A novel treatment approach for paediatric Gorham-Stout syndrome with chylothorax

Acta Paediatr. 2011 Nov;100(11):1448-53. doi: 10.1111/j.1651-2227.2011.02361.x. Epub 2011 Jun 15.

Abstract

Aim: To expand the treatment options in paediatric Gorham-Stout syndrome (GSS) when conventional therapy is ineffective.

Method: Two children with biopsy confirmed GSS, a rare disorder with progressive lymphangiomatosis, were treated with a combination of interferon-α-2b, low anticoagulant, low molecular weight heparin, radiotherapy and surgery.

Results: The combined therapy resolved the symptoms in the acute phase, and both patients have since been free of symptoms for >2 years.

Conclusion: The successful addition of a low anticoagulant, low molecular weight heparin (tafoxiparin) to the treatment protocol in two paediatric cases of the GSS may justify the use of this approach in similar cases.

Publication types

  • Case Reports

MeSH terms

  • Anticoagulants / therapeutic use
  • Child, Preschool
  • Chylothorax / etiology
  • Chylothorax / therapy*
  • Female
  • Heparin, Low-Molecular-Weight / therapeutic use
  • Humans
  • Interferon-alpha / therapeutic use
  • Lymphangioleiomyomatosis / therapy*
  • Male
  • Osteolysis, Essential / complications
  • Osteolysis, Essential / therapy*
  • Radiotherapy
  • Salvage Therapy
  • Surgical Procedures, Operative
  • Syndrome
  • Thoracoscopy
  • Thoracostomy
  • Vascular Endothelial Growth Factors / blood

Substances

  • Anticoagulants
  • Heparin, Low-Molecular-Weight
  • Interferon-alpha
  • Vascular Endothelial Growth Factors