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Holt-Oram syndrome

Insights

Holt-Oram syndrome links upper limb skeletal defects with congenital heart disease. This study highlights left-sided asymmetry and the potential for transmission from affected individuals, even with isolated skeletal issues.

Area of Science:

  • Genetics
  • Cardiology
  • Orthopedics

Background:

  • Holt-Oram syndrome is an autosomal dominant disorder.
  • It involves upper extremity skeletal malformations and congenital heart disease.
  • The severity of skeletal and cardiac defects often varies.

Purpose of the Study:

  • To review clinical experiences with Holt-Oram syndrome.
  • To identify and emphasize previously under-recognized features of the syndrome.
  • To improve understanding of its genetic transmission and clinical presentation.

Main Methods:

  • Retrospective review of 39 patients with Holt-Oram syndrome.
  • Analysis of skeletal and cardiac defect types and severity.
  • Assessment of family history and mutation status.

Main Results:

  • Observed wide variability in skeletal and cardiac defects.
  • Noted striking left-sided asymmetry in skeletal involvement.
  • Identified transmission of both skeletal and cardiac defects from parents with only skeletal issues.
  • Found hypoplastic peripheral vessels complicating cardiac catheterization.
  • Observed inconsistent electrocardiographic findings in secundum atrial septal defects.

Conclusions:

  • Holt-Oram syndrome exhibits significant phenotypic variability.
  • Asymmetry, particularly left-sided, is a notable feature.
  • Genetic transmission can occur even with isolated skeletal abnormalities.
  • Associated vascular anomalies and diagnostic challenges exist.
  • ECG findings may not be uniformly present in all cases.

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