Propranolol as a treatment option in Gorham-Stout syndrome: a case report

Vered Nir1, Ludmila Guralnik, Galit Livnat

  • 1Pediatric Pulmonary Unit, Meyer Children's Hospital, Rambam Health Care Campus, Israel; Pediatric Department, Hillel Yaffe Medical Center, Hadera, Israel.

Pediatric Pulmonology
|September 4, 2013
PubMed

Insights

Gorham-Stout syndrome, a rare condition causing blood and lymphatic vessel overgrowth, showed improvement with propranolol. This suggests a potential new treatment for this complex disease.

Area of Science:

  • Vascular anomalies
  • Pediatric rare diseases
  • Medical case study

Background:

  • Gorham-Stout syndrome is a rare, progressive condition involving widespread angiomatosis and lymphangiomas.
  • Current treatments like interferon and bisphosphonates have limited efficacy.

Observation:

  • A pediatric case of Gorham-Stout syndrome is presented, diagnosed at age 13.
  • The disease affected multiple organs including the mediastinum, pericardium, spine, ribs, and skull.
  • Initial treatments with interferon and bisphosphonates were ineffective.

Findings:

  • Propranolol treatment was initiated at age 18.
  • Significant improvement in pulmonary function tests was observed.
  • Mediastinal and hilar hemangiomatous lesions showed involution following propranolol administration.

Implications:

  • Propranolol demonstrates potential therapeutic benefits in Gorham-Stout syndrome.
  • Further investigation into propranolol's efficacy for this rare condition is warranted.
  • This case highlights a possible novel treatment avenue for complex vascular anomalies.

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