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Unclassifiable arrhythmic cardiomyopathy associated with Emery-Dreifuss caused by a mutation in FHL1.
San Román, I; Navarro, M; Martínez, F; Albert, L; Polo, L; Guardiola, J; García-Molina, E; Muñoz-Esparza, C; López-Ayala, J M; Sabater-Molina, M; Gimeno, J R.
Afiliación
  • San Román I; Inherited Cardiac Disease Unit, University Hospital Virgen de la Arrixaca, Murcia, Spain.
  • Navarro M; Inherited Cardiac Disease Unit, University Hospital Virgen de la Arrixaca, Murcia, Spain.
  • Martínez F; Neurology Department, University Hospital Virgen de la Arrixaca, Murcia, Spain.
  • Albert L; Inherited Cardiac Disease Unit, University Hospital Virgen de la Arrixaca, Murcia, Spain.
  • Polo L; Pathology Department, University Hospital Virgen de la Arrixaca, Murcia, Spain.
  • Guardiola J; Pneumology Department, University Hospital Virgen de la Arrixaca, Murcia, Spain.
  • García-Molina E; Inherited Cardiac Disease Unit, University Hospital Virgen de la Arrixaca, Murcia, Spain.
  • Muñoz-Esparza C; Inherited Cardiac Disease Unit, University Hospital Virgen de la Arrixaca, Murcia, Spain.
  • López-Ayala JM; Inherited Cardiac Disease Unit, University Hospital Virgen de la Arrixaca, Murcia, Spain.
  • Sabater-Molina M; Inherited Cardiac Disease Unit, University Hospital Virgen de la Arrixaca, Murcia, Spain.
  • Gimeno JR; Internal Medicine Department, University of Murcia, Murcia, Spain.
Clin Genet ; 90(2): 171-6, 2016 08.
Article en En | MEDLINE | ID: mdl-26857240
Emery-Dreifuss muscular dystrophy (EDMD) is a heterogeneous genetic disorder characterized by peripheral muscular weakness often associated with dilated cardiomyopathy. We characterize clinically a large family with a mutation in FHL1 gene (p.Cys255Ser). Penetrance was 44%, 100% for males and 18% for females. The heart was the main organ involved. Affected adult males had mild hypertrophy, systolic dysfunction and restriction with non-dilated ventricles. Carriers had significant QTc prolongation. The proband presented with resuscitated cardiac arrest. There were two transplants. Pathological study of explanted heart showed fibrofatty replacement and scarring consistent with arrhythmogenic cardiomyopathy and prominent left ventricular trabeculations. Myopathic involvement was evident in all males. Females had no significant neuromuscular disease. Mutations in FHL1 cause unclassifiable cardiomyopathy with coexisting EDMD. Prognosis is poor and systolic impairment and arrhythmias are frequent. Thrombopenia and raised creatine phosphokinase should raise suspicion of an FHL-1 disorder in X-linked cardiomyopathy.
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Arritmias Cardíacas / Cardiomiopatía Dilatada / Distrofia Muscular de Emery-Dreifuss / Péptidos y Proteínas de Señalización Intracelular / Proteínas con Dominio LIM / Proteínas Musculares / Mutación Tipo de estudio: Prognostic_studies / Risk_factors_studies Límite: Adolescent / Adult / Aged / Child / Child, preschool / Female / Humans / Male / Middle aged Idioma: En Revista: Clin Genet Año: 2016 Tipo del documento: Article País de afiliación: España Pais de publicación: Dinamarca

Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Arritmias Cardíacas / Cardiomiopatía Dilatada / Distrofia Muscular de Emery-Dreifuss / Péptidos y Proteínas de Señalización Intracelular / Proteínas con Dominio LIM / Proteínas Musculares / Mutación Tipo de estudio: Prognostic_studies / Risk_factors_studies Límite: Adolescent / Adult / Aged / Child / Child, preschool / Female / Humans / Male / Middle aged Idioma: En Revista: Clin Genet Año: 2016 Tipo del documento: Article País de afiliación: España Pais de publicación: Dinamarca