Your browser doesn't support javascript.
loading
Delineation of the clinical and radiological features of Stuve-Wiedemann syndrome childhood survivors, four new cases and review of the literature.
Siccha, Sofía M; Cueto, Anna María; Parrón-Pajares, Manuel; González-Morán, Gaspar; Pacio-Miguez, Marta; Del Pozo, Ángela; Solís, Mario; Rodriguez-Jimenez, Carmen; Caino, Silvia; Fano, Virginia; Heath, Karen E; García-Miñaúr, Sixto; Palomares-Bralo, María; Santos-Simarro, Fernando.
Afiliación
  • Siccha SM; Institute of Medical & Molecular Genetics (INGEMM), Hospital Universitario La Paz, Universidad Autónoma de Madrid, IdiPAZ, Madrid, Spain.
  • Cueto AM; Department of Pediatrics, Hospital Universitario la Paz, Madrid, Spain.
  • Parrón-Pajares M; Department of Clinical and Molecular Genetics, Vall d'Hebron Hospital Universitari, Vall d'Hebron Barcelona Hospital Campus, Barcelona, Spain.
  • González-Morán G; Medical Genetics Group, Vall d'Hebron Institut de Recerca (VHIR), Vall d'Hebron Hospital Universitari, Vall d'Hebron Barcelona Hospital Campus, Barcelona, Spain.
  • Pacio-Miguez M; European Reference Network, ERN CRANIO, Barcelona, Spain.
  • Del Pozo Á; Department of Radiology, Hospital Universitario La Paz, Madrid, Spain.
  • Solís M; Skeletal Dysplasia Multidisciplinary Unit (UMDE) and ERN BOND, Hospital Universitario La Paz, Madrid, Spain.
  • Rodriguez-Jimenez C; European Reference Network, ERN ITHACA, Madrid, Spain.
  • Caino S; Skeletal Dysplasia Multidisciplinary Unit (UMDE) and ERN BOND, Hospital Universitario La Paz, Madrid, Spain.
  • Fano V; European Reference Network, ERN ITHACA, Madrid, Spain.
  • Heath KE; Department of Orthopaedic Surgery and Traumatology, Hospital Universitario la Paz, Madrid, Spain.
  • García-Miñaúr S; Institute of Medical & Molecular Genetics (INGEMM), Hospital Universitario La Paz, Universidad Autónoma de Madrid, IdiPAZ, Madrid, Spain.
  • Palomares-Bralo M; Institute of Medical & Molecular Genetics (INGEMM), Hospital Universitario La Paz, Universidad Autónoma de Madrid, IdiPAZ, Madrid, Spain.
  • Santos-Simarro F; European Reference Network, ERN ITHACA, Madrid, Spain.
Am J Med Genet A ; 185(3): 856-865, 2021 03.
Article en En | MEDLINE | ID: mdl-33305909
Stuve-Wiedemann syndrome (SWS; MIM 601559) is a rare autosomal recessive disease caused by mutations in the leukemia inhibitor factor receptor gene (LIFR). Common clinical and radiological findings are often observed, and high neonatal mortality occurs due to respiratory distress and hyperthermic episodes. Despite initially considered as a lethal disorder during the newborn period, in recent years, several SWS childhood survivors have been reported. We report a detailed clinical and radiological characterization of four unrelated childhood SWS molecularly confirmed patients and review 22 previously reported childhood surviving cases. We contribute to the definition of the childhood survival phenotype of SWS, emphasizing the evolving phenotype, characterized by skeletal abnormalities with typical radiological findings, distinctive dysmorphic features, and dysautonomia. Based on the typical features and clinical course, early diagnosis is possible and crucial to plan appropriate management and prevent potential complications. Genetic confirmation is advisable in order to improve genetic counseling to the patients and their families.
Asunto(s)
Palabras clave

Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Asunto principal: Osteocondrodisplasias / Anomalías Múltiples / Enfermedades del Desarrollo Óseo / Exostosis Múltiple Hereditaria / Subunidad alfa del Receptor del Factor Inhibidor de Leucemia Tipo de estudio: Screening_studies Límite: Child, preschool / Female / Humans / Infant / Male / Newborn Idioma: En Revista: Am J Med Genet A Asunto de la revista: GENETICA MEDICA Año: 2021 Tipo del documento: Article País de afiliación: España

Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Asunto principal: Osteocondrodisplasias / Anomalías Múltiples / Enfermedades del Desarrollo Óseo / Exostosis Múltiple Hereditaria / Subunidad alfa del Receptor del Factor Inhibidor de Leucemia Tipo de estudio: Screening_studies Límite: Child, preschool / Female / Humans / Infant / Male / Newborn Idioma: En Revista: Am J Med Genet A Asunto de la revista: GENETICA MEDICA Año: 2021 Tipo del documento: Article País de afiliación: España