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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.
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.
Abstract:
Gorham-Stout syndrome is a very rare syndrome characterized by progressive angiomatosis and lymphangiomas involving multiple organs. We describe herein a girl with progressive Gorham-Stout syndrome since the age of 13 years. Her disease involved the mediastinum, pericardium, vertebrae, ribs, and skull and did not respond to interferon and bisphosphonates. Propranolol administration was initiated at the age of 18 years and was associated with improvement in pulmonary function tests and involution of the mediastinal and hilar hemangiomatous lesions. The possible beneficial effect of propranolol in Gorham-Stout syndrome should be further investigated.
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