., Cortel-Fuster, Alejandro, Lopez-Garrido, Miguel, Fontalba-Romero, Ana, Ripoll-Vera, Tomas, Llano-Rivas, Isabel, Fernandez-Fernandez, Xusto, Isidoro-Garcia, Maria, Garcia-Giustiniani, Diego, Barriales-Villa, Roberto, Ortiz-Genga, Martin, Garcia-Pavia, Pablo, Elliott, Perry M., Gimeno, Juan R., Monserrat, Lorenzo
Tipo de recurso: artículo
Estado:Versión publicada
Fecha de publicación:2018
País:España
Institución:Instituto de Investigación Biomédica y Sanitaria de Alicante (ISABIAL)
Repositorio:r-ISABIAL. Repositorio Institucional de Producción Científica del Instituto de Investigación Biomédica y Sanitaria de Alicante
OAI Identifier:oai:isabial.fundanetsuite.com:p2878
Acceso en línea:https://isabial.portalinvestigacion.com/publicaciones2878
Access Level:acceso abierto
Palabra clave:cardiomyopathies
FHOD3
formins
genetics
hypertrophic cardiomyopathy
sudden death
id ES_012daa50fa8b0b0b4dae46e408dcb95d
oai_identifier_str oai:isabial.fundanetsuite.com:p2878
network_acronym_str ES
network_name_str España
repository_id_str
dc.title.none.fl_str_mv Formin Homology 2 Domain Containing 3 (FHOD3) Is a Genetic Basis for Hypertrophic Cardiomyopathy
title Formin Homology 2 Domain Containing 3 (FHOD3) Is a Genetic Basis for Hypertrophic Cardiomyopathy
spellingShingle Formin Homology 2 Domain Containing 3 (FHOD3) Is a Genetic Basis for Hypertrophic Cardiomyopathy
Pablo Ochoa, Juan
cardiomyopathies
FHOD3
formins
genetics
hypertrophic cardiomyopathy
sudden death
title_short Formin Homology 2 Domain Containing 3 (FHOD3) Is a Genetic Basis for Hypertrophic Cardiomyopathy
title_full Formin Homology 2 Domain Containing 3 (FHOD3) Is a Genetic Basis for Hypertrophic Cardiomyopathy
title_fullStr Formin Homology 2 Domain Containing 3 (FHOD3) Is a Genetic Basis for Hypertrophic Cardiomyopathy
title_full_unstemmed Formin Homology 2 Domain Containing 3 (FHOD3) Is a Genetic Basis for Hypertrophic Cardiomyopathy
title_sort Formin Homology 2 Domain Containing 3 (FHOD3) Is a Genetic Basis for Hypertrophic Cardiomyopathy
dc.creator.none.fl_str_mv Pablo Ochoa, Juan
Sabater-Molina M
Manuel Garcia-Pinilla, Jose
Mogensen J
Restrepo-Córdoba A
Palomino-Doza J
Villacorta E
Martinez-Moreno M
Ramos-Maqueda J
Zorio E
Pena-Pena, Maria L.
García-Granja PE
Rodríguez-Palomares JF
Cardenas-Reyes, Ivonne J.
de la Torre-Carpente, Maria M.
Bautista-Paves, Alicia
Akhtar, Mohammed M.
Cicerchia, Marcos N.
Bilbao-Quesada, Raquel
Victoria Mogollon-Jimenez, Maria
Salazar-Mendiguchia, Joel
Mesa Latorre, Jose M.
Arnaez, Blanca
Olavarri-Miguel, Ivan
Fuentes-Canamero, Maria E.
Lamounier, Jr., Arsonval
Perez Ruiz, Jose Maria
Climent-Paya, Vicente
Perez-Sanchez, Inmaculada
Trujillo-Quintero, Juan P.
Lopes, Luis R.
Reparaz-Andrade, Alfredo
Marin-Iglesias, Rosario
Rodriguez-Vilela, Alejandro
Sandin-Fuentes, Maria
Garrote, Jose A.
Cortel-Fuster, Alejandro
Lopez-Garrido, Miguel
Fontalba-Romero, Ana
Ripoll-Vera, Tomas
Llano-Rivas, Isabel
Fernandez-Fernandez, Xusto
Isidoro-Garcia, Maria
Garcia-Giustiniani, Diego
Barriales-Villa, Roberto
Ortiz-Genga, Martin
Garcia-Pavia, Pablo
Elliott, Perry M.
Gimeno, Juan R.
Monserrat, Lorenzo
author Pablo Ochoa, Juan
author_facet Pablo Ochoa, Juan
Sabater-Molina M
Manuel Garcia-Pinilla, Jose
Mogensen J
Restrepo-Córdoba A
Palomino-Doza J
Villacorta E
Martinez-Moreno M
Ramos-Maqueda J
Zorio E
Pena-Pena, Maria L.
García-Granja PE
Rodríguez-Palomares JF
Cardenas-Reyes, Ivonne J.
de la Torre-Carpente, Maria M.
Bautista-Paves, Alicia
Akhtar, Mohammed M.
Cicerchia, Marcos N.
Bilbao-Quesada, Raquel
Victoria Mogollon-Jimenez, Maria
Salazar-Mendiguchia, Joel
Mesa Latorre, Jose M.
Arnaez, Blanca
Olavarri-Miguel, Ivan
Fuentes-Canamero, Maria E.
Lamounier, Jr., Arsonval
Perez Ruiz, Jose Maria
Climent-Paya, Vicente
Perez-Sanchez, Inmaculada
Trujillo-Quintero, Juan P.
Lopes, Luis R.
Reparaz-Andrade, Alfredo
Marin-Iglesias, Rosario
Rodriguez-Vilela, Alejandro
Sandin-Fuentes, Maria
Garrote, Jose A.
Cortel-Fuster, Alejandro
Lopez-Garrido, Miguel
Fontalba-Romero, Ana
Ripoll-Vera, Tomas
Llano-Rivas, Isabel
Fernandez-Fernandez, Xusto
Isidoro-Garcia, Maria
Garcia-Giustiniani, Diego
Barriales-Villa, Roberto
Ortiz-Genga, Martin
Garcia-Pavia, Pablo
Elliott, Perry M.
Gimeno, Juan R.
Monserrat, Lorenzo
author_role author
author2 Sabater-Molina M
Manuel Garcia-Pinilla, Jose
Mogensen J
Restrepo-Córdoba A
Palomino-Doza J
Villacorta E
Martinez-Moreno M
Ramos-Maqueda J
Zorio E
Pena-Pena, Maria L.
García-Granja PE
Rodríguez-Palomares JF
Cardenas-Reyes, Ivonne J.
de la Torre-Carpente, Maria M.
Bautista-Paves, Alicia
Akhtar, Mohammed M.
Cicerchia, Marcos N.
Bilbao-Quesada, Raquel
Victoria Mogollon-Jimenez, Maria
Salazar-Mendiguchia, Joel
Mesa Latorre, Jose M.
Arnaez, Blanca
Olavarri-Miguel, Ivan
Fuentes-Canamero, Maria E.
Lamounier, Jr., Arsonval
Perez Ruiz, Jose Maria
Climent-Paya, Vicente
Perez-Sanchez, Inmaculada
Trujillo-Quintero, Juan P.
Lopes, Luis R.
Reparaz-Andrade, Alfredo
Marin-Iglesias, Rosario
Rodriguez-Vilela, Alejandro
Sandin-Fuentes, Maria
Garrote, Jose A.
Cortel-Fuster, Alejandro
Lopez-Garrido, Miguel
Fontalba-Romero, Ana
Ripoll-Vera, Tomas
Llano-Rivas, Isabel
Fernandez-Fernandez, Xusto
Isidoro-Garcia, Maria
Garcia-Giustiniani, Diego
Barriales-Villa, Roberto
Ortiz-Genga, Martin
Garcia-Pavia, Pablo
Elliott, Perry M.
Gimeno, Juan R.
Monserrat, Lorenzo
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
author
author
author
author
author
author
author
author
author
author
dc.subject.none.fl_str_mv cardiomyopathies
FHOD3
formins
genetics
hypertrophic cardiomyopathy
sudden death
topic cardiomyopathies
FHOD3
formins
genetics
hypertrophic cardiomyopathy
sudden death
description BACKGROUND The genetic cause of hypertrophic cardiomyopathy remains unexplained in a substantial proportion of cases. Formin homology 2 domain containing 3 (FHOD3) may have a role in the pathogenesis of cardiac hypertrophy but has not been implicated in hypertrophic cardiomyopathy. OBJECTIVES This study sought to investigate the relation between FHOD3 mutations and the development of hypertrophic cardiomyopathy. METHODS FHOD3 was sequenced by massive parallel sequencing in 3,189 hypertrophic cardiomyopathy unrelated probands and 2,777 patients with no evidence of cardiomyopathy (disease control subjects). The authors evaluated protein-altering candidate variants in FHOD3 for cosegregation, clinical characteristics, and outcomes. RESULTS The authors identified 94 candidate variants in 132 probands. The variants' frequencies were significantly higher in patients with hypertrophic cardiomyopathy (74 of 3,189 [2.32%]) than in disease control subjects (18 of 2,777 [0.65%]; p < 0.001) or in the gnomAD database (1,049 of 138,606 [0.76%]; p < 0.001). FHOD3 mutations cosegregated with hypertrophic cardiomyopathy in 17 families, with a combined logarithm of the odds score of 7.92, indicative of very strong segregation. One-half of the disease-causing variants were clustered in a small conserved coiled-coil domain (amino acids 622 to 655); odds ratio for hypertrophic cardiomyopathy was 21.8 versus disease control subjects (95% confidence interval: 1.3 to 37.9; p < 0.001) and 14.1 against gnomAD (95% confidence interval: 6.9 to 28.7; p < 0.001). Hypertrophic cardiomyopathy patients carrying (likely) pathogenic mutations in FHOD3 (n = 70) were diagnosed after age 30 years (mean 46.1 +/- 18.7 years), and two-thirds (66%) were males. Of the patients, 82% had asymmetric septal hypertrophy (mean 18.8 +/- 5 mm); left ventricular ejection fraction <50% was present in 14% and hypertrabeculation in 16%. Events were rare before age 30 years, with an annual cardiovascular death incidence of 1% during follow-up. CONCLUSIONS FHOD3 is a novel disease gene in hypertrophic cardiomyopathy, accounting for approximately 1% to 2% of cases. The phenotype and the rate of cardiovascular events are similar to those reported in unselected cohorts. The FHOD3 gene should be routinely included in hypertrophic cardiomyopathy genetic testing panels. (C) 2018 The Authors. Published by Elsevier on behalf of the American College of Cardiology Foundation. This is an open access article under the CC BY-NC-ND license (http://creativecommons.org/licenses/by-nc-nd/4.0/).
publishDate 2018
dc.date.none.fl_str_mv 2018
dc.type.none.fl_str_mv info:eu-repo/semantics/article
info:eu-repo/semantics/publishedVersion
format article
status_str publishedVersion
dc.identifier.none.fl_str_mv https://isabial.portalinvestigacion.com/publicaciones2878
url https://isabial.portalinvestigacion.com/publicaciones2878
dc.language.none.fl_str_mv Inglés
language_invalid_str_mv Inglés
dc.rights.none.fl_str_mv info:eu-repo/semantics/openAccess
eu_rights_str_mv openAccess
dc.publisher.none.fl_str_mv ELSEVIER SCIENCE INC
publisher.none.fl_str_mv ELSEVIER SCIENCE INC
dc.source.none.fl_str_mv JACC-JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY
ISSN: 07351097
ISSNe: 15583597
reponame:r-ISABIAL. Repositorio Institucional de Producción Científica del Instituto de Investigación Biomédica y Sanitaria de Alicante
instname:Instituto de Investigación Biomédica y Sanitaria de Alicante (ISABIAL)
instname_str Instituto de Investigación Biomédica y Sanitaria de Alicante (ISABIAL)
reponame_str r-ISABIAL. Repositorio Institucional de Producción Científica del Instituto de Investigación Biomédica y Sanitaria de Alicante
collection r-ISABIAL. Repositorio Institucional de Producción Científica del Instituto de Investigación Biomédica y Sanitaria de Alicante
repository.name.fl_str_mv
repository.mail.fl_str_mv
_version_ 1869402563336470528
spelling Formin Homology 2 Domain Containing 3 (FHOD3) Is a Genetic Basis for Hypertrophic CardiomyopathyPablo Ochoa, JuanSabater-Molina MManuel Garcia-Pinilla, JoseMogensen JRestrepo-Córdoba APalomino-Doza JVillacorta EMartinez-Moreno MRamos-Maqueda JZorio EPena-Pena, Maria L.García-Granja PERodríguez-Palomares JFCardenas-Reyes, Ivonne J.de la Torre-Carpente, Maria M.Bautista-Paves, AliciaAkhtar, Mohammed M.Cicerchia, Marcos N.Bilbao-Quesada, RaquelVictoria Mogollon-Jimenez, MariaSalazar-Mendiguchia, JoelMesa Latorre, Jose M.Arnaez, BlancaOlavarri-Miguel, IvanFuentes-Canamero, Maria E.Lamounier, Jr., ArsonvalPerez Ruiz, Jose MariaCliment-Paya, VicentePerez-Sanchez, InmaculadaTrujillo-Quintero, Juan P.Lopes, Luis R.Reparaz-Andrade, AlfredoMarin-Iglesias, RosarioRodriguez-Vilela, AlejandroSandin-Fuentes, MariaGarrote, Jose A.Cortel-Fuster, AlejandroLopez-Garrido, MiguelFontalba-Romero, AnaRipoll-Vera, TomasLlano-Rivas, IsabelFernandez-Fernandez, XustoIsidoro-Garcia, MariaGarcia-Giustiniani, DiegoBarriales-Villa, RobertoOrtiz-Genga, MartinGarcia-Pavia, PabloElliott, Perry M.Gimeno, Juan R.Monserrat, LorenzocardiomyopathiesFHOD3forminsgeneticshypertrophic cardiomyopathysudden deathBACKGROUND The genetic cause of hypertrophic cardiomyopathy remains unexplained in a substantial proportion of cases. Formin homology 2 domain containing 3 (FHOD3) may have a role in the pathogenesis of cardiac hypertrophy but has not been implicated in hypertrophic cardiomyopathy. OBJECTIVES This study sought to investigate the relation between FHOD3 mutations and the development of hypertrophic cardiomyopathy. METHODS FHOD3 was sequenced by massive parallel sequencing in 3,189 hypertrophic cardiomyopathy unrelated probands and 2,777 patients with no evidence of cardiomyopathy (disease control subjects). The authors evaluated protein-altering candidate variants in FHOD3 for cosegregation, clinical characteristics, and outcomes. RESULTS The authors identified 94 candidate variants in 132 probands. The variants' frequencies were significantly higher in patients with hypertrophic cardiomyopathy (74 of 3,189 [2.32%]) than in disease control subjects (18 of 2,777 [0.65%]; p < 0.001) or in the gnomAD database (1,049 of 138,606 [0.76%]; p < 0.001). FHOD3 mutations cosegregated with hypertrophic cardiomyopathy in 17 families, with a combined logarithm of the odds score of 7.92, indicative of very strong segregation. One-half of the disease-causing variants were clustered in a small conserved coiled-coil domain (amino acids 622 to 655); odds ratio for hypertrophic cardiomyopathy was 21.8 versus disease control subjects (95% confidence interval: 1.3 to 37.9; p < 0.001) and 14.1 against gnomAD (95% confidence interval: 6.9 to 28.7; p < 0.001). Hypertrophic cardiomyopathy patients carrying (likely) pathogenic mutations in FHOD3 (n = 70) were diagnosed after age 30 years (mean 46.1 +/- 18.7 years), and two-thirds (66%) were males. Of the patients, 82% had asymmetric septal hypertrophy (mean 18.8 +/- 5 mm); left ventricular ejection fraction <50% was present in 14% and hypertrabeculation in 16%. Events were rare before age 30 years, with an annual cardiovascular death incidence of 1% during follow-up. CONCLUSIONS FHOD3 is a novel disease gene in hypertrophic cardiomyopathy, accounting for approximately 1% to 2% of cases. The phenotype and the rate of cardiovascular events are similar to those reported in unselected cohorts. The FHOD3 gene should be routinely included in hypertrophic cardiomyopathy genetic testing panels. (C) 2018 The Authors. Published by Elsevier on behalf of the American College of Cardiology Foundation. This is an open access article under the CC BY-NC-ND license (http://creativecommons.org/licenses/by-nc-nd/4.0/).ELSEVIER SCIENCE INC2018info:eu-repo/semantics/articleinfo:eu-repo/semantics/publishedVersionhttps://isabial.portalinvestigacion.com/publicaciones2878JACC-JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGYISSN: 07351097ISSNe: 15583597reponame:r-ISABIAL. Repositorio Institucional de Producción Científica del Instituto de Investigación Biomédica y Sanitaria de Alicanteinstname:Instituto de Investigación Biomédica y Sanitaria de Alicante (ISABIAL)Inglésinfo:eu-repo/semantics/openAccessoai:isabial.fundanetsuite.com:p28782026-06-12T10:20:37Z
score 15,81155